Everything You Need to Know About TRACTION-2: Clinical Opportunity for FSGS and MCD Patients June 23, 2022 by Kylie Karley Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on delivering disease-modifying precision medicines that bring hope and renewed quality of life to people living with kidney diseases. We recently spoke with Goldfinch Bio’s Chief Medical Officer, Ed Tucker, MD, who explains more about their Phase 2 clinical research trial for GFB-887, TRACTION-2, which is currently enrolling patients. Goldfinch Bio’s Chief Medical Officer, Ed Tucker, MD What patients are you hoping to enroll for this trial? In the TRACTION-2 study that is currently enrolling, we are looking for patients with focal segmental glomerulosclerosis (FSGS) and treatment resistant minimal change disease (TR-MCD) who are between the ages of 18 and 75 years old, have a UPCR greater than or equal to 1.0 g/g, and an eGFR greater than or equal to 30 mL/min/1.73 m2. Other inclusion and exclusion criteria will apply; please consult your healthcare provider for additional details. Why is FSGS and TR-MCD your target population? In the United States, there are currently no approved therapies for FSGS or MCD. Our treatment, GFB-887, was designed specifically for diseases like FSGS and TR-MCD because it targets and protects the podocytes that are damaged and lost in the disease development and progression. Other diseases involving podocyte injury/loss may be examined in the future as well. If someone enrolled in your Phase 2 study, what is the time commitment? After a patient has been evaluated (for up to 6 weeks) for eligibility in the study they will begin to receive the treatment and will continue for up to a total of 17 visits over approximately 26 weeks. Some of these visits are done via a phone call. After completion of the full study, patients will have the option to enroll in another “extension” study where they will continue to receive treatment for up to three years. An extension study is where all patients receive GFB-887 and allows for doctors to observe the effects of the therapy over a longer period of time. How many trial sites are there available? Where are these sites located? Currently, there are 76 locations throughout the United States where patients may participate in the study. Locations of these sites may be found here. What type of treatment is GFB-887? And what makes this treatment different from other ones being tested in clinical trials? GFB-887 is a tablet and is taken by mouth with water once a day. It differs from other medications in that it was created specifically to target and protect podocytes in the kidney to prevent damage and loss. No other therapies being tested for FSGS and TR-MCD work the same way that GFB-887 does on the kidneys. How is precision medicine integrated in this clinical trial? As this is a phase 2 trial, we are looking at different markers (including testing for genetic) for response to GFB-887, both before and after treatment has begun. Hopefully, we will be able to better integrate these findings into future studies and potentially improve outcomes for patients. Is there flexibility where lab samples can be collected for the participant? Is there an option for home care visits to collect these samples? Yes, patients have the flexibility to schedule follow-up visits within a few days before and after the expected date. Furthermore, if conditions outside the control of the patient change (Covid for example), or if the patient is unable to travel due to health limitations, the study site will work with the patient on scheduling and potentially offer telemedicine options for select visits. Additionally, your study site may be able to assist with travel services to and from office visits. What medicines can participants remain on while in the TRACTION-2 trial? Some medications, including calcineurin inhibiters (CNIs; cyclosporine for example), will need to be discontinued. Others, including angiotensin converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARB) should be continued, while various other drugs may be continued if on a stable dose. Please consult your healthcare provider for additional details. Does this trial have a placebo comparison? Yes. The placebo is used because comparing results in study participants who receive GFB-887 with results in participants who receive placebo is the best way to explore how well GFB-887 works and how safe it is. 66% of patients will receive GFB-887 (33% will receive placebo), but all patients will have the option to join an extension study where all patients will be offered GFB-887 for up to three years. What will happen to the results of this clinical trial? Can the participant stay on the medicine if it works? The results of this study will help guide the additional exploration of GFB-887 in these diseases and potentially others. It is one of many steps required before the FDA will approve a drug for broader availability. Currently, participants, once completing the initial study, have the option to participate in another “extension” study for up to three years. If a patient was taking placebo during the initial trial, they will receive GFB-887 in the “extension” study. Do you have plans for expanding into a pediatric trial? The current trial does not allow for patients under the age of 18, but we hope to offer additional options for younger patients in the future. Will the patients be paid for participating in the trial? Patients may be eligible to receive compensation for time and reasonable out-of-pocket expenses related to taking part in this trial. For more information, please talk to your trial doctor and trial staff. Goldfinch Bio’s TRACTION-2 clinical research trial is evaluating an investigational precision medicine, GFB-887, for the potential treatment of TRMCD and FSGS. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities and find the right trial for you, visit KidneyHealthGateway.com. This article was developed in partnership with Goldfinch Bio, Inc.
NephCure Celebrates Introduction of New Era of Preventing End-Stage Kidney Disease Act April 15, 2022 by Kylie Karley KING OF PRUSSIA, Pa. (April 15, 2022) — NephCure Kidney International (NephCure) applauds Friday’s introduction of the New Era of Preventing End-Stage Kidney Disease Act (H.R. 7506) sponsored by Reps. G.K. Butterfield and Gus Bilirakis. This legislation would help transform the delivery of care to rare kidney disease patients by increasing community and healthcare provider awareness and education, addressing kidney health disparities in communities of color, and advancing rare kidney disease research. “I’m proud to introduce the New Era of Preventing End-Stage Kidney Disease Act, which is a critical step toward the transformation of kidney disease care,” said Butterfield, lead sponsor of the legislation. “For far too long, the rare kidney disease community has been overlooked, leaving many families in a chronic state of uncertainty as they navigate the many hurdles in getting diagnosed, treatment and care. There is a new era of hope on the horizon for these families.” “Our life-saving legislation will help remove diagnostic and treatment barriers for many patients suffering with a rare disease,” said Bilirakis. “Through increased education, we will empower providers to better identify the signs and symptoms of rare kidney disease, which will lead to improved treatment options and better patient outcomes.” Rare kidney diseases contribute to the more than $84 billion spent on treating Medicare beneficiaries with chronic kidney disease, including the $36 billion spent on treating people with end-stage kidney disease (ESKD), yet there has been little to no innovation in treatment for kidney disease patients since the 1960s when dialysis and immunosuppressants became commonplace. These treatment and diagnostic barriers, coupled with the lack of rare kidney disease awareness and education, often cause a delay in diagnosis that can result in a rapid decline in kidney function and, ultimately, kidney failure. For many rare kidney disease patients, their only options are dialysis, transplant or death. Additionally, communities of color are disproportionately affected by rare kidney diseases and face a lack of adequate treatment options due to existing health disparities — Black Americans are 4-5 times more likely to develop ESKD than white Americans. Many rare kidney disease patients struggle to find a nephrologist well-versed in their rare conditions who can provide an accurate diagnosis and expert care. FDA-approved treatments are lacking for most rare kidney diseases, but thanks to the 21st Century Cures Act we are on the cusp of a new era of rare kidney disease treatment and care — giving hope to thousands of patients and their families. By taking action now, Congress can promote health equity, save lives, and conserve valuable healthcare resources. “We applaud the Rare Disease Caucus Co-Chairs Mr. Butterfield and Mr. Bilirakis for their leadership. For decades there have been no breakthrough treatments for individuals experiencing rare kidney disease. However, we believe we are entering a ‘New Era of Kidney Care,’ and this comprehensive legislation will help forge a new frontier for innovation to thrive,” said Josh Tarnoff, NephCure CEO. “Dialysis and transplants cannot be the only options. It is time to modernize treatments and the delivery of care to kidney patients and bring it into the 21st century.” Revolutionizing rare kidney disease treatment has been a longstanding priority for NephCure. In 2020, a core group of partners, comprised of NephCure, Travere Therapeutics and the American Association of Kidney Patients (AAKP), facilitated the first-ever Rare Kidney Disease Roundtable and the resulting white paper entitled “We Deserve Better: Revolutionizing Rare Kidney Disease,” which outlined urgent needs for rare kidney disease patients and their families and helped inform the creation of the New Era of Preventing End-Stage Kidney Disease Act. NephCure will work with Congress to advance this bill as its key legislative priority. Patients and advocates are encouraged to join the NephCure Action Network, a movement of patients, caregivers, physicians and other partners who are committed to raising their voices to help educate policymakers on what’s important to those directly impacted by rare kidney diseases. For more information about the New Era of Preventing End-Stage Kidney Disease Act, visit NephCure.org/NewEraAct. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now more than 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. exempt tax-exempt 501(c)(3) public charity. ###
NephCure Recognizes National Minority Health Month this April April 4, 2022 by Kylie Karley National Minority Health Month celebrates the inclusion of Asian and Pacific Islander, Black, Hispanic, Native American, and other communities of color in the conversation about health education and disease detection, disparities, and treatments. Chronic kidney disease (CKD) affects 1 in 7 adults in the U.S., however, for people of color, a combination of systemic racism, lack of inclusion in medical advancements, and other uncontrollable risk factors contribute to even higher CKD rates. CKD in Communities of Color If you are Black or African American, Hispanic or Latino, Asian American, Pacific Islander, American Indian, or Alaska Native, or Native Hawaiian or Other Pacific Islander, heritage you may be at an increased risk for kidney disease. Minority populations are also at higher rates of high blood pressure, diabetes, obesity and heart disease — all of these factors increase the risk for kidney disease. Did You Know? Asian Americans and Native Hawaiians are the most understudied racial group in CKD research. Black Americans represent 13% of the population, but account for 32% of CKD patients. Hispanic Americans are 1.3 times more likely to develop kidney disease, and CKD rates have risen 70% since 2000. Our Health Equity Initiative With the inequities in kidney health in mind, NephCure launched a Health Equity Initiative in 2021 to address racial disparities in rare kidney diseases. NephCure acknowledges the voices of these communities, and we are excited to continue the work of addressing health disparities for all multicultural communities by featuring culturally relevant health education and resources, partnering with community-based organizations, and advocating for diverse and just medical research.
April is National Volunteer Month April 1, 2022 by Kylie Karley This month, the (much-deserved) spotlight is on you: our volunteers! April is National Volunteer Month, and NephCure is dedicating it to acknowledging the important role our volunteers serve in our community throughout the year. By generously giving their time to further our mission, our volunteers truly help us save kidneys and save lives. The past two years have changed the way volunteers serve in many ways, and in some instances completely paused certain volunteer activities. It’s been a difficult time for both nonprofits and volunteers as we push through and adapt to the ever-changing challenges of the pandemic — we’re incredibly appreciative and humbled by our 263 volunteers who have stuck through the transition or who are jumping back in full force to fight for rare kidney disease patients. To our volunteers: thank you, thank you, THANK YOU! We can’t emphasize our gratitude for your dedication enough, and we love working with you! NephCure offers a variety of unique and engaging opportunities for volunteers across the globe. Our volunteers make essential contributions to our mission by: Serving as Regional and State NephCure Community Leaders Serving on NephCure’s Board of Directors Leading committees for signature NephCure fundraising events, such as our Countdown to a Cure galas, nationwide series of Pig Jigs, and more Participating in our advocacy work through the NephCure Action Network Planning community fundraisers, including local walks and golf outings Providing one-on-one support to other patients and families in need through our Patient Connections support program Sharing their stories through our Voice of Patient program Helping with social media marketing And more! In celebration of our volunteers this month, we’re excited to launch our new NephCure Service Award Program as a small token of our appreciation for our volunteers’ dedication and generosity. Volunteers will receive an annual service award pin recognizing each year of their contributions to NephCure, with special awards for 5, 10, 15, and 20 years of service. To our current volunteers: look out for service award pins coming your way! We’ll also be recognizing those who have gone above and beyond with the Dedicated Service Award, NephCure Impact Award, and Leadership Award. Stay tuned to learn more about our award recipients and the amazing work they’ve done for NephCure. Are you interested in helping NephCure save kidneys and save lives? Contact Kelly Helm at khelm@nephcure.org to learn more about volunteer opportunities.
Get To Know Your Kidneys: Understanding the Warning Signs of Kidney Disease March 29, 2022 by Kylie Karley Did you know that your kidneys are only about the size of your fists? These small but powerful organs carry the huge responsibility of filtering waste from our blood. With such an important function, it’s crucial to maintain your kidneys’ health — but how do you know if your kidneys are healthy? And how can you protect them from damage? Read on to learn more. What do my kidneys do and why are they important? Our kidneys perform a host of vital functions. They maintain a healthy balance of water and electrolytes, such as sodium, potassium, and magnesium. The kidneys also regulate blood pressure and help produce red blood cells. They are the only organs that filter our blood. Without them, toxins can build up in the body — this build-up can potentially turn deadly. How do I know if my kidneys are healthy? Know Your Family History: Does anyone in your family have kidney issues or renal failure? Does high blood pressure or diabetes run in your family? If you answered yes, don’t panic. Instead, be proactive and informed. Many of us have family members with these health concerns. These factors could be a clue about your own kidney health and your risk for developing kidney disease. If you are unsure of your family history, consider starting the conversation with your family about kidney health. Know Your Medical History: Have you been diagnosed with any medical conditions or diseases? Uncontrolled diabetes or high blood pressure, also known as hypertension, can lead to kidney damage. One of the most important warning signs is if your blood pressure is abnormally high despite taking medication. If this is the case, it’s time to get tested for kidney disease. Look for Signs and Symptoms: One way to monitor your kidney health is being aware of what to look for. These signs and symptoms may mask themselves as other issues that have little effect on your daily life. This is why kidney disease is sometimes called a “silent disease” and can be difficult to catch early on. Signs and symptoms include: Family History of Kidney Disease Dry, itchy skin Always feeling cold Frequent fainting or dizzy spells Prolonged swelling of the legs and feet Blood in the urine Foamy or brown-colored urine Unrelenting tiredness Trouble concentrating Puffiness around the eyes Lack of appetite and persistent nausea Talk With Your Doctor: As essential as the kidneys are, their decline can go unnoticed for months, or even years because signs and symptoms are usually not immediate. Staying up to date on your kidney health often begins with a conversation with your doctor. Ask them about kidney labs, also referred to as renal labs, and inform them of any abnormal signs and symptoms you’ve noticed. If appropriate, the doctor may run blood and urine tests that help determine your level of kidney function. I want to be proactive about my kidney health. What’s next? Take the opportunity to tackle kidney disease head on. Remember the four tips for staying on top of your kidney health: know your family history, know your medical history, look for signs and symptoms, and talk with your doctor. If you or someone you know is dealing with these warning signs, make contacting a doctor a priority, and visit NephCure.org for more information and resources.
The Difference a Doctor Makes: Christine’s Fight for Daughter’s Health December 8, 2021 by Kylie Karley Christine Floto and her daughter, Madi Madi was only 4 years old when she faced potential kidney failure. It took Madi’s mom Christine years to realize these harsh medications were not only failing to improve Madi’s kidneys but were making her sicker overall. Madi’s nephrologist insisted the current medications were the best they would get. Christine asked for further testing, but they denied her requests as Madi’s kidneys worsened. Then Christine found NephCure. She attended a patient summit and broke down in tears when she learned there were better options for her daughter. Madi no longer had to suffer. Generous support from people like you helped NephCure connect Christine with an expert doctor who listened to her concerns. He ordered a genetic test, revealing that Madi has an extremely rare genetic cause of focal segmental glomerulosclerosis (FSGS). Madi was enrolled in a clinical trial, which gave her access to cutting-edge medication. With the help of NephCure and her new doctor, Madi got her life back. Her kidney disease is slowing, she gained 30 pounds, and is doing better than she has in years! “Through NephCure, I learned more about Madi’s disease and gained the confidence to seek care from top specialists,” Christine said. We have entered a new, unprecedented era of rare kidney disease care. Now more than ever, NephCure is laser-focused on ensuring all patients have access to the latest research and care. NephCure offers online education and support programs that empower rare kidney disease patients and their families, just like Madi and Christine. Through our NephCure Specialists program, we help connect patients with expert doctors who provide the latest treatment options. We are expanding local communities to help connect people to peer support, but more work is needed. “We will forever be grateful for NephCure’s education and support,” Christine said. “It’s given our family our life back.” Will you donate to help us ensure that every patient receives the best care and support possible? To make a contribution to our organization before the year end, please click here. Thank you in advance for your generosity.
NBA Hall-of-Famer Alonzo Mourning Shares Personal Story to Raise Kidney Disease Awareness November 18, 2021 by Kylie Karley KING OF PRUSSIA (Nov. 18, 2021) – NBA hall-of-famer Alonzo Mourning was at the height of his basketball career—he had just won gold in the 2000 Summer Olympics when he noticed extreme swelling throughout his body and a lack of energy. A routine physical exam showed abnormalities, and eventually, Mourning received a diagnosis of a rare, protein-spilling kidney disease called focal segmental glomerulosclerosis (FSGS). “I said ‘Doc, am I going to die?’ He paused; he took too long to answer me. And he said, ‘We have no known cure’… ultimately, he said, ‘In about 10-12 months you’ll probably be on dialysis,’” Mourning said as he recalled the jarring day. Mourning, who missed the entire 2002-2003 season due to kidney disease and later went on to win the World Championship with the Miami Heat in 2006, publicly recounted his personal journey with FSGS in a video for NephCure Kidney International’s kidney disease awareness campaign, debuting Nov. 18. In highlighting Mourning’s story, NephCure aims to reach those who are at risk for kidney disease and to educate the public about the signs and symptoms of these conditions. Black Americans are 4-5 times more likely to develop kidney failure than white Americans, and 1 in 8 are at risk for a genetic form of kidney disease. A variation on the APOL1 gene contributes to this disparity. “We are proud to work with Alonzo Mourning in sharing such an important message that will inspire and inform others who fall within this at-risk population. Our goal is to find a cure for this debilitating kidney disease, as well as educate and support those who are affected by it,” said Michael Levine, NephCure Board President and kidney disease patient parent. This awareness campaign comes just months after NephCure launched its Health Equity Initiative, with the goal of ensuring equitable access to advancements in research, treatments, and care, and to reach individuals from communities of color earlier in their disease progression, preventing or delaying their need for dialysis and transplantation. Support for this video and the awareness campaign was provided by Vertex Pharmaceuticals, Travere Therapeutics, and other funders of the Health Equity Initiative. To watch the entire video with Alonzo Mourning, click here. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now more than 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. Learn more at NephCure.org. ###
The Eyes Behind the Microscope: Meet Dr. Astrid Weins September 30, 2021 by Kylie Karley Astrid Weins, MD, PhD, is an Associate Pathologist at Brigham and Women’s Hospital in Boston, MA, and an Assistant Professor of Pathology at Harvard Medical School. Her research focuses on understanding acute Nephrotic Syndrome and podocyte injury. She does this by combining observations with state-of-the-art imaging and tissue interrogation techniques. Learn more about her work on Minimal Change Disease (MCD) and about the TRACTION-2 trial, Goldfinch Bio’s clinical research trial investigating GFB-887 with the hope of preventing the progression of kidney disease in patients with treatment-resistant Minimal Change Disease, FSGS, and Diabetic Nephropathy. The TRACTION-2 trial is the first large-scale clinical trial opportunity for Minimal Change Disease patients, and is also available for patients with FSGS and Diabetic Nephropathy. Why are you interested in Minimal Change Disease (MCD)? Astrid Weins, MD, PhD I have studied kidney, and specifically, podocyte biology for many years. The podocyte is a unique and fascinating cell with a critical function in regulating urine filtration. It is the main target of injury in MCD. As a kidney pathologist, I love scientific investigation, and my strong belief is that knowing the cause of a disease is the key to its cure. Minimal Change Disease deserves so much more attention than is has received in the past. People often refer to it as a “benign” condition, because in many cases it responds well and fast to steroids. However, there are some children, and even more adults that don’t respond to this therapy, and we have currently no way of predicting who will respond and who won’t. Once we understand what causes a disease, we can develop rationales for choosing and developing specific, targeted therapies. What makes MCD different from other protein-spilling kidney diseases? First, it’s of rapid onset and in most cases also shows a rapid response to therapy. Second, we have had no idea what causes it, other than that we presume it’s caused by a circulating factor. Third, in contrast to most other protein-spilling diseases, it is more common in children than in adults. Fourth, the kidney biopsy shows no significant changes by light microscopy, although the ultrastructural changes to the podocytes are widespread and severe. Hence, the term “minimal change” disease (which refers to its unremarkable light microscopy appearance) is really a misnomer. Can you summarize your research on MCD and its significance? In our clinical practice, my colleagues and I came to recognize a unique feature in biopsies from patients with MCD, which suggested that the injury might be caused by an autoantibody against podocytes. An autoantibody-mediated cause would make sense, since antibodies are circulating in the blood, are filtered by the kidney and can access the podocytes. We hypothesized that the target of such an autoantibody would have to be an exposed podocyte protein, which led us to test for autoantibodies against nephrin, an essential component of the filtration barrier. Indeed, we identified anti-nephrin autoantibodies in a subset of adults and children with MCD both circulating in their blood and bound to nephrin in their kidney biopsy. This is a huge breakthrough in our understanding of this condition, as we can now define a subset of patients as suffering from an autoimmune disease. What are you looking at in your NephCure-funded grant, “Autoimmunity in Primary or Recurrent Podocytopathies – Clinicopathologic correlation and mechanistic studies”? In this grant, we are now extending our studies to patients with primary FSGS and recurrent FSGS after transplant. Since the changes to the podocytes in MCD and primary and recurrent FSGS are virtually indistinguishable, we hypothesized that a subset of patients with primary and recurrent FSGS also have circulating antibodies that target nephrin. Our preliminary findings demonstrate that this is indeed the case in a subset of patients from our own institution. Now we need to expand our studies to more samples. Can you tell us about treatment-resistant MCD versus steroid-sensitive MCD? The vast majority of MCD is steroid-sensitive, meaning that these patients will respond well to steroids. A small subset of these patients, however, will remain steroid-dependent, meaning they cannot come off this therapy or their Nephrotic Syndrome will relapse. This is not a good thing because 1) steroids have harsh side effects, especially during long-term use, and 2) often additional immunosuppressive agents are given that may add even worse side effects. Treatment-resistant patients never fully respond to steroids, and often receive a long list of unsuccessful immunosuppressive therapies, and despite all efforts may continue to spill protein and experience ongoing podocyte damage. Why do you think so many people are diagnosed with MCD to later be re-diagnosed with FSGS? Two possible reasons: 1) Sampling: With exception of the scarring, both MCD and Primary FSGS are indistinguishable on biopsy. Since we only look at a tiny sample of biopsied kidney tissue, and early FSGS can be very focal, we may miss scarring already present in your kidney. 2) Disease progression: A disease does not start out with scarring. Being diagnosed with MCD in your first and with FSGS in a later biopsy does not mean you were re-diagnosed; it just means that the initial injury was severe enough to have led to scarring during the course of the disease. Based on my experience, I believe that MCD and Primary FSGS are related and can represent expressions of the same initial disease process. This means that understanding one could enhance our understanding of the other. Why do you think there have not been trials for Minimal Change Disease patients until now? MCD has been generally perceived as a rather “benign” condition. Many patients respond well to a limited course of steroids, and complications remain rare, so there was no perceived acuity to start trials. We also haven’t had sufficient molecular understanding of this disease to provide a rationale for trying new or different treatments. Our studies may change how clinicians view this disease and may initiate such trials. Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on advancing kidney precision medicine. Its mission is to deliver disease-modifying precision medicine that brings hope and renewed quality of life to people living with kidney diseases. Goldfinch Bio’s TRACTION-2 clinical research trial is evaluating an investigational precision medicine, GFB-887, for the potential treatment of treatment-resistant Minimal Change Disease, FSGS, and Diabetic Nephropathy. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities and find the right trial for you, visit KidneyHealthGateway.com. This article was developed in partnership with Goldfinch Bio, Inc.
Everything You Need to Know About Travere’s New Clinical Trial for Children, EPPIK September 2, 2021 by Kylie Karley Travere Therapeutics has recently launched their Phase 2 pediatric clinical trial, EPPIK (Evaluating Problematic Proteinuria in Kids) which is aimed at helping children with FSGS, MCD, IgAN and other rare kidney diseases. We know that with a clinical study specifically designed for kids, parents and caregivers may have some questions. We asked Travere to answer some of these questions and have complied an FAQ list for our community around EPPIK. What is the EPPIK study? EPPIK (Evaluating Problematic Proteinuria in Kids) is a Phase 2 study of children 1-17 years old with focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), IgA nephropathy (IgAN), also known as Berger’s disease, IgA vasculitis (IgAV), also known as Henoch-Schönlein purpura, or Alport syndrome (AS). These rare kidney diseases are associated with progression to end-stage kidney disease and currently have limited or no treatment options. This study aims to further understand if an investigational therapy, sparsentan, can help the kidneys filter protein and slow the decline of kidney function. The amount of protein found in the urine (called proteinuria) is seen as a marker of kidney function. Lowering proteinuria levels is associated with better kidney health outcomes. What is sparsentan? Sparsentan is a novel Dual Endothelin Angiotensin Receptor Antagonist (DEARA) – this means it is a dual acting, highly selective antagonist of both the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). In the EPPIK Study, sparsentan is taken once a day orally. What is a Phase 2 study? A Phase 2 study is a clinical trial that helps researchers determine how safe, tolerable and effective an investigational drug may be in specific patient populations, such as those noted above. While the EPPIK study is a Phase 2 study, sparsentan has been studied in another Phase 2 study as well as two Phase 3 studies involving approximately 880 patients (some of whom were children). The purpose of the EPPIK study is to gain further experience on the use of sparsentan in children. How long does this study take? Patients who meet all eligibility criteria and are enrolled in the study may expect to participate in the study for about 2 years and 3 months. Will you be enrolling children with congenital Nephrotic Syndrome or infantile Nephrotic Syndrome? Yes, children ages 1-17 with one of those syndromes who meet the study criteria will be able to participate in the EPPIK Study. The specific disorders we are looking for are noted above in the answer to question 1. How many children and young people are you looking to enroll in this study? We will be looking to enroll approximately 57 children in the EPPIK study. How many study sites are there available? Where are these sites located? Sites will be in the United States, UK, and various countries in the EU (France, Germany, Italy, Spain, Netherlands, Poland, Sweden). The final list of sites has not been fully determined yet. Because this study is for children, how is it different than a study for adults? The main difference is how much blood is drawn from the children to do all the tests necessary to evaluate sparsentan in the study. Patient safety is always our number one priority, and, with children, we won’t sample as much blood as we would from an adult. We narrowed the number of tests and worked with the central lab to be able to get results from a smaller sample size. We also must ensure that we adequately inform both the child who might participate in the study and the child’s parents/guardians about all the requirements the study has as well as any risks that may be associated with participating in the study. We do this by developing different study assents or ways to communicate what the study entails so that participating families understand the purpose of the study and what is involved. Are you asking kids about the taste, smell, etc. of the study drug? Yes, we will be asking the children to answer simple questions on how the drug tastes, smells, feels, etc. How do you determine which participants will be required to have pregnancy testing or take birth control? We realize this is a delicate subject for some children and their parents, but it is very important that any female child who has had her first menstruation period take birth control according to the protocol. This class of drugs is known to potentially cause harm to a developing fetus, and therefore we do not want any female participant who is able to have a child to become pregnant. Is there flexibility where lab samples can be collected for my child? Is there an option for home care visits to collect these samples? Yes, there is some flexibility in where labs are taken. If the child can’t make it to the clinical site to have the labs drawn, which is always the preferred way, we have contracted with a company that will come to the patient’s home or other convenient location, to draw the labs and send them in for testing. Does my child have to stop the medications they’re on? What drugs can they remain on? That depends, if they are enrolled in this study, they will need to stop any drug that functions similarly to the investigational drug. That is, if they are on an ACE inhibitor or ARB, they will need to stop those drugs at least 2 weeks before they begin taking the experimental drug and will not be able to take them while they are participating in the study. They will be able to continue with certain blood pressure medications if not in the same drug class, as well as many other medications they may be on. The Investigator will have a complete list of drugs that are allowed and not allowed. What will happen to the results of this clinical study? After all the patients have been enrolled and have completed the study, the data will be analyzed to see if it was successful in reducing the amount of protein in the urine and if it slowed the decline in kidney function as measured by estimated glomerular filtration rate, or eGFR. How well the drug was tolerated, that is, the safety profile, will also be analyzed. Those results will, in all likelihood, be put into a document known as a manuscript and published in a scientific journal. Data will also be added to the study listing on the clinicaltrials.gov website at the end of the study. If my child is in another trial studying sparsentan, do they have to enroll in this new study? If so, will their study site change? No, if your child is participating in another trial with sparsentan they would not be eligible to join the EPPIK study. They would stay in the study they are currently in. For more information about EPPIK, contact medinfo@travere.com
Kidney-Friendly Recipe: Italian Herb Grilled Chicken with Grilled Veggies and Quinoa September 1, 2021 by Kylie Karley As kids head back to school and the long summer nights slowly slip away, celebrate the end of the season with a delicious meal on the grill—perfect for Labor Day Weekend! This recipe comes from NephCure’s Chef Sachet, a patient parent whose 12-year-old son Aiden was diagnosed with FSGS in January 2017. Aiden’s kidney disease rapidly progressed—just five months after diagnosis, he ended up on dialysis and eventually went on to receive a kidney transplant. As a kidney disease caregiver, Chef Sachet says ‘it’s important to me to keep kidney-friendly recipes in mind!’ Yield: 4 people Prep time: 15 min Cook time: 25 minutes Sodium: 139mg per serving Ingredients: 4 6-oz chicken breasts (with tenders attached) IMPORTANT NOTE: Check the sodium content on the chicken breasts to make sure they are no higher than 75 mg of sodium per 4 ounce serving or no higher than 113 mg per 6 ounce package. 2 cups of quinoa (any kind works) 1 large zucchini or squash 1 lime 1 lemon ½ handful of fresh parsley, minced 2 sprigs of fresh oregano, minced 2 sprigs of fresh thyme, minced 2 heads of fresh garlic, minced ¼ cup of extra virgin olive oil (EVOO) Pepper (to taste) 4 cups of no-salt veggie stock 2 Tbsp. of unsalted butter Instructions: First, set your chicken breasts aside in a large bowl. Mince all herbs and toss in chicken with garlic, pepper, lemon juice, and a drizzle of EVOO. Coat chicken well and marinate for 15 minutes. Heat the vegetable stock in large pot on medium-high heat until simmering. Add quinoa. Lower heat, add 2 Tbsp. of butter and a sprig of thyme. Let cook until all of the liquid is absorbed. Once most liquid is gone, turn off the heat, and cover the quinoa until serving time. Once the quinoa is cooking, cut vegetables into thick slices. Add pepper, EVOO, and a squeeze of lime, and then place them on the grill. Remove chicken from the fridge and start to grill it: 7-10 minutes on each side until the internal temperature reaches 165 degrees Fahrenheit. Chef’s Tips: Do not flip your chicken constantly when grilling. It will take a long time to cook and will dry out. Plus, you won’t get those awesome grill marks! Raw veggies can take a while on the grill. Season and lubricate them with oil ahead of time, but don’t add too much oil. It will just burn off and char the veggies. When using store-bought stock, always use low-sodium or no-salt varieties.