AZ-3102 for Tay-Sachs and Niemann-Pick Diseases

(RAINBOW Trial)

Not currently recruiting at 4 trial locations

Overseen By Cécile Paquet-Luzy

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Azafaros A.G.

Must not be taking: Anti-epileptics, Azasugars

Disqualifiers: Inherited neurologic disease, Renal impairment, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment, AZ-3102, for individuals with Tay-Sachs and Niemann-Pick type C diseases. The goal is to assess the treatment's safety and behavior in the body over 12 weeks. Participants will receive either AZ-3102 or a placebo (a non-active substance) daily, and those who complete the study may continue for an extended period. This trial suits individuals diagnosed with Tay-Sachs or Niemann-Pick type C who have not recently used certain treatments like miglustat. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in treatment.

Will I have to stop taking my current medications?

The trial requires that if you have been taking miglustat, it must be stopped at least 1 month before starting the study. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that AZ-3102 has a promising safety record for people with Niemann-Pick disease type C (NP-C) and GM2 Gangliosidosis. In earlier studies, most patients tolerated the treatment well. These studies found early signs that the treatment might work without causing major safety problems. Participants took AZ-3102 as a pill, which they swallowed instead of receiving an injection or IV.

Some studies tested different doses of AZ-3102, including amounts similar to those in the current trial, to assess safety. So far, reports of serious side effects have not emerged. However, like any medication, some people might still experience mild side effects, which is why ongoing trials are important.

This treatment is still under investigation. While early evidence suggests safety, researchers continue to gather data to confirm this. For those considering joining a trial, it may be helpful to know that AZ-3102 has been studied in people before, and the results have been encouraging so far. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

AZ-3102 is unique because it offers a new approach for treating Tay-Sachs and Niemann-Pick diseases, conditions that currently have limited treatment options primarily focused on symptom management. Unlike existing treatments, AZ-3102 targets the underlying causes of these diseases with a novel mechanism of action, potentially altering disease progression. Researchers are excited about AZ-3102 because it could provide more effective and lasting relief than current therapies, offering hope for improved quality of life for patients. Additionally, AZ-3102 is being tested in two different doses, which may help identify the most effective regimen for patients.

What evidence suggests that this trial's treatments could be effective for Tay-Sachs and Niemann-Pick diseases?

Research shows that AZ-3102 could help treat Tay-Sachs and Niemann-Pick diseases. Earlier studies found promising signs of symptom improvement related to these conditions. In this trial, participants will receive either AZ-3102 at different doses or a placebo. The treatment acts as an azasugar, addressing the root causes of these rare diseases. This approach aims to help the body manage the buildup of harmful substances. These early findings offer hope that AZ-3102 could become a helpful option for patients with these conditions. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

This trial is for 12-20 year olds with GM2 Gangliosidosis or Niemann-Pick type C disease. Participants need a confirmed diagnosis, and if they're sexually active, must follow contraceptive guidance. They can't have severe kidney issues, be pregnant/breastfeeding, or have taken certain drugs recently.

Inclusion Criteria

Total SARA score ≥ 1 at Baseline

Willing and able to complete protocol assessments

Parent and/or legal guardian is able to read, understand, and sign the informed consent. Assent will also be sought for patients who have not reached the age of majority or who are not able to sign the consent form

Exclusion Criteria

Platelet count < 100 x 10^9/L

Positive serum pregnancy test at baseline for women of childbearing potential

Ongoing breastfeeding at baseline or planned during the study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily oral administration of AZ-3102 or placebo for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Double-blind extension

Participants who complete the 12-week study may enter a double-blind extension period

What Are the Treatments Tested in This Trial?

Interventions

• AZ-3102
• Placebo

Trial Overview The study tests the safety and effects of AZ-3102 (in two doses) compared to a placebo in patients over 12 weeks. It's randomized and double-blind, meaning neither doctors nor patients know who gets the real drug versus placebo.

How Is the Trial Designed?

Group I: AZ-3102 (Dose 2) Experimental Treatment 1 Intervention

Participant will receive AZ-3102 (Dose 2) once daily during the course of the study (12 weeks) and the study extension (if applicable).

Group II: AZ-3102 (Dose 1) Experimental Treatment 1 Intervention

Participant will receive AZ-3102 (Dose 1) once daily during the course of the study (12 weeks) and the study extension (if applicable).

Group III: Placebo Placebo Group 1 Intervention

Participant will receive placebo once daily during the course of the study (12 weeks).

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. 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 ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05758922

NCT05758922 | Phase 2 Study Evaluating the Safety, ... This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and ...

4. withpower.com withpower.com/trial/phase-2-pick-disease-of-the-brain-3-2023-d3d95

AZ-3102 for Tay-Sachs and Niemann-Pick Diseases This trial tests an oral medication called AZ-3102 in patients with GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C).

5. azafaros.com azafaros.com/news/azafaros-announces-enrollment-of-first-patient-in-phase-2-rainbow-study-evaluating-az-3102-in-gm2-and-np-c-patients/l92c14

Azafaros Announces Enrollment of First Patient in Phase 2 ... AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage ...

6. mayo.edu mayo.edu/research/clinical-trials/cls-20547788

ORAL AZ-3102 in Patients With GM2 Gangliosidosis or ... The purpose of this study is to assess the safety and tolerability of daily administration of 3 mg and 9 mg AZ-3102 versus placebo when ...