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Conditions: Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII (FVIII), Factor VIII (FVIII) Deficiency, Factor VIII (FVIII) Gene, Factor VIII (FVIII) Protein, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Gene Therapy, Gene Transfer, Hematologic Diseases, Hemorrhagic Disorders, Recombinant, Vector, Inhibitors
Interventions: SPK-8016; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 11
States / cities: Los Angeles, California • Peoria, Illinois • Ann Arbor, Michigan + 7 more

Conditions: Hemophilia A
Interventions: SPK-8011; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 11
States / cities: Sacramento, California • Gainesville, Florida • Boston, Massachusetts + 7 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Liver Biopsy; Procedure
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 18 Years to 80 Years · Male only
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Hemophilia A
Interventions: SPK-8011; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2035
U.S. locations: 27
States / cities: Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more

Conditions: Hemophilia B
Interventions: PF-06838435 (formerly SPK-9001); Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 17
States / cities: Sacramento, California • Metairie, Louisiana • New Orleans, Louisiana + 4 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Testing of hepatic AAV Vector integration; Diagnostic Test
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 173 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2040
U.S. locations: 14
States / cities: Rancho Cordova, California • Sacramento, California • San Francisco, California + 5 more

Conditions: Hemophilia A
Interventions: Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII; Biological
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: 18 Years and older · Male only
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2033
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: Hemophilia A
Interventions: ASC618; Biological
Lead sponsor: ASC Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Little Rock, Arkansas

Conditions: Hemophilia A
Interventions: BAY2599023 (DTX201); Drug
Lead sponsor: Bayer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 3
States / cities: Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin

Conditions: Hemophilia A
Interventions: SIG-001; Combination Product
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 3
States / cities: Indianapolis, Indiana • Boston, Massachusetts • Seattle, Washington

Conditions: Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Interventions: Valoctocogene roxaparvovec; Biological
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Hemophilia A
Interventions: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 77 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 19
States / cities: Mountain View, California • Oakland, California • Palo Alto, California + 5 more

Conditions: Hemophilia A
Interventions: valoctocogene roxaparvovec; Biological
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 3
States / cities: Sacramento, California • St Louis, Missouri • Columbus, Ohio

Conditions: Hemophilia B
Interventions: PF-06838435/ fidanacogene elaparvovec; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2031
U.S. locations: 17
States / cities: San Francisco, California • Aurora, Colorado • Indianapolis, Indiana + 3 more

Conditions: Hemophilia A
Interventions: SB-525 (PF-07055480); Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 22
States / cities: Little Rock, Arkansas • Duarte, California • Sacramento, California + 7 more

Conditions: Hemophilia A
Interventions: SPK-8011QQ; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 3
States / cities: Orange, California • San Francisco, California • Vallejo, California

Conditions: Hemophilia A
Interventions: Valoctocogene Roxaparvovec; Biological
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Hemophilia B
Interventions: AAV5-hFIXco-Padua, Factor IX (FIX); Genetic · Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 19
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more

Conditions: Hemophilia A
Interventions: valoctocogene roxaparvovec; Biological
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 144 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 15
States / cities: Los Angeles, California • Sacramento, California • San Diego, California + 12 more

Conditions: Hemophilia A
Interventions: Not listed
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 172 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2040
U.S. locations: 13
States / cities: Los Angeles, California • Sacramento, California • San Diego, California + 10 more

Conditions: Hemophilia B, Hemophilia A
Interventions: Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 212 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 13
States / cities: Palo Alto, California • San Francisco, California • Stanford, California + 7 more

Conditions: Hemophilia A
Interventions: AAV2/8-HLP-FVIII-V3; Biological
Lead sponsor: University College, London; Other
Eligibility: 18 Years and older · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2029
U.S. locations: 3
States / cities: Boise, Idaho • Lexington, Kentucky • Memphis, Tennessee

Conditions: Hemophilia A
Interventions: SPK-8011, SPK-8016; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2032
U.S. locations: 9
States / cities: Boston, Massachusetts • Madison, Mississippi • Kansas City, Missouri + 5 more

Conditions: Hemophilia B
Interventions: AAV5-hFIXco-Padua (AMT-061); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 4
States / cities: Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more

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Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

A Gene Transfer Study for Hemophilia A

Liver Biopsy Following Gene Therapy For Hemophilia

Study of a Gene Therapy Treatment for Hemophilia A

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

ASC618 Gene Therapy in Hemophilia A Patients

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Gene Therapy for Haemophilia A.

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

Dose Confirmation Trial of AAV5-hFIXco-Padua