Nizubaglustat for Niemann-Pick Disease

Recruiting at 31 trial locations

Overseen By Contact for Healthcare Professionals

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Azafaros A.G.

Must not be taking: Miglustat

Disqualifiers: Neurologic disease, Hepatic impairment, Renal impairment, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 6

Duration: 18 months

72 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called nizubaglustat for individuals with Niemann-Pick type C disease, a rare condition that impairs the body's ability to process certain fats, leading to neurological issues. The main goal is to determine if nizubaglustat is safe and effective compared to a placebo (a harmless pill with no treatment effect). Suitable candidates for this trial include those diagnosed with Niemann-Pick type C, experiencing neurological symptoms, and not responding to the common treatment miglustat. Participants will receive either nizubaglustat or a placebo during the study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

If you are currently taking miglustat, you will need to stop and have a 1-month period without it before starting the study medication. For other medications, the trial protocol does not specify, so it's best to discuss with the study team.

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

Research has shown that nizubaglustat is safe and well-tolerated. In a previous trial, participants taking nizubaglustat experienced few side effects and reported no major safety issues. The results supported further development of the treatment. This provides confidence that nizubaglustat could be a safe option for patients with Niemann-Pick disease type C. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Nizubaglustat is unique because it offers a new approach for treating Niemann-Pick Disease, which currently relies on enzyme replacement therapies like miglustat. Unlike these treatments, Nizubaglustat is an orally administered tablet, making it potentially more convenient for patients. Additionally, it works by inhibiting glucosylceramide synthase, which could help reduce the accumulation of harmful substances in cells more effectively. Researchers are excited about this treatment because it could provide a simpler and potentially more effective option for managing this condition.

What evidence suggests that nizubaglustat might be an effective treatment for Niemann-Pick disease?

Research has shown that nizubaglustat, which participants in this trial may receive, may be effective in treating Niemann-Pick disease type C. Early studies have found promising signs of its potential benefits for the condition. Specifically, the RAINBOW trial reported that the treatment is safe and may work well. This suggests that nizubaglustat could help manage symptoms or slow the disease's progression. These early results offer hope for its use in treating Niemann-Pick disease type C. ^{3 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with late-infantile and juvenile forms of Niemann-Pick Type C Disease. Specific eligibility criteria are not provided, but typically participants must meet certain health conditions to be included.

Inclusion Criteria

Onset of neurological symptoms from 2 to 15 years

Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline

I am a man willing to follow guidelines to prevent pregnancy during the study.

Exclusion Criteria

The presence of another neurologic disease

My body weight is less than 10 kg.

My liver is not working well.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral nizubaglustat or placebo once daily for 18 months

18 months

Visits at baseline, months 6, 12, and 18

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Nizubaglustat

Trial Overview The study tests the safety and effectiveness of a new oral medication called Nizubaglustat (AZ-3102) compared to a placebo over an 18-month period in a double-blind, randomized, controlled setting across multiple centers.

How Is the Trial Designed?

Group I: Nizubaglustat Experimental Treatment 1 Intervention

Once daily oral dispersible tablets

Group II: Placebo Placebo Group 1 Intervention

Matching placebo

Who Is Running the Clinical Trial?

Azafaros A.G.

Lead Sponsor

Trials

2

Recruited

40+

Citations

1. azafaros.com azafaros.com/news/positive-niemann-pick-disease-type-c-npc-and-gm2-gangliosidosis-data-from-nizubaglustat-phase-2-rainbow-study-conducted-by-azafaros-presented-at-major-metabolic-disease-conference/l188c14

Positive Niemann-Pick disease type C (NPC) and GM2 ... The RAINBOW trial results support the positive safety profile of nizubaglustat and provide us with encouraging early efficacy trends for the compound.

2. inpda.org inpda.org/azafaros-announces-initiation-two-global-phase-3-studies-nizubaglustat-niemann-pick-disease-type-c-npc-gm1gm2-gangliosidoses-respectively/

Azafaros Announces Initiation of two Global Phase 3 ... Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, ...

3. nnpdf.org nnpdf.org/wp-content/uploads/2024/12/Update-from-Azafaros-12.19.2024.pdf

Newsletter for patients on Azafaros' current program The double-blind, placebo-controlled Phase 2 clinical trial is investigating lead asset, nizubaglustat, in patients with either Niemann-Pick disease type C (NPC) ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT07054515

NCT07054515 | A Study to Evaluate the Safety and ... This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple ...

5. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-nizubaglustat-in-patients-with-niemann-pick-type-c-gm1-or-gm2-gangliosidosis/

Study on the Effectiveness and Safety of Nizubaglustat in ... This clinical trial investigates the effectiveness and safety of nizubaglustat in treating Niemann-Pick Type C, GM1, and GM2 Gangliosidosis, ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07054515

NCT07054515 | A Study to Evaluate the Safety and ... This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple ...

7. azafaros.com azafaros.com/news/azafaros-announces-positive-topline-phase-2-study-data-with-nizubaglustat-in-gm2-gangliosidosis-and-niemann-pick-disease-type-c/l187c14

Azafaros announces positive topline Phase 2 study data ... Study designed to support safety profile showed nizubaglustat was safe and well tolerated; Results support continued development of ...

8. forbion.com forbion.com/news-insights/news/azafaros-announces-initiation-of-two-global-phase-3-studies-with-nizubaglustat-in-niemann-pick-disease-type-c-npc-and-gm1gm2-gangliosidoses-respectively/

Azafaros Announces Initiation of two Global Phase 3 studies ... safety and efficacy of the company's lead asset, nizubaglustat, in patients with Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses.