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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ManNAc for individuals with focal segmental glomerulosclerosis (FSGS), a kidney disease that causes scarring and can lead to kidney failure. ManNAc, a powder mixed with water and taken orally, aims to improve kidney function and reduce protein loss in urine, a common issue with FSGS. Individuals who have had a kidney biopsy showing FSGS within the past 10 years and maintain stable doses of any current treatments might be suitable for this trial. Participants will visit the clinic several times over 14 weeks, with some overnight stays for detailed monitoring. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that ManNAc is likely to be safe for humans?

Research has shown that ManNAc is safe and well-tolerated in people. Long-term use of ManNAc has not raised major safety concerns in past studies. Specifically, earlier research confirmed its safety for those who used it over time. While researchers continue to study ManNAc for FSGS, these results are promising for its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for FSGS (Focal Segmental Glomerulosclerosis), such as corticosteroids and immunosuppressants, work by suppressing the immune system to reduce inflammation. But ManNAc works differently, focusing on a new approach by targeting the metabolic pathway. Researchers are excited about ManNAc because it aims to increase the production of sialic acid, a crucial component for kidney health, which may address the root cause of the disease rather than just managing symptoms. This unique mechanism could offer a new avenue for effective treatment with potentially fewer side effects compared to the current standard therapies.

What evidence suggests that ManNAc might be an effective treatment for FSGS?

Research suggests that ManNAc might help reduce protein loss in urine for people with focal segmental glomerulosclerosis (FSGS), a kidney condition. Earlier studies found that ManNAc is safe and may lower protein levels in urine for those with certain kidney problems. This is encouraging because reducing protein loss can help protect kidney health. However, more research is needed to confirm ManNAc's effectiveness specifically for FSGS. ^{1 4 6 7 8}

Who Is on the Research Team?

William A Gahl, M.D.

Principal Investigator

National Human Genome Research Institute (NHGRI)

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06664814

NCT06664814 | An Open-Label Phase 2 Study of N-Acetyl ... Primary Efficacy Objective: Determine the efficacy of ManNAc therapy in reducing proteinuria in subjects with primary FSGS. Primary Safety Objective: Assess the ...

2. clinicalstudies.info.nih.gov clinicalstudies.info.nih.gov/protocoldetails.aspx?id=002066-HG&&query=

NIH Clinical Center: Search the Studies An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects with Primary Focal Segmental Glomerulosclerosis. This study is currently recruiting ...

3. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06664814/study-of-n-acetyl-d-mannosamine-mannac-in-subjects-with-primary-focal-segmental-glomerulosclerosis

An Open-Label Phase 2 Study of N-Acetyl-D ... The study will characterize the long-term safety, tolerability, pharmacokinetics, and efficacy of ManNAc for proteinuria reduction in subjects ...

4. journals.lww.com journals.lww.com/jasn/fulltext/2020/10001/phase_1_study_of_n_acetylmannosamine__mannac__for.2088.aspx

Phase 1 Study of N-Acetylmannosamine (ManNAc) for... Oral ManNAc was safe and well tolerated in glomerular disease subjects. ManNAc supplementation showed a trend towards proteinuria reduction.

5. sciencedirect.com sciencedirect.com/science/article/pii/S2468024919313956

Rationale and Design for a Phase 1 Study of N ... We recently demonstrated that ManNAc was safe and well tolerated in a first-in-human phase 1 study in subjects with UDP-N-acetylglucosamine (GlcNAc) 2-epimerase ...

6. nephcure.org nephcure.org/trials/mannac-nih-bethesda-md/

ManNAc NIH- Bethesda, MD 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 ...

7. techtransfer.nih.gov techtransfer.nih.gov/tech/tab-3848

Treating Kidney Disorders and Diabetic Nephropathy with ... Long-term ManNAc administration has been shown to be safe and well-tolerated in humans. ... focal segmental glomerulosclerosis has been approved ...

8. withpower.com withpower.com/trial/phase-2-glomerulosclerosis-focal-segmental-10-2024-f08ac

ManNAc for FSGS · Recruiting Participants for Phase ... This Phase 2 medical study run by National Human Genome Research Institute (NHGRI) is evaluating whether ManNAc will have tolerable side effects & efficacy ...