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

August 4, 2026 updated by: SwanBio Therapeutics, Inc.

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Amsterdam, Netherlands
        • Amsterdam UMC
  • United States
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Chan Medical School

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosed with X-linked adrenoleukodystrophy (ALD), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by elevated circulating VLCFA levels.
  2. Clinical evidence of spinal cord involvement but still able to ambulate independently

Exclusion Criteria:

  1. Evidence of or past diagnosis of inflammatory cerebral disease.
  2. 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.
  3. Contraindications for MRI procedure and/or contrast materials.
  4. Contraindication to steroids, sirolimus, tacrolimus, and/or anesthetic medications.
  5. Unstable adrenal function (e.g., untreated or inappropriately treated adrenal insufficiency).
  6. History of diabetes or abnormal fasting plasma glucose (≥126 mg/dL) or hemoglobin A1C ≥6.5%.
  7. Patients who have received a gene therapy.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Active Treatment - Cohort 1- Dose Level 1
Patients treated with SBT101 (1.0E14 vector genomes)
Genetic: SBT101 Treatment
SBT101 Treatment
Experimental: Active Treatment - Cohort 2 - Dose Level 2
Patients treated with SBT101 (3.0E14 vector genomes).
Genetic: SBT101 Treatment
SBT101 Treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
2 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Disease progression
Time Frame: 2 years
Characterize disease progression in adults diagnosed with AMN through serial clinical assessments of walking ability measured by 6-Minute Walk Test
2 years

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Quality of Life
Time Frame: 2 years
Characterize the change in multiple quality of life parameters over time using questionnaires including the Multiple Sclerosis Quality of Life-54 (MS-QOL)
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

SwanBio Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 17, 2022

Primary Completion (Actual)

June 30, 2025

Study Completion (Actual)

August 31, 2025

Study Registration Dates

First Submitted

May 16, 2022

First Submitted That Met QC Criteria

May 23, 2022

First Posted (Actual)

May 27, 2022

Study Record Updates

Last Update Posted (Actual)

August 26, 2026

Last Update Submitted That Met QC Criteria

August 4, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SBT101-CT101
  • 2021-004410-19 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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