NCT07380165

Brief Summary

This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.

Trial Health

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 23, 2026

Completed
10 days until next milestone

First Posted

Study publicly available on registry

February 2, 2026

Completed
Last Updated

February 24, 2026

Status Verified

February 1, 2026

First QC Date

January 23, 2026

Last Update Submit

February 20, 2026

Conditions

Ataxia-Telangiectasia (A-T)

Keywords

Ataxia-TelangiectasiaLouis Bar Syndrome

Interventions

LevacetylleucineDRUG

1 gram levacetylleucine as granules in unit-dose packet. Investigational drug is administered as oral suspension, up to three times per day, based on body weight.

Also known as: AQNEURSA, N-Acetyl-L-leucine

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patient has a genetically confirmed diagnosis of Ataxia-Telangiectasia, a rare and serious disease with no alternative approved treatments.
  • Patient and/or their legal representative signed a written informed consent and, if applicable, written assent.
  • Patient weighs ≥15 kg.
  • If female and of reproductive potential, the treating physician verified the patient is not pregnant prior to enrollment and will use adequate contraceptive methods to avoid pregnancy during and up to 7 days after participating in the program.
  • Patient resides in the United States at time of enrollment and throughout the duration of their participation in the EAP.

You may not qualify if:

  • Patient is enrolled in or early withdrew participation from a clinical trial with levacetylleucine for A-T.
  • Patient is pregnant, breastfeeding, or considering pregnancy.
  • Patient has a physical, cognitive, psychiatric condition, or an active or suspected malignancy that, in the judgment of the treating physician, may pose a safety risk or interfere with the patient's ability to comply with the EAP procedures.
  • Patient is receiving N-acetyl-DL-leucine or N-acetyl-D-leucine and is unwilling to stop use throughout the duration of their participation in the EAP.
  • Patient is receiving another investigational drug for any reason.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Ataxia Telangiectasia

Interventions

acetylleucine

Condition Hierarchy (Ancestors)

Spinocerebellar AtaxiasCerebellar AtaxiaCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurocutaneous SyndromesAtaxiaDyskinesiasNeurologic ManifestationsTelangiectasisVascular DiseasesCardiovascular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPrimary Immunodeficiency DiseasesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Central Study Contacts

IntraBio Medical Affairs

CONTACT

1-833-306-9677medinfo@intrabio.com

Megan Grosso

CONTACT

ibmedical@intrabio.com

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 23, 2026

First Posted

February 2, 2026

Last Updated

February 24, 2026

Record last verified: 2026-02