protocolSection identificationModule nctId: NCT06351592, orgStudyIdInfo id: ALN-SOD-ALS-2351, secondaryIdInfos id: 2023-510344-20-00, type: REGISTRY, domain: EU CT Number, briefTitle: First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS), statusModule overallStatus: NOT_YET_RECRUITING, startDateStruct date: 2024-06-28, primaryCompletionDateStruct date: 2029-04-28, completionDateStruct date: 2029-04-28, studyFirstPostDateStruct date: 2024-04-08, lastUpdatePostDateStruct date: 2024-04-08, sponsorCollaboratorsModule leadSponsor name: Regeneron Pharmaceuticals, class: INDUSTRY, descriptionModule briefSummary: This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with amyotrophic lateral sclerosis (ALS) who have a mutation in a gene called the superoxide dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is.The study is looking at several other research questions, including:* The effect the study drug has on specific biomarkers, which are molecules in the blood or in the fluid that surrounds the brain and spinal cord, known as cerebrospinal fluid (CSF)* How much study drug is in the blood and in the CSF, at different times* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)* What effects the study drug has on ALS symptoms, conditionsModule conditions: Amyotrophic Lateral Sclerosis (ALS), conditions: Mutation in the Superoxide Dismutase-1 (SOD1) Gene, designModule studyType: INTERVENTIONAL, phases: PHASE1, designInfo allocation: RANDOMIZED, interventionModel: SEQUENTIAL, primaryPurpose: TREATMENT, maskingInfo masking: QUADRUPLE, maskingDescription: No masking during Open-Label Treatment Period after 4-week Double-Blind Treatment Period. This part of the study consists of a crossover design., whoMasked: PARTICIPANT, whoMasked: CARE_PROVIDER, whoMasked: INVESTIGATOR, whoMasked: OUTCOMES_ASSESSOR, enrollmentInfo count: 42, type: ESTIMATED, armsInterventionsModule interventions name: ALN-SOD, interventions name: Diluent, interventions name: Placebo (PB), outcomesModule primaryOutcomes measure: Incidence of treatment-emergent adverse event (TEAEs) in participants treated with ALN-SOD, primaryOutcomes measure: Severity of TEAEs in participants treated with ALN-SOD, secondaryOutcomes measure: Concentration of SOD1 protein in cerebrospinal fluid (CSF) over time, secondaryOutcomes measure: Change in concentration of SOD1 protein in CSF over time, secondaryOutcomes measure: Concentration of neurofilament light chain (NfL) in plasma over time, secondaryOutcomes measure: Change in concentration of NfL in plasma over time, secondaryOutcomes measure: Concentration of NfL in CSF over time, secondaryOutcomes measure: Change in concentration of NfL in CSF over time, secondaryOutcomes measure: Concentration of ALN-SOD in plasma over time, secondaryOutcomes measure: Concentration of ALN-SOD in CSF over time, secondaryOutcomes measure: Incidence of anti-drug antibodies (ADAs) to ALN-SOD in serum over time, secondaryOutcomes measure: Titer of ADAs to ALN-SOD in serum over time, eligibilityModule sex: ALL, minimumAge: 18 Years, stdAges: ADULT, stdAges: OLDER_ADULT, contactsLocationsModule, hasResults: False