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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

PROPEL Trial 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.Check my eligibility

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.See study design

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.

PROPEL Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with X-linked adrenoleukodystrophy (ALD) confirmed by genetic testing.

PROPEL Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Adverse Events to SBT101

Secondary outcome measures

Disease progression

Other outcome measures

Change in Quality of Life

PROPEL 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

0 / 1Eligibility criteria met

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What criteria must be met to qualify for participation in this scientific experiment?

"This medical study requires 16 participants within the mandated age bracket of 18-65 who are diagnosed with X-linked adrenoleukodystrophy (ALD) and have a concomitant proven mutation in the ABCD1 gene, based on confirmatory genetic testing which is further supported by elevated circulating VLCFA levels. Additionally, patients must demonstrate clinical evidence of spinal cord involvement but be able to ambulate independently."

Answered by AI

Are there still opportunities for individuals to become involved in this research?

"Clinicaltrials.gov confirms that this experiment is still recruiting participants, having first been advertised on June 30th 2022 and most recently updated the following month."

Answered by AI

What is the aggregate figure of individuals participating in this research?

"Yes, the most recent changes to clinicaltrials.gov suggest that this medical experiment has recommenced its search for test subjects. First posted on June 30th 2022 and last updated a month later, the study requires 16 participants from 1 location."

Answered by AI

Is this research study open to participants younger than 30 years of age?

"This trial is specifically for individuals aged 18-65. In addition, there are 8 clinical trials tailored to minors and 7 aimed at the elderly population."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2022. "A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05394064.

All > Adrenoleukodystrophy > Neuropathy