Evaluating QRL-201 for safety in ALS patients
A Multi-Center, Randomized, Double-Blind Placebo Controlled Multiple-Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis
This study is testing a new drug called QRL-201 to see if it's safe for people with ALS.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 64 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | QurAlis Corporation Industry-sponsored |
| Locations | 14 sites (Leuven and 13 other locations) |
| Trial ID | NCT05633459 on ClinicalTrials.gov |
What this trial studies
This Phase 1 study aims to assess the safety and tolerability of QRL-201, an investigational drug, in individuals diagnosed with Amyotrophic Lateral Sclerosis (ALS). Participants will receive multiple ascending doses of QRL-201 administered intrathecally, with a portion receiving a placebo for comparison. The study will involve two dose escalation cohorts of 8 participants each, followed by an additional 48 participants, to gather data on the drug's pharmacokinetics and overall safety profile.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 to 80 diagnosed with ALS within the last 24 months and with a slow vital capacity greater than 50%.
Not a fit: Patients with specific genetic variants related to ALS or those currently participating in other clinical studies may not benefit from this trial.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option that improves the safety and management of ALS.
How similar studies have performed: While this approach is novel, previous studies involving antisense oligonucleotides have shown promise in treating other neurological conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male or female participants aged 18 to 80 years diagnosed with ALS * ALS symptom onset within 24 months of Screening * Slow vital capacity \>50% * Clinical or electrodiagnostic evidence of lower motor neuron involvement * Not pregnant and not nursing * Willing and able to practice effective contraception * Able to tolerate lumbar puncture * If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion) Exclusion Criteria: * Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes * Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device * Prior exposure to stem cell or gene therapy products * Any contraindication to intrathecal drug administration * Abnormal laboratory values deemed clinically significant by the Investigator * Significant infection or known inflammatory process * Any sign and/or history of neurological conditions and other neuromuscular disorders that could affect the electrophysiological recordings. * An EEG that shows signs of abnormal electrical activity (e.g., epilepsy)
Where this trial is running
Leuven and 13 other locations
- Universitaire Ziekenhuizen Leuven (UZ Leuven) — Leuven, Belgium (Recruiting)
- University of Calgary — Calgary, Alberta, Canada (Recruiting)
- University of Alberta — Edmonton, Alberta, Canada (Recruiting)
- Sunnybrook Health Science Centre — Toronto, Ontario, Canada (Recruiting)
- CHUM - Hopital Notre-Dame — Montréal, Quebec, Canada (Recruiting)
- Montreal Neurological Institute-Hospital — Montréal, Quebec, Canada (Recruiting)
- Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE) — Bonn, North Rhine-Westphalia, Germany (Recruiting)
- Charité Research Organisation — Berlin, Germany (Recruiting)
- University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology — Lübeck, Germany (Recruiting)
- Universitätsklinikum Ulm — Ulm, Germany (Recruiting)
- St James's Hospital — Dublin, Ireland (Recruiting)
- Universitair Medisch Centrum Utrecht — Utrecht, Netherlands (Recruiting)
- Kings College Hospital NHS Foundation Trust — London, United Kingdom (Recruiting)
- The University of Sheffield, Royal Hallamshire Hospital — Sheffield, United Kingdom (Recruiting)
Study contacts
- Study coordinator: QurAlis Corporation
- Email: clinicaltrials@quralis.com
- Phone: 617-720-9566
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.