EN 
ES 
Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
Brief Description
This is a phase-2 clinical study, which means it tests whether a new medicine in a small group of people with a specific illness works well and is safe before larger studies are done. It comes usually comes after Phase 1, which checks basic safety of the drug in humans. This study will use the drug called N-Acetyl-D-Mannosamine (ManNAc) in patients with a disease called primary FSGS at a dose of 2g by mouth twice a day during a period of 12 weeks.
Trial Physician / Study Coordinator
Anirban Ganguli
Email
Site Name
National Institutes of Health (NIH) Clinical Center
10 Center Dr, Bethesda, MD 20892
Sponsor
National Human Genome Research Institute (NHGRI)/National Institutes of Health (NIH)
Study Drug
N-Acetyl-D-Mannosamine (ManNAc)
Estimated enrollment
15
Estimated end date
July 2028
Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
Brief Description
This is a phase-2 clinical study, which means it tests whether a new medicine in a small group of people with a specific illness works well and is safe before larger studies are done. It comes usually comes after Phase 1, which checks basic safety of the drug in humans. This study will use the drug called N-Acetyl-D-Mannosamine (ManNAc) in patients with a disease called primary FSGS at a dose of 2g by mouth twice a day during a period of 12 weeks.
Trial is for people with
Primary Focal Segmental Glomerulosclerosis (FSGS)
Study Goal
This study wants to learn more about how the new drug ManNAc works in the body, how much of it stays in the body at different times, if it is safe as was shown in earlier studies, and if it helps people with FSGS by lowering the amount of protein in their urine. It will also check if patients feel better and if their kidney tests improve during the 12 weeks of treatment.
What is involved for the Patient?
Patients in this study will be asked to let the researchers look at their medical records, past medications, and kidney biopsy reports. The study team may also ask for a small sample of kidney tissue from the hospital where the biopsy was done. If the patient qualifies for the study, the team will take blood and urine tests to confirm they have a kidney condition called primary FSGS. Next, the patient will stay at the NIH Clinical Center for 3 days. During this time, more blood and urine tests will be done, and the patient will be given the study medicine. The medicine, called ManNAc, is a powder that is mixed into water and taken twice a day. The research nurses will show the patient how to take it the right way. If the patient does well with the medicine during the hospital stay, they will go home with a 12-week supply of ManNAc. After that, the patient will visit the clinic at NIH 4 times—every 2 weeks. Between these visits, they will also get phone calls once a week to check for any side effects from the medicine. At each clinic visit, the study team may do more blood and urine tests to make sure the medicine is safe and working. After 12 weeks, the patient will return to the hospital for a final 3-day stay. During this time, study team will check again how safe the drug is, how much of it is in the blood and urine, and whether it is helping. The study will end at week 14, which is two weeks after the medicine is stopped. At that time, the patient will have a final clinic visit at NIH for more tests, questions, and a check-up. Patients and their insurance will not have to pay for the medicine, tests, or care during the study. The study is paid for by the National Human Genome Research Institute at the National Institutes of Health (NIH). However, patients will not be given any monetary compensation for the study.
About the drug or intervention
The medicine used in this study is called N-Acetyl-D-Mannosamine, or ManNAc for short. ManNAc is a type of sugar, but it’s not like the sugar we eat in food. It doesn’t seem to change blood sugar levels. ManNAc helps make another important molecule in the body called sialic acid. Sialic acid is found at the ends of proteins that stick out of every cell, and it helps cells talk to each other. In the kidney, some studies with animals have shown that when they have a disease called nephrotic syndrome (where a lot of protein leaks into the urine), they have less sialic acid on certain kidney cells called podocytes. Podocytes are part of a special structure in the kidney called the glomerulus, which filters the blood. If the filter (called the glomerular filtration barrier) becomes leaky, protein from the blood can spill into the urine. This leak is the main cause of many kidney diseases, including one called FSGS, which this study is focused on. Our team and others have found in animal studies that ManNAc can help podocytes make more sialic acid. This can fix the leaky filter and lower the amount of protein lost in urine. Losing too much protein in urine can lead to kidney failure if not treated, so stopping this loss may help protect the kidneys in people with FSGS. While ManNAc has shown benefits in animals, we don’t yet know if it works the same in people. It is also being tested as a treatment for a rare muscle disease called GNE Myopathy, and those studies showed it is mostly safe. In this study, we want to learn more about how safe it is to use ManNAc for a long time and whether it can lower protein loss in the urine of people with FSGS.
