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

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.

Why the study stopped: The SBT101 program for AMN was terminated for business/strategic reasons, and there were no safety concerns

Terminated

Looking for future studies?

Notify Me

Key information

Conditions

AMN AMN Gene Mutation Adrenal Gland Diseases Adrenal Insufficiency Adrenoleukodystrophy Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Diseases Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Hereditary Sensory and Motor Neuropathy Heredodegenerative Disorders, Nervous System Intellectual Disability Leukoencephalopathies Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nervous System Malformations Neurobehavioral Manifestations Neurodegenerative Diseases Neurologic Manifestations Neuromuscular Diseases Nutritional and Metabolic Diseases Paralysis Paraplegia Pathological Conditions, Signs and Symptoms Peripheral Nervous System Diseases Peroxisomal Disorders Polyneuropathies Signs and Symptoms Spastic Paraplegia, Hereditary X-ALD X-Linked Intellectual Disability

Age range

18 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Amsterdam UMC, Amsterdam, Netherlands

About this study

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 consist of 2 phases:

Phase 1: Dose-Escalation Phase: Two (2) doses of SBT101 (Dose level 1 cohort and Dose Level 2 cohort) will be evaluated to establish the maximum tolerated dose (MTD).

Phase 2: Dose-Expansion Phase: Additional patients will be enrolled to receive SBT101 at the MTD

Part 2: An unblinded 3-year long-term safety follow-up period with annual follow-up visits to evaluate the safety of SBT101 and disease progression.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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 criteria

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.

Treatment and study plan

SBT101 Treatment

Genetic

SBT101 Treatment

Primary outcomes

Adverse Events to SBT101. Any Serious TEAE. Any Serious TEAE Related to the Study Procedure or Study Drug.

Time frame: 2 years

Any Treatment Emergent Adverse Event (TEAE). TEAE was defined as any adverse event which started during or after the administration of IMP or the immunosuppressant pre-medication.

Sponsors and collaborators

Lead sponsor

SwanBio Therapeutics, Inc.

Industry

Registry information

Official study title

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

Acronym: PROPEL

Important dates

Study start: 2022
Primary completion: 2025
Study completion: 2025
First posted: May 27, 2022
Registry last updated: Aug 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed