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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05394064
Recruitment Status : Recruiting
First Posted : May 27, 2022
Last Update Posted : June 2, 2022
Sponsor:
Information provided by (Responsible Party):
SwanBio Therapeutics, Inc.

Study Description
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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.


Condition or disease Intervention/treatment Phase
AMN AMN Gene Mutation X-ALD Genetic: SBT101 Procedure: Imitation Procedure Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: 3 Cohorts, each with active treatment and imitation procedure
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Maintain masking to all but those are perform the actual procedure
Primary Purpose: Treatment
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
Estimated Study Start Date : June 30, 2022
Estimated Primary Completion Date : March 30, 2026
Estimated Study Completion Date : March 30, 2029

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Active Treatment
Patients treated with SBT101
 Genetic: SBT101
SBT101 Treatment
Sham Comparator: Imitation Procedure
Procedure that mimics treatment with SBT101, but does not infuse anything into the spinal cord
 Procedure: Imitation Procedure
Procedure that mimics SBT101 infusion, but contains no drug administered
Other Name: Sham Procedure


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events to SBT101 [ Time Frame: 2 years ]
    Safety and tolerability of SBT101 administration


Secondary Outcome Measures :
  1. 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


Other Outcome Measures:
  1. 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)


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Based on genetic gender
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05394064


Contacts
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Contact: Clinical Trial Recruitment 267-417-6356 clinicaltrials@swanbiotx.com

Locations
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United States, Massachusetts
University of Massachusetts Chan Medical School Recruiting
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
SwanBio Therapeutics, Inc.
More Information
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Responsible Party: SwanBio Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05394064    
Other Study ID Numbers: SBT101-CT101
2021-004410-19 ( EudraCT Number )
First Posted: May 27, 2022    Key Record Dates
Last Update Posted: June 2, 2022
Last Verified: June 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by SwanBio Therapeutics, Inc.:
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
Additional relevant MeSH terms:
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Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases