Ignite Creation Date:
2025-12-25 @ 1:46 AM
Ignite Modification Date:
2026-01-06 @ 9:41 PM
Study NCT ID:
NCT06556394
Status:
RECRUITING
Last Update Posted:
2025-01-08
First Post:
2024-08-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation
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']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 32}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-12-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2026-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-06', 'studyFirstSubmitDate': '2024-08-01', 'studyFirstSubmitQcDate': '2024-08-13', 'lastUpdatePostDateStruct': {'date': '2025-01-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-08-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-02', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Adverse Events(AEs)', 'timeFrame': 'Before treatment and within 57 days after treatment', 'description': 'Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)'}], 'secondaryOutcomes': [{'measure': 'Plasma Pharmacokinetic (PK) Parameter: AUC0-last', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Area Under the Plasma Concentration-Time Curve from Time 0 to the Last Measurable Non-zero Concentration'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: AUC0-inf', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Area Under the Plasma Concentration-Time Curve from Time 0 Extrapolated to Infinity'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: Cmax', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Peak Plasma Concentration'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: Tmax', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Time to reach Cmax. If the maximum value occurs at more than one time point, Tmax is defined as the first time point with this value'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: λz', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Elimination Rate Constant'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: T½', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Apparent Terminal Elimination Half-life of Study Drug'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: CL/F', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Apparent Clearance'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: Vz/F', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Apparent Volume of Distribution'}, {'measure': 'Plasma Pharmacokinetic (PK) Parameter: MRT', 'timeFrame': 'Before treatment and within 48 hours after treatment', 'description': 'Mean Residence Time'}, {'measure': 'CSF Pharmacokinetic (PK) Parameter: Concentration', 'timeFrame': 'Before treatment and within 29 days after treatment', 'description': 'Concentration in Cerebrospinal Fluid(CSF)'}, {'measure': 'CSF Pharmacokinetic (PK) Parameter: T½', 'timeFrame': 'Before treatment and within 29 days after treatment', 'description': 'Half-life in Cerebrospinal Fluid(CSF)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'This is a Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation', 'detailedDescription': 'The study is a phase 1, randomized, double-blind, placebo controlled study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RAG-17 in patients with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) gene mutation. The dose levels will be evaluated sequentially across separate cohorts using a rules-based design, wherein participants will receive RAG-17 or placebo at a ratio of 3:1.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.\n2. ≥ 18 years of age at the time of informed consent.\n3. Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.\n4. Documented SOD1 mutation.\n5. Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).\n6. If taking riluzole or edaravone, subject must be on a stable dose or ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.\n\nExclusion Criteria:\n\n1. Documented p.F21C SOD1 mutation.\n2. Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.\n3. Current enrollment in any other interventional study.\n4. History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.\n5. Pregnant or currently breastfeeding.'}, 'identificationModule': {'nctId': 'NCT06556394', 'briefTitle': 'A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation', 'organization': {'class': 'OTHER', 'fullName': 'Ractigen Therapeutics.'}, 'officialTitle': 'A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability,Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation', 'orgStudyIdInfo': {'id': 'RGN17-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RAG-17', 'description': 'RAG-17 is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation', 'interventionNames': ['Drug: RAG-17']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'RAG-17', 'type': 'DRUG', 'description': 'RAG-17 is a therapeutic small interfering RNA (siRNA).', 'armGroupLabels': ['RAG-17']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo will be administered via intrathecal injection', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Beijing', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Yilong Wang, PhD&MD', 'role': 'CONTACT', 'email': 'yilong528@gmail.com'}], 'facility': 'Beijing Tiantan Hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}, {'city': 'Chengdu', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Huifang Shang, PhD&MD', 'role': 'CONTACT', 'email': 'hfshang2002@163.com'}], 'facility': 'West China Hospital of Sichuan University', 'geoPoint': {'lat': 30.66667, 'lon': 104.06667}}, {'city': 'Hangzhou', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Zhiying Wu, PhD&MD', 'role': 'CONTACT', 'email': 'zhiyingwu@zju.edu.cn'}], 'facility': 'The Second Affiliated Hospital Zhejiang University School of Medicine', 'geoPoint': {'lat': 30.29365, 'lon': 120.16142}}], 'centralContacts': [{'name': 'Long-Cheng Li', 'role': 'CONTACT', 'email': 'lilc@ractigen.com', 'phone': '+86 18051622388'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ractigen Therapeutics.', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}