N-Acetyl-L-Leucine for A-T

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, N-acetyl-L-leucine, to determine its potential benefits for individuals with Ataxia-Telangiectasia (A-T), a rare condition affecting movement and coordination. The study aims to assess the safety and effectiveness of this treatment compared to usual care. Participants will receive either the new treatment or a placebo, both administered as a drink mix. Individuals with a confirmed diagnosis of A-T who experience its symptoms may be suitable candidates for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

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 there any evidence suggesting that N-Acetyl-L-Leucine is likely to be safe for humans?

Research has shown that N-Acetyl-L-Leucine (IB1001) has undergone safety testing in several clinical trials. In these studies, most patients tolerated the treatment well. An independent group of experts monitored the trials to ensure safety. Some patients experienced improvements in symptoms such as nausea and constipation, indicating positive effects alongside its safety. While no treatment is entirely risk-free, evidence suggests that N-Acetyl-L-Leucine is safe for people, particularly as it has advanced to this late stage of testing. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for A-T?

N-Acetyl-L-Leucine is unique because it offers a new approach for treating Ataxia-Telangiectasia (A-T) by focusing on a novel compound. Unlike the current options, which mainly include symptom management and supportive therapies, N-Acetyl-L-Leucine is a modified amino acid that may directly impact the underlying mechanisms of the disease. Researchers are excited about this treatment because it is administered orally as a granule that can be mixed with water, orange juice, or almond milk, making it potentially more convenient and easier to incorporate into daily routines. This ease of administration, combined with its novel mechanism of action, positions N-Acetyl-L-Leucine as a promising candidate in the search for more effective A-T therapies.

What evidence suggests that N-Acetyl-L-Leucine might be an effective treatment for A-T?

This trial will evaluate N-acetyl-L-leucine (NALL) as a potential treatment for ataxia-telangiectasia (A-T). Research has shown that NALL might help slow the progression of A-T. Previous studies found that NALL can ease symptoms and improve the quality of life for people with A-T, with patients experiencing less nausea and constipation, common issues with this condition. Additionally, NALL has demonstrated the ability to protect nerve cells from damage. Overall, these findings suggest that NALL could effectively manage symptoms and slow the disease's progression in people with A-T. Participants in this trial will receive either NALL or a placebo comparator. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

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

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

Simultaneous participation in another clinical study involving investigational medicinal products for at least 42 days prior to Visit 1

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. intrabio.com intrabio.com/news/intrabio-reports-positive-data-from-ib1001-multinational-clinical-trial-for-the-treatment-of-niemann-pick-disease-type-c/

IntraBio Reports Positive Data from IB1001 Multinational ... IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03759678

Study Details | NCT03759678 | N-Acetyl-L-Leucine for ... The primary purpose of the study is to evaluate the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the treatment of A-T investigating the efficacy in ...

3. neurologylive.com neurologylive.com/view/n-acetyl-l-leucine-reduces-disease-progression-phase-2-extension-study-ataxia-telangiectasia

N-Acetyl-L-Leucine Reduces Disease Progression in ... N-acetyl-L-leucine (NALL) showed potential neuroprotective effects in ataxia-telangiectasia, slowing disease progression in phase 2 trial ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9990862/

Efficacy and Safety of N-Acetyl-l-Leucine in Children and ... In total, this study provides Class IV evidence that NALL improves outcomes for patients with GM2 gangliosidoses. Discussion. In this phase IIb ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1090379824000552

Efficacy and safety of N-acetyl-L-leucine in patients with ... This was the first placebo-controlled trial studying NALL's effects on AT. NALL improved nausea and constipation in AT patients, providing a clinical benefit.

6. intrabio.com intrabio.com/our-lead-program/

Lead Program IB1001 is an orally administered, modified amino acid (N-Acetyl-L-Leucine). Its safety and tolerability profile has been studied extensively in clinical trials.

7. medrxiv.org medrxiv.org/content/10.1101/2020.03.11.20034256v1.full-text

A master protocol to investigate a novel therapy acetyl-L- ... IB1001-203 investigates N-acetyl-L-leucine for the treatment of Ataxia Telangiectasia (A-T). A-T is an ultra-rare (1:40,000-100,000), autosomal ...