A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
Amyotrophic Lateral Sclerosis
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide
Eligibility 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 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
Sites / Locations
- Universitaire Ziekenhuizen Leuven (UZ Leuven)
- University of CalgaryRecruiting
- University of AlbertaRecruiting
- CHUM - Hopital Notre-DameRecruiting
- Montreal Neurological Institute-HospitalRecruiting
- University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision NeurologyRecruiting
- Universitätsklinikum Ulm
- St James's HospitalRecruiting
- Universitair Medisch Centrum UtrechtRecruiting
- The University of Sheffield, Royal Hallamshire Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm 8
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
QRL-201 - Arm 1
QRL-201 - Arm 2
QRL-201 - Arm 3
QRL-201 - Arm 4
QRL-201 - Arm 5
QRL-201 - Arm 6
QRL-201 - Arm 7
QRL-201 - Arm 8
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201
Placebo Comparator: Placebo consists of the same components as the formulation buffer for QRL-201