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Gene therapy breakthrough offers hope for rare brain disease

NCT ID NCT04833907

Disease control Sponsor: Myrtelle Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a single dose of AVASPA gene therapy given directly into the brain of children with Canavan disease, a rare and severe genetic disorder that damages white matter. The therapy aims to restore a missing enzyme to help the brain produce myelin and slow disease progression. Up to 24 children will be enrolled across three age groups to evaluate safety and early signs of benefit.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
AVASPA gene therapy (MYR-101)
What this could lead to
If successful, this could provide a treatment that slows or stops the progression of Canavan disease, improving quality of life and extending survival for affected children.
What could go wrong
This is an early-phase trial with only 24 participants, so results may not apply to all. Gene therapy carries risks like immune reactions or surgical complications, and long-term effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2021

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 60 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Definitive diagnosis of typical CD by a board certified neurologist. * Written informed consent from parent(s)/guardian(s). Consent to enroll into the study will include a written agreement to comply with all the conditions of the study, including attendance at follow-up visits. * For cohort 1: age more than 36 months and up to 60 months. * For cohort 2: age between 15 months and 36 months. * For cohort 3: age less than 15 months. Exclusion Criteria: * At the discretion of the PI, any significant chronic medical condition, including, but not limited to neurological, cardiac, hepatic, renal, hematological, gastrointestinal, endocrine, pulmonary, or infectious disease, which would put the subject at increased risk during surgery or which would interfere with participation in the study, interpretation of safety monitoring, or the integrity of the study data. * History of severe allergic reaction or anaphylaxis. * Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment. * Prior intracranial surgery. * Any absolute contraindication to immunosuppression. * Any absolute contraindication to MRI. * Any vaccination less than 1 month prior to gene therapy. * Anticipated life expectancy of less than 12 months for any reason. * GMFM-88 total raw score \>35%. * Clinically significant out-of-range lab values, at the discretion of clinical PI.

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Conditions

The condition(s) this trial relates to.

Canavan disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Canavan disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Dayton Children's Hospital

    Dayton, Ohio, 45404, United States

More trials for these conditions

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