AMNPhase 1/2PROPELNCT05394064
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
A Phase 1/2 study testing SBT101 Treatment in people with AMN and AMN Gene Mutation, run by SwanBio Therapeutics, Inc. at 2 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- AMN, AMN Gene Mutation, X-ALD
- What is being tested?
- SBT101 Treatment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 17 Nov 2022to 31 Aug 2025
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655
About this study
Official 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
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.
Read the detailed description
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.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Being tested
- SBT101 Treatment
- 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
- Medical subject headings
- Adrenoleukodystrophy, Paraplegia, Spastic Paraplegia, Hereditary, Brain Diseases, Metabolic, Inborn, Brain Diseases, Metabolic, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Demyelinating Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SBT101 TreatmentGenetic
SBT101 Treatment
| Study group | Role | What they receive |
|---|---|---|
| Active Treatment - Cohort 1- Dose Level 1 | Experimental | Patients treated with SBT101 (1.0E14 vector genomes) Receives: Genetic: SBT101 Treatment |
| Active Treatment - Cohort 2 - Dose Level 2 | Experimental | Patients treated with SBT101 (3.0E14 vector genomes). Receives: Genetic: SBT101 Treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 May 2022
- Study start17 Nov 2022
- Primary completion30 Jun 2025
- Study completion31 Aug 2025
Why it stopped: The SBT101 program for AMN was terminated for business/strategic reasons, and there were no safety concerns
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AMNStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 2 Cohorts, each with active treatment
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05394064
- Sponsor's protocol ID
- SBT101-CT101
- Other IDs
- 2021-004410-19
- Acronym
- PROPEL
- Status last verified
- Aug 2026
- First submitted
- 16 May 2022
- Last updated
- 26 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No