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

Stopped early · Phase 1/Phase 2 · Has a placebo group

Conditions studied: AMN, AMN Gene Mutation, X-ALD

In brief

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.

Key facts

Study ID

NCT05394064

Run by

SwanBio Therapeutics, Inc.

People needed

8

Starts

2022-11-17

Expected to finish

2025-08-31

Last updated by the study team

2025-10-31

Who can join

Age: 18 and older, up to 65. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States
• Amsterdam UMC — Amsterdam, Netherlands

Full record on ClinicalTrials.gov

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