A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Pat
A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients With Adrenomyeloneuropathy
What it found
The trial was stopped early for safety reasons.
Reason given: The SBT101 program for AMN was terminated for business/strategic reasons, and there were no safety concerns
What was tested
Treatment tested
- SBT101 Treatment
Results by group
Adverse Events to SBT101. Any Serious TEAE. Any Serious TEAE Related to the Study Procedure or Study Drug.
2 years
| Group | Value | Interval | Number analysed |
|---|---|---|---|
| Cohort 1 - Dose Level 1 | 4 Participants | 4 | |
| Cohort 2 - Dose Level 2 | 4 Participants | 4 | |
| Cohort 1 - Dose Level 1 | 2 Participants | 4 | |
| Cohort 2 - Dose Level 2 | 0 Participants | 4 |
Cohort 1 - Dose Level 1
Cohort 2 - Dose Level 2
Cohort 1 - Dose Level 1
Cohort 2 - Dose Level 2
Values as posted on ClinicalTrials.gov. Outcomes measured differently across trials are not directly comparable.
Safety
| Group | Participants at risk | Serious adverse events | Deaths |
|---|---|---|---|
| Cohort 1 - Dose Level 1 | 4 | 3 (75.0%) | 1 |
| Cohort 2 - Dose Level 2 | 4 | 2 (50.0%) | 0 |
As posted on ClinicalTrials.gov. Serious adverse events include any serious health event during the trial, whether or not the treatment caused it.
What it measured
Main measures
Adverse Events to SBT101. Any Serious TEAE. Any Serious TEAE Related to the Study Procedure or Study Drug.
2 years
Safety
Who could take part
MS (type not specified) · 18 to 65
Show the full criteria
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.
As registered. Criteria can change, so ask the trial team or your neurologist.
About this trial
In the sponsor's words
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years. Patients will receive a single dose of SBT101 via IT route (or an imitation procedure) and will be followed for safety and efficacy for 2 years. Patients receiving SBT101 will be followed for an additional 3 years (5 total) for Safety. Patients receiving an imitation procedure will be offered the opportunity to receive SBT101 after 2 years, as data indicate.
Papers
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History
Started
Nov 2022
Main results due
Jun 2025
Finished
Aug 2025
Results posted
Aug 2026