N-Acetyl-L-Leucine for A-T

Recruiting at 10 trial locations

Overseen By Taylor Fields, MSt

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: IntraBio Inc

Must not be taking: N-Acetyl-DL-Leucine, Sulfasalazine, Rosuvastatin

Disqualifiers: Hypersensitivity, Pregnancy, Severe vision/hearing, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 7

60 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

You can continue taking your current medications if they are not on the prohibited list and if you have been on a stable dose for at least 42 days before the study starts. However, you must stop taking certain medications like N-Acetyl-DL-Leucine, N-Acetyl-L-Leucine (unless provided in the trial), Sulfasalazine, and Rosuvastatin for 42 days before the study begins and throughout the study.

Is N-Acetyl-L-Leucine safe for humans?

In a study with a 9-year-old girl with Ataxia-Telangiectasia, N-acetyl-DL-leucine was taken for 16 weeks and was generally safe, with only mild nausea and constipation in the first week. ^{1 2 3 4 5}

How is the drug N-Acetyl-L-Leucine unique for treating A-T?

N-Acetyl-L-Leucine is unique because it is being explored as a treatment for A-T (Ataxia-Telangiectasia), a condition with limited treatment options, and it may offer a novel approach by potentially modifying disease mechanisms rather than just alleviating symptoms. ^{6 7 8 9 10}

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 am not sexually active or using effective birth control.

I am a woman who cannot have children because I had surgery for it over 6 months ago.

Understanding of study participation implications and willingness to comply with instructions and attend required study visits

Exclusion Criteria

Patients with physical or psychiatric conditions that may put them at risk or interfere with study participation

Patients with known hypersensitivity to specific substances

Known or persistent use, misuse, or dependency of medication, drugs, or alcohol

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

1 visit (in-person)

Baseline

Baseline assessments conducted before randomization

2 weeks

1 visit (in-person)

Treatment Period I

Participants receive either IB1001 or Placebo for 12 weeks

12 weeks

2 visits (in-person)

Treatment Period II

Participants crossover to receive the opposite treatment for 12 weeks

12 weeks

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

Extension Phase

Participants may opt into continuation of treatment with IB1001

Long-term

Treatment Details

Interventions

• N-Acetyl-L-Leucine

Trial Overview 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.

Participant Groups

Group I: N-acetyl-L-leucine (IB1001) Experimental Treatment 1 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 comparator Placebo Group 1 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.

Who Is Running the Clinical Trial?

IntraBio Inc

Lead Sponsor

Trials

5

Recruited

220+

References

1. United States pubmed.ncbi.nlm.nih.gov

Predictors of outcome after acetaminophen poisoning in children and adolescents. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

The Effect of N-Acetyl-DL-Leucine on Neurological Symptoms in a Patient with Ataxia-Telangiectasia: a Case Study. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Plasma and urinary methionine levels in one-year-old infants after oral loading with L-methionine and N-acetyl-L-methionine. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

What is the rate of adverse events after oral N-acetylcysteine administered by the intravenous route to patients with suspected acetaminophen poisoning? [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Portal and vena caval plasma methionine concentrations in young pigs administered L-methionine, N-acetyl-L-methionine and N-acetyl-D-methionine. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Molecular characterization of a novel N-acetyltransferase from Chryseobacterium sp. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Reversible lysine acetylation regulates activity of human glycine N-acyltransferase-like 2 (hGLYATL2): implications for production of glycine-conjugated signaling molecules. [2021]

8. Netherlands pubmed.ncbi.nlm.nih.gov

N-acetyltransferases from three different organisms displaying distinct selectivity toward hexosamines and N-terminal amine of peptides. [2019]

9. United States pubmed.ncbi.nlm.nih.gov

Identification of a novel allele at the human NAT1 acetyltransferase locus. [2007]

10. England pubmed.ncbi.nlm.nih.gov

Preparation of optically pure tert-leucine by penicillin G acylase-catalyzed resolution. [2013]