A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuro… (NCT05394064) | Clinical Trial Compass
TerminatedPhase 1/2
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
Stopped: The SBT101 program for AMN was terminated for business/strategic reasons, and there were no safety concerns
United States, Netherlands8 participantsStarted 2022-11-17
Plain-language 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) 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.
Who can participate
Age range18 Years – 65 Years
SexMALE
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Inclusion criteria
âś“. Diagnosed with X-linked adrenoleukodystrophy (ALD), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by elevated circulating VLCFA levels.
âś“. Clinical evidence of spinal cord involvement but still able to ambulate independently
Exclusion criteria
âś•. Evidence of or past diagnosis of inflammatory cerebral disease.
âś•. 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.
âś•. Contraindications for MRI procedure and/or contrast materials.
âś•. Contraindication to steroids, sirolimus, tacrolimus, and/or anesthetic medications.
âś•. 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%.