SBT101 Gene Therapy for Adrenomyeloneuropathy
(PROPEL Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, if you have contraindications (reasons not to use) for steroids, sirolimus, tacrolimus, or anesthetic medications, you may not be eligible to participate.
How does the SBT101 treatment for Adrenomyeloneuropathy differ from other treatments?
SBT101 is a gene therapy that offers a novel approach by potentially targeting the underlying genetic cause of Adrenomyeloneuropathy, unlike traditional treatments that may only address symptoms. This therapy involves delivering a specific gene to affected cells, which could lead to more effective and long-lasting results.12345
What data supports the effectiveness of the SBT101 treatment for Adrenomyeloneuropathy?
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose-Escalation
Two doses of SBT101 will be evaluated to establish the maximum tolerated dose
Dose-Expansion
Additional patients will be enrolled to receive SBT101 at the maximum tolerated dose
Long-term Safety Follow-up
An unblinded 3-year follow-up period to evaluate the safety of SBT101 and disease progression
What Are the Treatments Tested in This Trial?
Interventions
- Imitation Procedure
- SBT101
Find a Clinic Near You
Who Is Running the Clinical Trial?
SwanBio Therapeutics, Inc.
Lead Sponsor