Status:
TERMINATED
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
Lead Sponsor:
SwanBio Therapeutics, Inc.
Conditions:
AMN
AMN Gene Mutation
Eligibility:
MALE
18-65 years
Phase:
PHASE1
PHASE2
Brief Summary
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) ...
Detailed Description
The study consists of two parts after infusion of SBT101: Part 1: A blinded 24-month core study period to evaluate the safety and potential impact of SBT101 on disease progression. Part 1 will consis...
Eligibility Criteria
Inclusion
- Diagnosed with X-linked adrenoleukodystrophy (ALD), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by elevated circulating VLCFA levels.
- Clinical evidence of spinal cord involvement but still able to ambulate independently
Exclusion
- Evidence of or past diagnosis of inflammatory cerebral disease.
- 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.
- Contraindications for MRI procedure and/or contrast materials.
- Contraindication to steroids, sirolimus, tacrolimus, and/or anesthetic medications.
- Unstable adrenal function (e.g., untreated or inappropriately treated adrenal insufficiency).
- History of diabetes or abnormal fasting plasma glucose (≥126 mg/dL) or hemoglobin A1C ≥6.5%.
- Patients who have received a gene therapy.
Key Trial Info
Start Date :
November 17 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 31 2025
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT05394064
Start Date
November 17 2022
End Date
August 31 2025
Last Update
October 31 2025
Active Locations (2)
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1
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States, 01655
2
Amsterdam UMC
Amsterdam, Netherlands