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Amino Acid

N-Acetyl-L-Leucine for A-T

Phase 3

Waitlist Available

Research Sponsored by IntraBio Inc

Eligibility Criteria Checklist

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

Must have

Non-vasectomized male patient agrees to use a condom with spermicide during the study until 90 days beyond the last dose of study medication

Patients must fall within specific SARA score criteria and be able to perform certain tests

Must not have

Patients diagnosed with arthritis or musculoskeletal disorders affecting mobility

Timeline

Screening 3 weeks

Treatment Varies

Follow Up end of period i (week 12) vs. end of period 2 (week 24)

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new medication called N-acetyl-L-leucine (IB1001) in patients aged 4 and older with Ataxia-Telangiectasia. The goal is

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, common ones may include digestive issues, headaches, dizziness or allergic reactions. The trial aims to assess these potential side effects thoroughly.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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

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

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I weigh at least 15 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ end of period i (week 12) vs. end of period 2 (week 24)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~end of period i (week 12) vs. end of period 2 (week 24)

This trial's timeline: 3 weeks for screening, Varies for treatment, and end of period i (week 12) vs. end of period 2 (week 24) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Scale for the Assessment and Rating of Ataxia

Secondary study objectives

EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale

International Cooperate Ataxia Rating Scale (ICARS)

Physician's / Caregiver's / Patient's Clinical Global Impressions (CGI)

+1 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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.

0 / 5Eligibility criteria met