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

Primary Purpose

AMN, AMN Gene Mutation, X-ALD

Status

Recruiting

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

SBT101

Imitation Procedure

About this trial

This is an interventional treatment trial for AMN focused on measuring 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

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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.

Sites / Locations

University of Massachusetts Chan Medical SchoolRecruiting
Amsterdam UMC

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Sham Comparator

Arm Label

Active Treatment

Imitation Procedure

Arm Description

Patients treated with SBT101

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

Outcomes

Primary Outcome Measures

Adverse Events to SBT101

Safety and tolerability of SBT101 administration

Secondary Outcome Measures

Disease progression

Characterize disease progression in adults diagnosed with AMN through serial clinical assessments of walking ability measured by 6-Minute Walk Test

Full Information

NCT ID

NCT05394064

First Posted

May 16, 2022

Last Updated

September 11, 2023

Sponsor

SwanBio Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05394064

Brief Title

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

Acronym

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Recruiting

Study Start Date

November 17, 2022 (Actual)

Primary Completion Date

March 30, 2026 (Anticipated)

Study Completion Date

March 30, 2029 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

SwanBio Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Model Description

3 Cohorts, each with active treatment and 1 with imitation procedure

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Masking Description

Maintain masking to all but those are perform the actual procedure

Allocation

Randomized

Enrollment

16 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Active Treatment

Arm Type

Experimental

Arm Description

Patients treated with SBT101

Arm Title

Imitation Procedure

Arm Type

Sham Comparator

Arm Description

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

Intervention Type

Genetic

Intervention Name(s)

SBT101

Intervention Description

SBT101 Treatment

Intervention Type

Procedure

Intervention Name(s)

Imitation Procedure

Other Intervention Name(s)

Sham Procedure

Intervention Description

Procedure that mimics SBT101 infusion, but contains no drug administered

Primary Outcome Measure Information:

Title

Adverse Events to SBT101

Description

Safety and tolerability of SBT101 administration

Time Frame

2 years

Secondary Outcome Measure Information:

Title

Disease progression

Description

Characterize disease progression in adults diagnosed with AMN through serial clinical assessments of walking ability measured by 6-Minute Walk Test

Time Frame

2 years

Other Pre-specified Outcome Measures:

Title

Change in Quality of Life

Description

Characterize the change in multiple quality of life parameters over time using questionnaires including the Multiple Sclerosis Quality of Life-54 (MS-QOL)

Time Frame

2 years

10. Eligibility

Sex

Male

Gender Based

Yes

Gender Eligibility Description

Based on genetic gender

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Clinical Trial Recruitment

Phone

267-417-6356

clinicaltrials@swanbiotx.com

Facility Information:

Facility Name

University of Massachusetts Chan Medical School

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

Individual Site Status

Recruiting

Facility Name

Amsterdam UMC

City

Amsterdam

Country

Netherlands

Individual Site Status

Not yet recruiting

12. IPD Sharing Statement

Learn more about this trial

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

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