DeepMed Research

Spark Therapeutics, Inc.

🇺🇸United States

Country: 🇺🇸United States

Ownership: Public, Subsidiary

Established: 2013-01-01

Employees: 501

Market Cap: -

Website: http://www.sparktx.com

Clinical Trials

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

Phase 1

Recruiting

Conditions: Huntington Disease

First Posted Date: 2025-02-14

Last Posted Date: 2025-05-22

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 53

Registration Number: NCT06826612

Locations: 🇺🇸
Ohio State University, Columbus, Ohio, United States
🇺🇸
University of Cincinnati/Cincinnati Children's Hospital, Cincinnati, Ohio, United States

Study of a Gene Therapy Treatment for Hemophilia A

Phase 3

Withdrawn

Conditions: Hemophilia A

Interventions: Genetic: SPK-8011

First Posted Date: 2024-03-07

Last Posted Date: 2024-12-13

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 85

Registration Number: NCT06297486

Locations: 🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸
Loma Linda University Health, Loma Linda, California, United States
🇺🇸
Orthopaedic Institute for Children/Orthopaedic Hemophilia Treatment Center, Los Angeles, California, United States

and more 24 locations

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

Phase 1

Active, not recruiting

Conditions: Glycogen Storage Disease Type II
Lysosomal Storage Diseases
Glycogen Storage Disease Type 2
LOPD
Pompe Disease
Pompe Disease (Late-onset)
Acid Maltase Deficiency

Interventions: Genetic: SPK-3006

First Posted Date: 2019-09-18

Last Posted Date: 2024-11-27

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 4

Registration Number: NCT04093349

Locations: 🇫🇷
Centre Hospitalier Régional Universitaire de Lille, Lille Cedex, France
🇬🇧
Salford Royal MHS Foundation Trust, Salford, United Kingdom
🇬🇧
New Queen Elizabeth Hospital Birmingham, Birmingham, GBR, United Kingdom

and more 26 locations

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

Not Applicable

Completed

Conditions: Lysosomal Storage Diseases
Glycogen Storage Disease Type 2
LOPD
Pompe Disease
Pompe Disease (Late-onset)
Acid Maltase Deficiency

Interventions: Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid

First Posted Date: 2019-03-28

Last Posted Date: 2023-03-24

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 61

Registration Number: NCT03893240

Locations: 🇩🇪
Klinikum der Universität München, München, Germany
🇺🇸
University of Minnesota Medical School, Minneapolis, Minnesota, United States
🇺🇸
University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States

and more 15 locations

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

Completed

Conditions: Blood Coagulation Disorders, Inherited
Hemorrhagic Disorders
Blood Coagulation Disorder
Coagulation Protein Disorders
Hemophilia A
Genetic Diseases, Inborn
Factor VIII Deficiency
Hematologic Diseases
Genetic Diseases, X-Linked

Interventions: Drug: Standard of Care FVIII Replacement therapy

First Posted Date: 2019-03-15

Last Posted Date: 2023-07-10

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 25

Registration Number: NCT03876301

Locations: 🇺🇸
University of Florida, Gainesville, Florida, United States
🇦🇺
The Alfred Hospital, Melbourne, Victoria, Australia
🇨🇦
Providence Hematology/St. Paul's Hosptial, Vancouver, British Columbia, Canada

and more 8 locations

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

Phase 1

Completed

Conditions: Coagulation Protein Disorders
Factor VIII (FVIII) Deficiency
Factor VIII (FVIII) Gene
Genetic Diseases, Inborn
Gene Transfer
Hemorrhagic Disorders
Gene Therapy
Factor VIII (FVIII)
Factor VIII (FVIII) Protein
Hematologic Diseases

Interventions: Genetic: SPK-8016

First Posted Date: 2018-11-08

Last Posted Date: 2024-02-23

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 4

Registration Number: NCT03734588

Locations: 🇺🇸
Orthopaedic Institute for Children, Los Angeles, California, United States
🇺🇸
Weill Cornell Medicine, New York, New York, United States
🇺🇸
Illinois Bleeding and Clotting Disorders Institute, Peoria, Illinois, United States

and more 8 locations

Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Active, not recruiting

Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations

First Posted Date: 2018-07-27

Last Posted Date: 2025-04-18

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 41

Registration Number: NCT03602820

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

Active, not recruiting

Conditions: Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

First Posted Date: 2018-07-24

Last Posted Date: 2025-04-18

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 87

Registration Number: NCT03597399

Locations: 🇺🇸
Children's Hospital of Los Angeles, Los Angeles, California, United States
🇺🇸
Bascom Palmer Eye Institute, Miami, Florida, United States
🇺🇸
University of Iowa Hospitals & Clinics, Iowa City, Iowa, United States

and more 7 locations

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

Active, not recruiting

Conditions: Hemophilia A

First Posted Date: 2018-02-14

Last Posted Date: 2024-11-29

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 29

Registration Number: NCT03432520

Locations: 🇺🇸
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸
Pennsylvania State University Milton S. Hershey Medical Center, Hershey, Pennsylvania, United States

and more 9 locations

A Gene Transfer Study for Hemophilia A

Phase 1

Completed

Conditions: Hemophilia A

Interventions: Genetic: SPK-8011

First Posted Date: 2016-12-28

Last Posted Date: 2024-12-30

Lead Sponsor: Spark Therapeutics, Inc.

Target Recruit Count: 25

Registration Number: NCT03003533

Locations: 🇺🇸
Jefferson University Hospitals, Philadelphia, Pennsylvania, United States
🇺🇸
University of California Davis - Hemostasis and Thrombosis Center, Sacramento, California, United States
🇮🇱
Chaim Sheba Center, Ramat Gan, Tel Hashomer, Israel

and more 13 locations

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