An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis

Recruiting now · Phase 2

Conditions studied: Focal Segmental Glomerulosclerosis

In brief

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.

Key facts

Study ID: NCT06664814
Run by: National Human Genome Research Institute (NHGRI)
People needed: 30
Starts: 2026-08-13
Expected to finish: 2027-12-01
Last updated by the study team: 2026-08-10

Who can join

Age: 18 and older, up to 115. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

An individual who meets any of the following criteria will be excluded from participation in this study:
Individuals who are unwilling or unable to provide informed consent.
Individual presenting for initial therapy of uncontrolled nephrotic syndrome which we define as proteinuria as measured by spot urine protein:creatinine ratio >3g/g or 24-hour urine protein excretion of >3.5g/day with symptomatic peripheral edema or pulmonary edema, hypoalbuminemia (serum albumin <3.0g/dl), electrolyte disturbances (related or unrelated to medical therapy), and/or active thromboembolism (initiated on systemic anticoagulation in the last 3 months). The rationale for this criterion is that patient presenting for initial therapy for uncontrolled nephrotic syndrome may need to be initiated on various medications including immunosuppressives as well as non-immunosuppressive drugs which can significantly change the level of proteinuria. Additionally, fluid shifts due to diuretic use for treating edema can alter GFR which could confound the pharmacokinetics and pharmacodymanics of the investigational drug.
Individuals who acutely require optimization of volume status with intravenous diuretics to control volume overload, as this may result in fluid shifts between the intravascular space and the remainder of extracellular fluid volume. This might alter drug pharmacokinetics and pharmacodynamics as mentioned above in # 2.
Individuals with a psychiatric illness or neurological disease that in the judgement of the investigators would interfere with the ability to adhere with the requirements of this protocol. This includes, but is not limited to, uncontrolled/untreated psychotic depression, bipolar disorder, schizophrenia, substance abuse or dependence, antisocial personality disorder, panic disorder, or behavioral problems, which might interfere with effective communication.
Vulnerable individuals, including those with impaired cognitive function or are incarcerated.
Individuals whose renal biopsy shows evidence of an additional pathology along with FSGS.
Renal biopsy showing interstitial fibrosis and tubular atrophy (IFTA) >50% per biopsy report.
Individuals with uncontrolled hypertension with blood pressures consistently >140/90 mmHg on 3 or more community clinic blood pressure measurements.
Individuals with clinical evidence of any type of active infection including but not limited to HIV, Hepatitis B and/or Hepatitis C or patients who are positive on screening test for HIV including antibody or viral load, HBV surface antigen and/or HCV antibody. If a patient is positive for HCV antibody, then an HCV viral load will be measured to identify subjects with active infection. Additional tests may include those to screen for active CMV, Infectious Mononucleosis (Epstein-Barr Virus), parvovirus B19 and/or COVID-19 infection as per investigator judgement.
Individuals with evidence and/or documented history of progressive deterioration of liver functions, including the production of clotting factors, removal of toxic metabolic products, bile excretion for at least 6 months, routine hepatic laboratory indices including but not limited to (AST, ALT, or GGT) greater than 3 times the upper limit of normal, and/or individuals with a documented history and/or diagnosis of chronic liver disease.
Individuals with hypertriglyceridemia >500 mg/dL.
Individuals with a documented history of malignancy (identified in last 5 years or on active therapy at time of screening).
Individuals with a documented history of Type I or Type II Diabetes Mellitus
Individuals with documented history of hematological conditions commonly associated with FSGS such as sickle cell disease and myeloproliferative disorders.
Individuals with a documented history of cardiac conditions commonly associated with FSGS such as congenital cyanotic heart disease. Individuals with a documented history of connective tissue disorders commonly associated with FSGS such as SLE and multiple sclerosis.
Individuals taking medications (either currently or within the last 6 months) associated with FSGS such as interferons, lithium, androgenic steroids, sirolimus, heroin, anthracyclines and bisphosphonates.
Individuals who are pregnant, will be breastfeeding or refuse birth control anytime during the study.
Individuals who have received treatment with another investigational drug, investigational device, or approved therapy for investigational use less than 60 days prior to planned ManNAc dosing.
Individuals with hypersensitivity to ManNAc.
Individuals who have been treated with ManNAc, sialic acid, IVIG, and/or other supplements containing sialic acid (e.g., sialyllactose) less than 60 days prior to planned ManNAc dosing.
Individuals who received a renal or any other solid organ and/or bone marrow/stem cell transplantation.
Individuals who in the judgment of the investigator, have a condition that places the subject at increased risk for AEs or has any other illness or clinical condition that might confound the results of the study or pose an additional risk in administering study drug to the subject.
Patients who need systemic immunosuppressive or glucocorticoid therapy for non-renal indication at any time throughout the trial.
Any patient receiving rituximab, cyclosphosphamide and/or plasmapheresis within 6 months of screening.

Where it is running

National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)

Full record on ClinicalTrials.gov

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