Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)

IntraBio

Status

Conditions: Ataxia-Telangiectasia (A-T)

Treatments: Drug: Levacetylleucine

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT07380165

IB1001-EA3

Details and patient eligibility

About

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.

Full description

This expanded access program provides access to investigational drug, levacetylleucine, for eligible patients with Ataxia-Telangiectasia that are not eligible for other clinical trials, and a medical doctor has decided there is potential benefit outweighing the risk of receiving an investigational therapy based on the patient's medical history and program eligibility criteria.

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patient has a genetically confirmed diagnosis of Ataxia-Telangiectasia, a rare and serious disease with no alternative approved treatments.
2. Patient and/or their legal representative signed a written informed consent and, if applicable, written assent.
3. Patient weighs ≥15 kg.
4. 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.
5. Patient resides in the United States at time of enrollment and throughout the duration of their participation in the EAP.

Exclusion criteria

1. Patient is enrolled in or early withdrew participation from a clinical trial with levacetylleucine for A-T.
2. Patient is pregnant, breastfeeding, or considering pregnancy.
3. 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.
4. 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.
5. Patient is receiving another investigational drug for any reason.