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

NCT05394064 | Terminated Phase 1 Results DMC FDA Drug

• 2 other identifiers: SBT101-CT1012021-004410-19
• Study Type: interventional
• Target: 9
• 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

X-ALD; AMN; AMN Gene Mutation

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. Any Serious TEAE. Any Serious TEAE Related to the Study Procedure or Study Drug.

  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.

  2 years

Study Arms (2)

Active Treatment - Cohort 1- Dose Level 1

EXPERIMENTAL

Patients treated with SBT101 (1.0E14 vector genomes)

Genetic: SBT101 Treatment

Active Treatment - Cohort 2 - Dose Level 2

EXPERIMENTAL

Patients treated with SBT101 (3.0E14 vector genomes).

Genetic: SBT101 Treatment

Interventions

SBT101 Treatment GENETIC

SBT101 Treatment

Active Treatment - Cohort 1- Dose Level 1; Active Treatment - Cohort 2 - Dose Level 2

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

Limitations and Caveats

Early termination leading to small numbers of subjects analyzed.

Results Point of Contact

• Title: Spur Clinical Trials Contact
• Organization: Spur Therapeutics Limited

clinicaltrials@spurtherapeutics.com

+441438906870

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

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: 2 Cohorts, each with active treatment

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: August 26, 2026
• Results First Posted: August 26, 2026

Record last verified: 2026-08

Locations

US (1); NL (1)