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

AMN Clinical Trial

— PROPEL  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05394064
• Other study ID #: SBT101-CT101
• Secondary ID: 2021-004410-19
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: November 17, 2022
• Est. completion date: March 30, 2029

Study information

• Verified date: September 2023
• Source: SwanBio Therapeutics, Inc.
• Contact: Clinical Trial Recruitment
• Phone: 267-417-6356
• Email: clinicaltrials[@]swanbiotx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 16
• Est. completion date: March 30, 2029
• Est. primary completion date: March 30, 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• AMN
• X-ALD

Intervention

Genetic:
• SBT101
SBT101 Treatment

Procedure:
• Imitation Procedure
Procedure that mimics SBT101 infusion, but contains no drug administered

Locations

Country	Name	City	State
Netherlands	Amsterdam UMC	Amsterdam
United States	University of Massachusetts Chan Medical School	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
SwanBio Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change in Quality of Life	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
Primary	Adverse Events to SBT101	Safety and tolerability of SBT101 administration	2 years
Secondary	Disease progression	Characterize disease progression in adults diagnosed with AMN through serial clinical assessments of walking ability measured by 6-Minute Walk Test	2 years