Clinical trial • Phase IV • Rare Disease
ISARALGAGENE CIVAPARVOVEC for Fabry disease
Phase IV trial of ISARALGAGENE CIVAPARVOVEC for Fabry disease. 44 participants.
Overview
- Trial Therapeutic Area
- Rare Disease
- Trial Disease
- Fabry disease
- Trial Stage
- Phase IV
- Drug Modality
- Gene therapy
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 25-10-2024
- First CTIS Authorization Date
- 31-01-2025
Trial design
Phase IV trial across 1 site in Germany.
- Target Sample Size
- 44
- Trial Duration For Participant
- 5479
Eligibility
Recruits 44 No vulnerable populations selected; adult subjects only; informed consent required from participants. Subject information sheet and informed consent form for adults available..
- Vulnerable Population
- No vulnerable populations selected; adult subjects only; informed consent required from participants. Subject information sheet and informed consent form for adults available.
Inclusion criteria
- {"criterion_text":"-Subjects who received ST-920 therapy in a separate parent trial"}
- {"criterion_text":"-Subjects who have consented to participate in this LTFU study."}
Exclusion criteria
- {"criterion_text":"-This study has no exclusion criteria"}
Endpoints
Primary endpoints
- {"endpoint_text":"-To evaluate long-term safety of ST-920 by assessment of incidence and severity of adverse events (AEs)","definition_or_measurement_approach":"Assessment of incidence and severity of adverse events (AEs)."}
Recruitment
- Planned Sample Size
- 44
- Recruitment Window Months
- 180
- Consent Approach
- Informed consent obtained from adult participants; subject information sheet and informed consent form for adults available. No assent/parental consent procedures specified; languages not specified.
Geography
- Total Number Of Sites
- 1
- Total Number Of Participants
- 44
Germany
- Earliest CTIS Part Ii Submission Date
- 10-01-2025
- Latest Decision Or Authorization Date
- 18-06-2025
- Processing Time Days
- 159
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Universitaetsklinikum Wuerzburg AöR
- Department Name
- Medical Clinic and Policlinic I - Cardiology
- Principal Investigator Name
- Peter Nordbeck
- Principal Investigator Email
- nordbeck_p@ukw.de
- Contact Person Name
- Peter Nordbeck
- Contact Person Email
- nordbeck_p@ukw.de
- Number Of Participants
- 1
Sponsor
Primary sponsor
- Full Name
- Sangamo Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Frontage Laboratories Inc.
- Responsibilities
- code:4
- Name
- PPD Development LP
- Responsibilities
- code:4
- Name
- Medpace Finland Oy
- Responsibilities
- codes:1,10,12,2,5,6,7,8,9
Third parties
- {"country":"United States","full_name":"Frontage Laboratories Inc.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"codes:1,10,12,2,5,6,7,8,9","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ST-920
- Active Substance
- ISARALGAGENE CIVAPARVOVEC
- Modality
- Gene therapy
- Routes Of Administration
- SOLUTION FOR INJECTION/INFUSION
- Route
- Other use
- Orphan Designation
- Yes
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