Spark Therapeutics, Inc.
- Country
- 🇺🇸United States
- Ownership
- Public, Subsidiary
- Established
- 2013-01-01
- Employees
- 501
- Market Cap
- -
- Website
- http://www.sparktx.com
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
- Conditions
- ChoroideremiaCHM (Choroideremia) Gene Mutations
- Interventions
- Biological: AAV2-hCHM
- First Posted Date
- 2015-01-19
- Last Posted Date
- 2024-01-25
- Lead Sponsor
- Spark Therapeutics, Inc.
- Target Recruit Count
- 15
- Registration Number
- NCT02341807
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸University of Pennsylvania, Philadelphia, Pennsylvania, United States
🇺🇸Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, United States
Hemophilia B Gene Therapy With AAV8 Vector
- Conditions
- Hemophilia B
- Interventions
- Biological: AAV8-hFIX19
- First Posted Date
- 2012-06-15
- Last Posted Date
- 2019-03-12
- Lead Sponsor
- Spark Therapeutics, Inc.
- Target Recruit Count
- 4
- Registration Number
- NCT01620801
- Locations
- 🇦🇺
Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia
🇺🇸The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸University of Pittsburgh, Pittsburgh, Pennsylvania, United States
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
- Conditions
- Leber Congenital Amaurosis
- Interventions
- First Posted Date
- 2010-09-24
- Last Posted Date
- 2025-04-29
- Lead Sponsor
- Spark Therapeutics, Inc.
- Target Recruit Count
- 12
- Registration Number
- NCT01208389
- Locations
- 🇺🇸
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
- Conditions
- Leber Congenital AmaurosisInherited Retinal Dystrophy Due to RPE65 Mutations
- Interventions
- Biological: AAV2-hRPE65v2,voretigene neparvovec-rzyl
- First Posted Date
- 2009-10-22
- Last Posted Date
- 2025-04-23
- Lead Sponsor
- Spark Therapeutics, Inc.
- Target Recruit Count
- 31
- Registration Number
- NCT00999609
- Locations
- 🇺🇸
University of Iowa, Iowa City, Iowa, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
Safety Study in Subjects With Leber Congenital Amaurosis
- Conditions
- Leber Congenital Amaurosis
- Interventions
- Biological: voretigene neparvovec-rzyl
- First Posted Date
- 2007-08-15
- Last Posted Date
- 2020-11-02
- Lead Sponsor
- Spark Therapeutics, Inc.
- Target Recruit Count
- 12
- Registration Number
- NCT00516477
- Locations
- 🇺🇸
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
LTFU for Gene Transfer Subjects With Hemophilia B
- Conditions
- Hemophilia B
- First Posted Date
- 2007-08-14
- Last Posted Date
- 2020-05-15
- Lead Sponsor
- Spark Therapeutics, Inc.
- Target Recruit Count
- 4
- Registration Number
- NCT00515710
- Locations
- 🇺🇸
Hemophilia Center of Western Pennsylvania, Pittsburgh, Pennsylvania, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States