What side effects are associated with this type of intervention?

Common treatments for Tay-Sachs Disease, such as gene therapy, aim to correct the underlying enzyme deficiency. In studies, gene therapy has shown promise in re-establishing Hex A activity and removing GM2 ganglioside storage without observed adverse effects related to the viral vector, injection site, or gene expression. For oral treatments like AZ-3102, side effects are still being evaluated, but generally, such treatments may include gastrointestinal issues, liver enzyme elevations, and potential risks of infections due to immunomodulation.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Tay-Sachs Disease. The management of Tay-Sachs primarily involves supportive care to manage symptoms and improve quality of life. The trial AZ-3102 is investigating a new drug for GM2 Gangliosidosis, which includes Tay-Sachs Disease, focusing on its safety and pharmacokinetics. This trial represents ongoing efforts to find effective treatments for this condition, but no similar drugs have received FDA approval to date.

What other types of research exist?

Promising novel alternatives to AZ-3102 for Tay-Sachs Disease patients include gene therapy approaches such as AXO-AAV-GM2, which delivers a functional copy of the HEXA gene, and substrate reduction therapies like miglustat, which reduces the accumulation of GM2 ganglioside. Additionally, enzyme replacement therapies (ERT) are being explored, although none are currently approved specifically for Tay-Sachs Disease.

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AZ-3102 for Tay-Sachs and Niemann-Pick Diseases (RAINBOW Trial)

Phase 2

Waitlist Available

Research Sponsored by Azafaros A.G.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial tests an oral medication called AZ-3102 in patients with GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C). It aims to see if the drug is safe and how it works in their bodies.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, common ones may include digestive discomfort, headaches, fatigue or allergic reactions. The trial will closely monitor participants for any adverse effects related to AZ-3102.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: AZ-3102 (Dose 2)Experimental Treatment1 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 Treatment1 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: PlaceboPlacebo Group1 Intervention

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

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Tay-Sachs Disease is primarily treated through approaches that aim to reduce the accumulation of GM2 gangliosides in neurons. Substrate reduction therapy (SRT) works by inhibiting the synthesis of glycosphingolipids, thereby reducing the substrate that accumulates due to the deficient enzyme. Enzyme replacement therapy (ERT) involves supplementing the deficient enzyme, hexosaminidase A, to break down GM2 gangliosides. These treatments are crucial for Tay-Sachs patients as they target the underlying cause of neurodegeneration, potentially slowing disease progression and alleviating symptoms. The trial AZ-3102, which evaluates a treatment for GM2 Gangliosidosis, is likely exploring similar mechanisms to improve patient outcomes.

Genetics and Therapies for GM2 Gangliosidosis.