ManNAc for FSGS

Overseen By William A Gahl, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: National Human Genome Research Institute (NHGRI)

Must not be taking: Interferons, Lithium, Androgenic steroids, others

Disqualifiers: Uncontrolled nephrotic syndrome, Uncontrolled hypertension, Active infection, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 8

Duration: 12 weeks

30 Participants Needed

Trial Summary

What is the purpose of this trial?

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 6 to 7 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.

Will I have to stop taking my current medications?

You may not need to stop your current medications. If you are on immunosuppressive therapy, you should have been on a stable dose for at least 4 weeks before the trial and continue it during the trial. You can also continue other standard treatments for FSGS if you maintain a stable dose throughout the trial.

Research Team

William A Gahl, M.D.

Principal Investigator

National Human Genome Research Institute (NHGRI)

Eligibility Criteria

This trial is for adults aged 18 and older diagnosed with Focal Segmental Glomerulosclerosis (FSGS), a kidney disease that causes scarring and can lead to protein loss in urine. Participants will need to attend several clinic visits, some requiring overnight stays.

Inclusion Criteria

I had a kidney biopsy showing FSGS within the last 5 years.

I have been on a stable dose of immunosuppressive therapy for at least 4 weeks.

Evidence of a personally signed and dated informed consent indicating willingness to comply with all aspects of the study protocol

Exclusion Criteria

Individuals with interstitial fibrosis and tubular atrophy (IFTA) >50% per biopsy report

I am seeking treatment for the first time for uncontrolled nephrotic syndrome.

Individuals with psychiatric illness or neurological disease that may interfere with protocol requirements

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ManNAc 2,000 mg orally twice daily for 12 weeks

12 weeks

6 to 7 visits (in-person), including 2 overnight stays

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

Follow-up phone calls every 2 weeks after each clinic visit

Treatment Details

Interventions

• ManNAc

Trial Overview The study is testing ManNAc, a white powder dissolved in water taken orally twice daily, as a potential treatment for FSGS. The trial includes regular clinic visits for physical exams, blood and urine tests, health questionnaires, and possible dietary consultations.

Participant Groups

Group I: open label, single arm Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

National Human Genome Research Institute (NHGRI)

Lead Sponsor

Trials

273

Recruited

299,000+