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Showing 1–14 of 14 matching trials from the live ClinicalTrials.gov search.

Filters: Sponsor: Spark Therapeutics, Inc. Clear all

Conditions: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions: SPK-3006; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2032
U.S. locations: 10
States / cities: Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more

Conditions: Leber Congenital Amaurosis
Interventions: voretigene neparvovec-rzyl; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 8 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2027
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

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: Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Hematologic Diseases, Hemorrhagic Disorders, Factor VIII Deficiency
Interventions: Standard of Care FVIII Replacement therapy; Drug
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 25 participants
Timeline: 2019 – 2023
U.S. locations: 6
States / cities: San Francisco, California • Gainesville, Florida • Atlanta, Georgia + 3 more

Conditions: Choroideremia, CHM (Choroideremia) Gene Mutations
Interventions: AAV2-hCHM; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2022
U.S. locations: 3
States / cities: Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Hemophilia B
Interventions: AAV8-hFIX19; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania

Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Interventions: AAV2-hRPE65v2,voretigene neparvovec-rzyl; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 3 Years and older
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2030
U.S. locations: 2
States / cities: Iowa City, Iowa • Philadelphia, Pennsylvania

Conditions: Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions: AAV2-hRPE65v2,voretigene neparvovec-rzyl; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 12 Months and older
Enrollment: 87 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 10
States / cities: Los Angeles, California • Miami, Florida • Iowa City, Iowa + 6 more

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
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: Not listed
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2017
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania

Conditions: Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions: Neutralizing Antibody to SPK-3006 capsid; Diagnostic Test
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 6
States / cities: Phoenix, Arizona • Orange, California • Kansas City, Kansas + 3 more

Conditions: Leber Congenital Amaurosis
Interventions: voretigene neparvovec-rzyl; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 8 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2018
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

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A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

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

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Hemophilia B Gene Therapy With AAV8 Vector

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

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

Study of a Gene Therapy Treatment for Hemophilia A

LTFU for Gene Transfer Subjects With Hemophilia B

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Safety Study in Subjects With Leber Congenital Amaurosis