Ataxia-Telangiectasia (A-T)NCT07380165
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
A study testing Levacetylleucine in people with Ataxia-Telangiectasia (A-T), run by IntraBio Inc.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Ataxia-Telangiectasia (A-T)
- What is being tested?
- Levacetylleucine
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
About this study
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.
Read the detailed 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.
- Conditions
- Ataxia-Telangiectasia (A-T)
- Being tested
- Levacetylleucine
- Keywords
- Ataxia-Telangiectasia, Louis Bar Syndrome
- Medical subject headings
- Ataxia Telangiectasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LevacetylleucineDrug
Also called AQNEURSA, N-Acetyl-L-leucine
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ataxia-Telangiectasia (A-T)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- IntraBio Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07380165
- Sponsor's protocol ID
- IB1001-EA3
- Status last verified
- Sep 2026
- First submitted
- 23 Jan 2026
- Last updated
- 28 Sep 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
- ClinicalTrials.gov ID
- NCT07380165
- Acronym / sponsor ID
- IB1001-EA3
- Conditions
- Ataxia-Telangiectasia (A-T)
- Keywords
- Ataxia-Telangiectasia, Louis Bar Syndrome
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- IntraBio IncIndustry (pharma, biotech, devices)
- First posted
- 2 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LevacetylleucineAlso: AQNEURSA, N-Acetyl-L-leucine1 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. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Levacetylleucine in people with Ataxia-Telangiectasia (A-T), run by IntraBio Inc.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2026.