Gene therapy for SOD1 Amyotrophic Lateral Sclerosis
A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).
This study is testing a new gene therapy for adults with SOD1-related ALS to see if it can safely reduce the effects of the harmful gene.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | UniQure Biopharma B.V. Industry-sponsored |
| Locations | 10 sites (Irvine, California and 9 other locations) |
| Trial ID | NCT06100276 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety, tolerability, and exploratory efficacy of AMT-162, an investigational gene therapy administered intrathecally to adults with SOD1-mediated Amyotrophic Lateral Sclerosis (ALS). The therapy aims to silence the expression of the mutant SOD1 gene, which is implicated in the progression of ALS. Participants will receive a single ascending dose of AMT-162, and the study will monitor their response and any adverse effects. The trial is designed to gather data that could support further development of this gene therapy approach.
Who should consider this trial
Good fit: Ideal candidates are adults with a confirmed diagnosis of SOD1-mediated ALS who exhibit signs of lower motor neuron dysfunction and meet specific health criteria.
Not a fit: Patients with certain SOD1 variants or those who have previously received specific SOD1 suppression therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could slow the progression of ALS in patients with SOD1 mutations.
How similar studies have performed: While gene therapy approaches for ALS are still emerging, this specific method targeting SOD1 has not been widely tested, making it a novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity). * ALSFRS-R score ≥ 25 at Screening. * Slow vital capacity (SVC) ≥50% of predicted normal value. * Capable of providing informed consent and complying with trial procedures, including: medically able to undergo lumbar puncture and has a responsible caregiver able to attend all clinic visit with the Participant. Exclusion Criteria: * SOD1 pathogenic or likely pathogenic variants in amino acid regions 43-47. * Pathogenic repeat expansion in the C9orf72 gene * Any of the following prior or concomitant treatments: * Any prior SOD1 suppression therapy with viral microRNA mediators * Prior SOD suppression therapy with antisense oligonucleotide (ASO) mediators such as tofersen (QALSODY™). Exception: Patients who previously received tofersen may be enrolled if the last dose of tofersen was received at least 20 weeks prior to the first Screening assessment and if there were no previous tofersen-related SAEs or ongoing tofersen-related adverse events that would increase the risk of receiving AMT-162, per Investigator judgment. * Other ALS medications riluzole (RILUTEK®, TIGLUTIK®), edaravone (RADICAVA®), and sodium phenylbutyrate and taururosdiol combination (RELYVRIO) or bioequivalents are allowed if dose is stable for 30 days prior to immunosuppression. * Any prior administration of an AAV gene therapy. * Participants must be willing to forego new ALS treatments through at least 6 months after infusion of AMT-162. After 6 months, Investigators and participants may decide to add new ALS medications or change existing ALS medications.
Where this trial is running
Irvine, California and 9 other locations
- University of California Irvine — Irvine, California, United States (Recruiting)
- California Pacific Medical Center — San Francisco, California, United States (Recruiting)
- Mayo Clinic Florida — Jacksonville, Florida, United States (Not_yet_recruiting)
- Winship Cancer Institute of Emory University — Atlanta, Georgia, United States (Not_yet_recruiting)
- Northwestern University Feinberg School of Medicine — Chicago, Illinois, United States (Not_yet_recruiting)
- University of Kansas Medical Center — Fairway, Kansas, United States (Not_yet_recruiting)
- Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research — Boston, Massachusetts, United States (Recruiting)
- Mayo Clinic Rochester — Rochester, Minnesota, United States (Not_yet_recruiting)
- Columbia University Irving Medical Center — New York, New York, United States (Recruiting)
- University of Pennsylvania School of Medicine — Philadelphia, Pennsylvania, United States (Not_yet_recruiting)
Study contacts
- Study coordinator: Director Clinical Operations
- Email: medinfo@uniqure.com
- Phone: 1-866-520-1257
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.