N-Acetyl-L-Leucine for A-T

Recruiting in Palo Alto (17 mi)

+8 other locations

Age: Any Age

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: IntraBio Inc

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Eligibility Criteria

This trial is for individuals aged 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T), which includes symptoms like cerebellar ataxia and epilepsy. Specific eligibility criteria are not provided, but typically include certain health requirements and no recent participation in other studies.

Inclusion Criteria

I agree to use a condom with spermicide during and for 90 days after the study.

My SARA score fits the trial's requirements and I can do specific tests.

I am 4 years or older with a genetic diagnosis of A-T.

I weigh at least 15 kg.

Exclusion Criteria

I have arthritis or a condition that affects my ability to move.

Participant Groups

The study tests the safety, tolerability, and effectiveness of N-Acetyl-L-Leucine (IB1001) compared to a placebo. Participants will be randomly assigned to either receive the actual drug or a placebo without knowing which one they're getting.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: N-acetyl-L-leucine (IB1001)Experimental Treatment1 Intervention

Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients will receive a total daily dose of 2-4 g/day based on weight-tiered doses.

Group II: Placebo comparatorPlacebo Group1 Intervention

Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients will receive a total daily dose of 2-4 g/day based on weight-tiered doses.