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AZ-3102 (Dose 1) for Tay-Sachs Disease (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 concentration versus time curve calculated from time 0 to 24 hours (auc0-24h)

Awards & highlights

RAINBOW Trial Summary

This trial tests a potential medicine for GM2 Gangliosidosis and Niemann-Pick type C diseases. It's double-blind, placebo-controlled and 12 weeks long. If approved, patients can extend their treatment.

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.Check my eligibility

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.See study design

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.

RAINBOW Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ concentration versus time curve calculated from time 0 to 24 hours (auc0-24h)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~concentration versus time curve calculated from time 0 to 24 hours (auc0-24h)

This trial's timeline: 3 weeks for screening, Varies for treatment, and concentration versus time curve calculated from time 0 to 24 hours (auc0-24h) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Assessment of PK parameters in plasma: AUC0-24h

Assessment of PK parameters in plasma: Tmax

Assessment of pharmacokinetic (PK) parameters in plasma: Cmax

+1 more

RAINBOW 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

Find a Location

Who is running the clinical trial?

Azafaros A.G.Lead Sponsor

1 Previous Clinical Trials

31 Total Patients Enrolled

1 Trials studying Tay-Sachs Disease

31 Patients Enrolled for Tay-Sachs Disease

Media Library

AZ-3102 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05758922 — Phase 2

Tay-Sachs Disease Research Study Groups: Placebo, AZ-3102 (Dose 1), AZ-3102 (Dose 2)

Tay-Sachs Disease Clinical Trial 2023: AZ-3102 Highlights & Side Effects. Trial Name: NCT05758922 — Phase 2

AZ-3102 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05758922 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the trial extend eligibility to individuals aged 65 and older?

"Patients aged between 12 to 20 are eligible for enlistment into this medical study, while those younger than 18 and older than 65 can join one of the 16 other trials."

Answered by AI

What level of jeopardy does AZ-3102 (Dose 1) present to individuals?

"Taking into account the Phase 2 status of AZ-3102 (Dose 1), our team at Power determined a safety score of two, denoting that there is some evidence for its safety but no data regarding efficacy."

Answered by AI

May I join the current clinical trial experiment?

"This clinical trial seeks adolescents aged between 12 and 20 who have been diagnosed with Tay-Sachs. A maximum of twelve participants will be admitted into the study."

Answered by AI

Is recruitment for this investigation still open?

"This investigation, which was first advertised on April 24th 2023, is currently looking for participants. The data hosted on clinicaltrials.gov has been newly revised as of May 20th 2023."

Answered by AI

How many venues is the clinical trial being conducted in?

"This medical trial has 4 available locations: Hospital Pequeno Principe in Curitiba, Hospital de Clinicas de Porto Alegre in Porto Alegre, Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira in Rio De Janeiro as well as 4 other sites."

Answered by AI

How many research participants are involved in this experiment?

"Indeed, according to clinicaltrials.gov the trial was first published on April 24th 2023 and is actively searching for 12 participants across 4 different sites. The study has recently been revised on May 20th 2021."

Answered by AI

Who else is applying?

What site did they apply to?

Hospital Pequeno Principe

What portion of applicants met pre-screening criteria?

Did not meet criteria

Why did patients apply to this trial?

My 8 year son Vasco Cardoso Lopes have been diagnostic this year whith Gangliosidosis GM2 variant B1, and the ONLY big Lost to this date its was de speech. He born in 5 August 2015, but we give our consent to been enroled on the study Azafaros - AZ 3102 if you gives us a chance. We live in Portugal but we maket possibel.

PatientReceived no prior treatments

Recent research and studies

clinicaltrials.govStudy

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

2023. "Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05758922.

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