Clinical trial NCT03432520: Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Official title: A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemophilia A
Lead sponsor: Spark Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-02-14
Enrollment: 29
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Males with hemophilia A, who received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored study.

Brief summary: 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.

Detailed 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).

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Up to 9 years
measure: Number of Participants with Adverse Events of Special Interest (AESIs)
timeFrame: Up to 9 years
measure: Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development
timeFrame: Up to 9 years
measure: Number of Participants with Clinical Laboratory Abnormalities
timeFrame: Up to 9 years
measure: Number of Participants with Vital Sign Abnormalities
timeFrame: Up to 9 years
measure: Number of Participants with Physical Exam Abnormalities
timeFrame: Up to 9 years
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Madison
country: United States
facility: Mississippi Center for Advanced Medicine
state: Mississippi
city: Kansas City
country: United States
facility: Truman Medical Centers
state: Missouri
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Hershey
country: United States
facility: Pennsylvania State University Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Hemophilia Center of Western Pennsylvania
state: Pennsylvania
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Sydney
country: Australia
facility: Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
state: New South Wales
city: Melbourne
country: Australia
facility: The Alfred Hospital & Monash Medical Centre
state: Victoria
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
Study officials:
affiliation: Spark Therapeutics, Inc.
name: Clinical Trial Director
role: STUDY_DIRECTOR
References:
citation: George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.