Nephrology
PHASE2
● Phase II
Phase 2 Study: ManNAc for FSGS Shows Promise in Reducing Proteinuria
ClinicalTrials.gov
Published March 27, 2026
National Human Genome Research Institute (NHGRI)
NCT06664814 ↗
This open-label, single-arm Phase 2 study investigates the efficacy and safety of N-Acetyl-D-Mannosamine (ManNAc) in patients with primary focal segmental glomerulosclerosis (FSGS). Conducted by the National Human Genome Research Institute, the trial aims to enroll 30 participants aged 18 and older. Participants will receive 2,000 mg of ManNAc orally twice daily for 12 weeks. The primary efficacy endpoint is the reduction of proteinuria, a key marker of disease progression in FSGS. The primary safety endpoint is the assessment of long-term safety and tolerability of ManNAc. Secondary objectives include further evaluation of pharmacokinetics and additional health outcomes. The study design includes 5 to 6 clinic visits over 14 weeks, with two requiring overnight stays for comprehensive monitoring, including 24-hour urine collection. Participants will be closely monitored through regular physical exams, blood and urine tests, and health questionnaires. Interim follow-up will occur via phone calls every two weeks post-clinic visits. While specific statistical outcomes are not yet reported, the study's design focuses on establishing both the safety profile and potential efficacy of ManNAc in reducing proteinuria. The trial's results could inform future therapeutic strategies for FSGS, a condition with limited effective treatment options.
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· Auto-published
This study focuses on a kidney disease called focal segmental glomerulosclerosis (FSGS), which causes scarring in the kidneys and leads to protein loss in urine. Researchers are testing a new drug named ManNAc to see if it can help reduce this protein loss and improve kidney function. Participants will take ManNAc twice a day for 12 weeks while visiting the clinic for check-ups and tests. The goal is to find out if ManNAc is safe and effective for treating FSGS. If successful, this could offer a new treatment option for patients who currently have limited choices. However, it’s important to remember that this is still a study, and more research will be needed to confirm the results and understand the long-term effects.
What this means for you: A new drug may help reduce protein loss in urine for patients with a specific kidney disease.
View Original Abstract ↓
Status: RECRUITING | Phase: PHASE2
Condition(s): Focal Segmental Glomerulosclerosis
Intervention(s): ManNAc (DRUG)
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.
Detailed: Study Description:
Phase 2, open-label, single-arm, single-center study of ManNAc 2,000 mg oral (PO) twice daily (BID) for 12 weeks in 15 subjects with primary focal segmental glomerulosclerosis (FSGS). The study will characterize the long-term safety, tolerability, pharmacokinetics, and efficacy of ManNAc for proteinuria reduction in subjects with primary FSGS. We hypothesize that ManNAc will be safe and well-tolerated and will reduce proteinuria in subjects with primary FSGS.
Objectives:
Primary Efficacy Objective:
Determine the efficacy of ManNAc therapy in reducing proteinuria in subjects with primary FSGS.
Primary Safety Objective:
Assess the long-term safety and tolerability of orally administered ManNAc to subjects with primary FSGS.
Secondary Objectives:
* Evaluate the long
Primary Outcome(s): Determine the efficacy of ManNAc therapy in reducing proteinuria in subjects with primary FSGS.; Assess the long-term safety and tolerability of orally administered ManNAc to subjects with primary FSGS.
Enrollment: 30 (ESTIMATED)
Lead Sponsor: National Human Genome Research Institute (NHGRI)
Start: 2026-04-01 | Primary Completion: 2027-11-30