Actively Recruiting
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
Led by National Human Genome Research Institute (NHGRI) · Updated on 2026-05-14
30
Participants Needed
1
Research Sites
80 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Focal segmental glomerulosclerosis (FSGS) is a disease that causes scarring in parts of the kidneys that filter waste. This can lead to protein loss in the urine, which can worsen kidney function. The kidneys may fail over time, and dialysis or a kidney transplant may be needed. Other treatments for this disease do not always work and often have adverse effects. Better treatments for FSGS are needed. Objective: To test a study drug (ManNAc) in people with FSGS. Eligibility: People aged 18 years and older with FSGS. Design: Participants will have 5 to 6 clinic visits over 14 weeks. Two of the visits will require overnight stays for 2 or 3 nights. ManNAc is a white powder that comes in a sachet. It is dissolved in water and taken twice a day by mouth. Participants will take their first dose at the clinic. They will learn how to store ManNAc and prepare each dose. They will record their doses in a diary. They will also write down any adverse effects or troubles they have using the drug at home. During clinic visits, participants will have physical exams with blood and urine tests. They will complete questionnaires about their health, sleep habits, and fatigue symptoms. During overnight visits, participants will also have 24-hour urine collection. A study team member will call participants 1 week after the first dose to check on their health. Follow-up phone calls will then be every 2 weeks after each clinic visit. Participants may meet with a dietitian to discuss nutrition while taking the ManNAc. Participants may choose to have genetic tests.
CONDITIONS
Official Title
An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Prior kidney biopsy showing FSGS within 10 years before screening.
- Age 18 years or older and weighing more than 50 kg.
- Stable immunosuppressive therapy for at least 4 weeks before trial if applicable, with no planned changes during the 12-week study.
- Stable doses of standard non-immunosuppressant antiproteinuric agents for at least 4 weeks before trial if applicable.
- Spot urine protein to creatinine ratio (PCR) of 2 g/g or higher on at least three measurements on two separate days, plus 24-hour urine protein of 2 g/day or higher.
- Estimated glomerular filtration rate (eGFR) of 45 mL/min/1.73 m² or higher.
- Willingness to use effective birth control if of reproductive potential, unless permanently sterilized.
- Signed informed consent and willingness to comply with study procedures, including diaries and visits.
You will not qualify if you...
- Unable or unwilling to provide informed consent.
- Uncontrolled nephrotic syndrome with proteinuria above specified thresholds and related symptoms or complications.
- Need for intravenous diuretics to control volume overload.
- Psychiatric or neurological conditions that interfere with study adherence.
- Vulnerable individuals such as those with impaired cognition or incarceration.
- Renal biopsy showing other kidney diseases or more than 50% interstitial fibrosis and tubular atrophy.
- Uncontrolled hypertension above 140/90 mmHg.
- Active infections including HIV, Hepatitis B or C, or other specified infections.
- Progressive liver disease or abnormal liver function tests.
- Hypertriglyceridemia above 500 mg/dL.
- History of malignancy within last 5 years or active therapy.
- History of Type I or Type II Diabetes Mellitus.
- Hematological or cardiac conditions associated with FSGS.
- Use of medications linked to FSGS within last 6 months.
- Pregnancy, breastfeeding, or refusal to use birth control during study.
- Recent use of other investigational drugs or ManNAc-related supplements.
- History of organ or stem cell transplantation.
- Conditions increasing risk of adverse events or confounding study results.
- Need for systemic immunosuppressive or glucocorticoid therapy for non-renal reasons during trial.
- Recent use of rituximab, cyclophosphamide, or plasmapheresis.
- Active alcohol or substance abuse within past 2 years.
- Collapsing FSGS variant.
- Clinical history suggesting post-adaptive FSGS or obesity with BMI over 40 kg/m².
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
M
Marjan Huizing, Ph.D.
CONTACT
W
William A Gahl, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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