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Active (not recruiting) NCT03432520

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

Conditions: Hemophilia A

Sponsor: Spark Therapeutics, Inc.

trial.available_in: БГ
Overview
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
Description
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).
Who can participate
Inclusion Criteria Participants are eligible to be included in the study only if all of the following criteria apply: 1. Have received a single intravenous administration of SPK-8011 or SPK-8016 in either Study SPK-8011-101 or SPK-8016-101, respectively. 2. Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures. Exclusion Criteria Participants are excluded from the study if any of the following criteria apply: 1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
Locations 3
Australia (1)
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
Sydney , New South Wales
Canada (1)
St. Michael's Hospital
Toronto , Ontario
United States (1)
Boston Children's Hospital
Boston , Massachusetts
Technical details
Status
Active (not recruiting)
Study type
OBSERVATIONAL
Sex
Male only
Minimum age
18 Years
Healthy volunteers
No
Start date
14.08.2018
Completion date
01.12.2032
Registry ID
NCT03432520
Source
anzctr
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