nctId: NCT06297486 id: SPK-8011-302 id: 2023-504537-46 type: EUDRACT_NUMBER briefTitle: Study of a Gene Therapy Treatment for Hemophilia A acronym: KEYSTONE 1 overallStatus: RECRUITING date: 2024-03-13 date: 2027-05 date: 2035-09-04 date: 2024-03-07 date: 2024-04-18 name: Spark Therapeutics, Inc. class: INDUSTRY briefSummary: The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis. conditions: Hemophilia A studyType: INTERVENTIONAL phases: PHASE3 allocation: NON_RANDOMIZED interventionModel: PARALLEL interventionModelDescription: Participants will be assigned into one of three cohorts, each receiving the same study treatment. primaryPurpose: TREATMENT masking: NONE count: 85 type: ESTIMATED name: SPK-8011 measure: Annualized Bleed Rate (ABR) for All Bleeds [Cohort A] measure: Median FVIII: C levels [Cohort A] measure: ABR for treated bleeds [Cohort A] measure: Percentage of Participants with ABR=0 for all bleeds; treated bleeds; treated spontaneous bleeds; and treated joint and target joint bleeds [Cohort A] measure: ABR for treated spontaneous, joint, and target joint bleeds [Cohort A] measure: Annualized FVIII dosage [Cohort A] measure: Proportion of Resolved Target Joints [Cohort A] measure: Mean Change of Total Hemophilia Joint Health Score [Cohort A] measure: FVIII:C levels over time [Cohort A] measure: Mean ABR for all bleeds and treated bleeds [Cohort C] measure: FVIII: C levels over time from Week 26 [Cohort C] measure: Proportion of Participants Who Receive IV Methylprednisolone (IVMP) Prior to Week 8 (Early IVMP) [Cohorts A, B, C] measure: Proportion of Participants Who Receive Secondary Oral Immunomodulation [Cohorts A, B, C] measure: Median Time to First Immunomodulation-related Corticosteroid Dose [Cohorts A, B, C] measure: Median Duration of Secondary Oral Immunomodulation [Cohorts A, B, C] measure: Proportion of Participants with FVIII Inhibitor Development [Cohorts A, B, C] measure: Proportion of participants with Treatment-related AEs of ALT Elevation [Cohorts A, B, C] measure: Proportion of participants with treatment-related AEs of Infusion Reaction [Cohorts A, B, C] measure: Number of participants with abnormal physical exam findings, abnormal vital signs, and abnormal selected clinical laboratory test results [Cohorts A, B, C] measure: Proportion of participants with AEs and SAEs [Cohorts A, B, C] measure: Proportion of participants with AESIs [Cohorts A, B, C] sex: MALE minimumAge: 18 Years stdAges: ADULT stdAges: OLDER_ADULT facility: Orthopaedic Institute for Children/Orthopaedic Hemophilia Treatment Center status: RECRUITING city: Los Angeles state: California zip: 90007 country: United States name: Doris Quon, MD, PhD role: CONTACT lat: 34.05223 lon: -118.24368 facility: Kaiser Permanente-Oakland Medical Center status: RECRUITING city: Oakland state: California zip: 94611 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 37.80437 lon: -122.2708 facility: Kaiser Permanente-Roseville Medical Center status: RECRUITING city: Roseville state: California zip: 95661 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 38.75212 lon: -121.28801 facility: Kaiser Permanente -Sacramento Medical Center status: RECRUITING city: Sacramento state: California zip: 95814 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 38.58157 lon: -121.4944 facility: Kaiser Permanente -San Francisco Medical Center status: RECRUITING city: San Francisco state: California zip: 94115 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 37.77493 lon: -122.41942 facility: University of California - San Francisco status: RECRUITING city: San Francisco state: California zip: 94143 country: United States name: Andrew Leavitt, MD role: CONTACT lat: 37.77493 lon: -122.41942 facility: Kaiser Permanente- Santa Clara Medical Center status: RECRUITING city: Santa Clara state: California zip: 94115 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 37.35411 lon: -121.95524 facility: Kaiser Permanente-Vallejo Medical Center status: RECRUITING city: Vallejo state: California zip: 94589 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 38.10409 lon: -122.25664 facility: Kaiser Permanente -Walnut Creek Medical Center status: RECRUITING city: Walnut Creek state: California zip: 94596 country: United States name: Tatjana Kolevska, MD role: CONTACT lat: 37.90631 lon: -122.06496 hasResults: False