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Gene Therapy

SBT101 Gene Therapy for Adrenomyeloneuropathy (PROPEL Trial)

Phase 1 & 2
Recruiting
Research Sponsored by SwanBio Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosed with X-linked adrenoleukodystrophy (ALD), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by elevated circulating VLCFA levels.
Be older than 18 years old
Must not have
Evidence of or past diagnosis of inflammatory cerebral disease.
Patients who have received a gene therapy.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years

Summary

This trial is evaluating a gene therapy to treat adrenomyeloneuropathy, a hereditary nervous system disorder. Patients will receive a single dose of the treatment or a fake treatment, and be closely monitored for safety and efficacy for 2 years.

Who is the study for?
Adults aged 18-65 with adrenomyeloneuropathy (AMN), able to walk, diagnosed with X-linked adrenoleukodystrophy (ALD) confirmed by genetic testing and high VLCFA levels. Not eligible if they have unstable adrenal function, prior gene therapy, MRI or anesthesia contraindications, diabetes/high blood sugar, inflammatory brain disease history, or over 15 years since myeloneuropathy onset.
What is being tested?
The trial is testing SBT101 gene therapy for AMN via a one-time spinal injection compared to an imitation procedure. It's a blind study where patients are monitored for safety and effectiveness over two years; those on SBT101 continue follow-up for three more years.
What are the potential side effects?
Potential side effects aren't specified but may include reactions related to the intrathecal route of administration such as headache or back pain, immune responses to the viral vector or inserted gene product, and typical risks associated with long-term monitoring like infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with X-linked adrenoleukodystrophy (ALD) confirmed by genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had inflammation in my brain.
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I have undergone gene therapy.
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I have diabetes or my blood sugar/A1C levels are high.
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I cannot take certain medications like steroids or anesthetics due to health reasons.
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My adrenal gland function is stable.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Adverse Events to SBT101
Secondary study objectives
Disease progression
Other study objectives
Change in Quality of Life

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Active TreatmentExperimental Treatment1 Intervention
Patients treated with SBT101
Group II: Imitation ProcedurePlacebo Group1 Intervention
Procedure that mimics treatment with SBT101, but does not infuse anything into the spinal cord

Find a Location

Who is running the clinical trial?

SwanBio Therapeutics, Inc.Lead Sponsor
1 Previous Clinical Trials
65 Total Patients Enrolled

Media Library

SBT101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05394064 — Phase 1 & 2
Adrenoleukodystrophy Research Study Groups: Imitation Procedure, Active Treatment
Adrenoleukodystrophy Clinical Trial 2023: SBT101 Highlights & Side Effects. Trial Name: NCT05394064 — Phase 1 & 2
SBT101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05394064 — Phase 1 & 2
~6 spots leftby Mar 2026
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