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

NCT05394064 | Terminated Phase 1 DMC FDA Drug

• 2 other identifiers: SBT101-CT1012021-004410-19
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 2

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) 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.

Conditions

AMN; AMN Gene Mutation; X-ALD

Keywords

AMN; X-ALD; Adrenoleukodystrophy; X-linked Adrenoleukodystrophy; Adrenomyeloneuropathy; Myeloneuropathy; Spastic paraplegia; Hereditary Spastic Paraplegia; HSP; ALD; ABCD1; ALDP; CALD; CCALD; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Adrenal Insufficiency; Gene Therapy; AAV9; Adeno-Associated Vector

Outcome Measures

Primary Outcomes (1)

• Adverse Events to SBT101

  Safety and tolerability of SBT101 administration

  2 years

Secondary Outcomes (1)

• Disease progression

  2 years

Other Outcomes (1)

• Change in Quality of Life

  2 years

Study Arms (2)

Active Treatment

EXPERIMENTAL

Patients treated with SBT101

Genetic: SBT101

Imitation Procedure

SHAM COMPARATOR

Procedure that mimics treatment with SBT101, but does not infuse anything into the spinal cord

Procedure: Imitation Procedure

Interventions

SBT101 GENETIC

SBT101 Treatment

Active Treatment

Imitation Procedure PROCEDURE

Procedure that mimics SBT101 infusion, but contains no drug administered

Also known as: Sham Procedure

Imitation Procedure

Eligibility Criteria

• Age: 18 Years - 65 Years
• Gender Eligibility Details: Based on genetic gender
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.
• 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.

Sponsors & Collaborators

• SwanBio Therapeutics, Inc.: lead

Study Sites (2)

University of Massachusetts Chan Medical School

Worcester, Massachusetts, 01655, United States

Location

Amsterdam UMC

Amsterdam, Netherlands

Location

MeSH Terms

Conditions

Adrenoleukodystrophy; Paraplegia; Spastic Paraplegia, Hereditary; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Adrenal Insufficiency

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Paralysis; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Maintain masking to all but those are perform the actual procedure
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 3 Cohorts, each with active treatment and 1 with imitation procedure

Study Record Dates

• First Submitted: May 16, 2022
• First Posted: May 27, 2022
• Study Start: November 17, 2022
• Primary Completion: June 30, 2025
• Study Completion: August 31, 2025
• Last Updated: October 31, 2025

Record last verified: 2025-10

Locations

US (1); NL (1)