Halted gene therapy study raises questions for AMN patients
NCT ID NCT05394064
First seen Jun 25, 2026 · Last updated Aug 27, 2026 · Updated 3 times
Summary
This early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and effectiveness. Researchers were measuring side effects and changes in walking ability over two years.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- SBT101 gene therapy (AAV9-ABCD1)
- What this could lead to
- If successful, this could point toward a treatment that slows or stops the progression of adrenomyeloneuropathy, a rare and disabling nerve disease.
- What could go wrong
- The trial was terminated early with only 8 participants, so results are very limited. Gene therapies can have unexpected side effects, and it is unclear if SBT101 will work in humans.
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
9 people
The number who actually took part.
- Started
-
Nov 2022
- Finished
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Aug 2025
- Lead sponsor
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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
-
18 to 65 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Diagnosed with X-linked adrenoleukodystrophy (ALD), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by elevated circulating VLCFA levels. 2. Clinical evidence of spinal cord involvement but still able to ambulate independently Exclusion Criteria: 1. Evidence of or past diagnosis of inflammatory cerebral disease. 2. 15 years or more have elapsed since the initial onset of myeloneuropathy manifestations such as walking or running difficulties, bladder dysfunction, increased muscular tone, spasticity, weakness, balance problems, etc. 3. Contraindications for MRI procedure and/or contrast materials. 4. Contraindication to steroids, sirolimus, tacrolimus, and/or anesthetic medications. 5. Unstable adrenal function (e.g., untreated or inappropriately treated adrenal insufficiency). 6. History of diabetes or abnormal fasting plasma glucose (≥126 mg/dL) or hemoglobin A1C ≥6.5%. 7. Patients who have received a gene therapy.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Amsterdam UMC
Amsterdam, Netherlands
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University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.