Ignite Creation Date:
2025-12-24 @ 2:47 PM
Ignite Modification Date:
2025-12-26 @ 1:19 AM
Study NCT ID:
NCT05633459
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-08-29
First Post:
2022-11-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'Multiple-ascending doses of QRL-201 or placebo will be administered. The dose levels may change subject to available nonclinical, clinical, safety, and PK data.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Parallel Assignment'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 64}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2022-12-16', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2026-10-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-22', 'studyFirstSubmitDate': '2022-11-21', 'studyFirstSubmitQcDate': '2022-11-21', 'lastUpdatePostDateStruct': {'date': '2025-08-29', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-12-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-10-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of participants with one or more treatment emergent adverse events and serious adverse events', 'timeFrame': 'Baseline through Day 421 [End of Study Visit', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Pharmacokinetics (plasma): Maximum observed concentration of QRL-201 (Cmax)', 'timeFrame': 'Predose up to 24 hours post dose', 'description': 'Endpoints: PK: Cmax of QRL-201'}, {'measure': 'Pharmacokinetics (plasma): Area under the concentration time curve from zero to infinity (AUCinf) of QRL-201', 'timeFrame': 'Predose up to 24 hours post dose', 'description': 'Endpoints: PK: AUC (0-inf) of QRL-201'}, {'measure': 'Pharmacokinetics (plasma): Time of maximum concentration (Tmax) of QRL-201', 'timeFrame': 'Predose up to 24 hours postdose', 'description': 'Endpoints: PK: Tmax of QRL-201'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['ALS, Stathmin-2, STMN2, ASO, Antisense Oligonucleotide'], 'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS', 'detailedDescription': 'This first-in-human, Phase 1 study will evaluate the safety, tolerability, and pharmacokinetics (PK) of QRL-201 administered intrathecal (IT) to participants with Amyotrophic Lateral Sclerosis. Two dose escalation cohorts of 8 participants each, followed by an additional 48 participants, receiving the study drug in a 6:2 ratio of QRL-201 to placebo.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Male or female participants aged 18 to 80 years diagnosed with ALS\n* ALS symptom onset within 24 months of Screening\n* Slow vital capacity \\>50%\n* Clinical or electrodiagnostic evidence of lower motor neuron involvement\n* Not pregnant and not nursing\n* Willing and able to practice effective contraception\n* Able to tolerate lumbar puncture\n* 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)\n\nExclusion Criteria:\n\n* Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes\n* Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device\n* Prior exposure to stem cell or gene therapy products\n* Any contraindication to intrathecal drug administration\n* Abnormal laboratory values deemed clinically significant by the Investigator\n* Significant infection or known inflammatory process\n* Any sign and/or history of neurological conditions and other neuromuscular disorders that could affect the electrophysiological recordings.\n* An EEG that shows signs of abnormal electrical activity (e.g., epilepsy)"}, 'identificationModule': {'nctId': 'NCT05633459', 'briefTitle': 'A Study Evaluating the Safety and Tolerability of QRL-201 in ALS', 'organization': {'class': 'INDUSTRY', 'fullName': 'QurAlis Corporation'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'QRL-201-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'QRL-201: Sporadic ALS', 'description': 'Multiple-ascending doses of QRL-201 will be intrathecally administered to individuals with ALS.', 'interventionNames': ['Drug: Multiple ascending doses of QRL-201']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo: Sporadic ALS', 'description': 'Multiple-ascending doses of placebo comparator will be intrathecally administered to individuals with ALS.', 'interventionNames': ['Drug: Multiple ascending doses of Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'QRL-201: C9orf72-ALS', 'description': 'QRL-201 will be intrathecally administered to individuals with C9orf72-ALS.', 'interventionNames': ['Drug: QRL-201']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo: C9orf72-ALS', 'description': 'Placebo comparator will be intrathecally administered to individuals with C9orf72-ALS.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Multiple ascending doses of QRL-201', 'type': 'DRUG', 'description': 'Multiple ascending doses of QRL-201 will be intrathecally administered to individuals with sporadic ALS.', 'armGroupLabels': ['QRL-201: Sporadic ALS']}, {'name': 'Multiple ascending doses of Placebo', 'type': 'DRUG', 'description': 'Multiple ascending doses of placebo comparator will be intrathecally administered to individuals with sporadic ALS.', 'armGroupLabels': ['Placebo: Sporadic ALS']}, {'name': 'QRL-201', 'type': 'DRUG', 'description': 'QRL-201 will be intrathecally administered to individuals with C9orf72 ALS.', 'armGroupLabels': ['QRL-201: C9orf72-ALS']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo comparator will be intrathecally administered to individuals with C9orf72 ALS.', 'armGroupLabels': ['Placebo: C9orf72-ALS']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'B-3000', 'city': 'Leuven', 'country': 'Belgium', 'facility': 'Universitaire Ziekenhuizen Leuven (UZ Leuven)', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'zip': 'T2N 1N4', 'city': 'Calgary', 'state': 'Alberta', 'country': 'Canada', 'facility': 'University of Calgary', 'geoPoint': {'lat': 51.05011, 'lon': -114.08529}}, {'zip': 'T6G 2G3', 'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': 'University of Alberta', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}, {'zip': 'M4N 3M5', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Sunnybrook Health Science Centre', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'H2L 4M1', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'CHUM - Hopital Notre-Dame', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': 'H3A 2B4', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Montreal Neurological Institute-Hospital', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': '53127', 'city': 'Bonn', 'state': 'North Rhine-Westphalia', 'country': 'Germany', 'facility': 'Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE)', 'geoPoint': {'lat': 50.73438, 'lon': 7.09549}}, {'zip': '10117', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charité Research Organisation', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Lübeck', 'country': 'Germany', 'facility': 'University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology', 'geoPoint': {'lat': 53.86893, 'lon': 10.68729}}, {'zip': '89081', 'city': 'Ulm', 'country': 'Germany', 'facility': 'Universitätsklinikum Ulm', 'geoPoint': {'lat': 48.39841, 'lon': 9.99155}}, {'zip': 'D08 A978', 'city': 'Dublin', 'country': 'Ireland', 'facility': "St James's Hospital", 'geoPoint': {'lat': 53.33306, 'lon': -6.24889}}, {'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Universitair Medisch Centrum Utrecht', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'zip': 'S10 2JF', 'city': 'Sheffield', 'state': 'United Kingdom', 'country': 'United Kingdom', 'facility': 'The University of Sheffield, Royal Hallamshire Hospital', 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}, {'zip': 'SE5 9RS', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Kings College Hospital NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'WC1N 3BG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'National Hospital for Neurology and Neurosurgery', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Angela Genge, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'QurAlis Corporation'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'QurAlis Corporation', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}