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Gene Therapy Trial Report

Summary

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)


NCTID NCT05394064 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Adrenomyeloneuropathy (AMN)
Disease Ontology Term DOID:10588
Compound Name SBT101
Sponsor SwanBio Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 8 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant ABCD1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 1.0E14 vg (n=4)
Dose 2 3.0E14 vg (n=4)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-05-16
Completion Date 2025-08-31
Last Update 2025-10-31

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations Netherlands,United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Study was terminated for business/strategic reasons, not due to safety concerns

Resources/Links