Amyotrophic Lateral Sclerosis Clinical Trial
Official title:
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
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
| Status | Recruiting |
| Enrollment | 64 |
| Est. completion date | May 6, 2025 |
| Est. primary completion date | May 6, 2025 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 80 Years |
| Eligibility | 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 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 |
| Country | Name | City | State |
|---|---|---|---|
| Belgium | Universitaire Ziekenhuizen Leuven (UZ Leuven) | Leuven | |
| Canada | University of Calgary | Calgary | Alberta |
| Canada | University of Alberta | Edmonton | Alberta |
| Canada | CHUM - Hopital Notre-Dame | Montréal | Quebec |
| Canada | Montreal Neurological Institute-Hospital | Montréal | Quebec |
| Germany | Charité Research Organisation | Berlin | |
| Germany | University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology | Lübeck | |
| Germany | Universitätsklinikum Ulm | Ulm | |
| Ireland | St James's Hospital | Dublin | |
| Netherlands | Universitair Medisch Centrum Utrecht | Utrecht | |
| United Kingdom | The University of Sheffield, Royal Hallamshire Hospital | Sheffield |
| Lead Sponsor | Collaborator |
|---|---|
| QurAlis Corporation |
Belgium, Canada, Germany, Ireland, Netherlands, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of participants with one or more treatment emergent adverse events and serious adverse events | Endpoints: A summary of treatment emergent adverse events, serious adverse events, and other non-serious adverse events, regardless of causality, will be reported in the Reported Adverse Events module. | Baseline through 253 Days | |
| Secondary | Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax) | Endpoints: PK: Cmax of QRL-201 | Predose up to 24 hours postdose | |
| Secondary | Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201 | Endpoints: PK: AUC (0-inf) of QRL-201 | Predose up to 24 hours postdose | |
| Secondary | Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201 | Endpoints: PK: Tmax of QRL-201 | Predose up to 24 hours postdose |
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