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

Hemophilia A Clinical Trial

Official title:

A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03432520
• Other study ID #: SPK-8011/8016-LTFU
• Status: Active, not recruiting
• Start date: August 14, 2018
• Est. completion date: December 2032

Study information

• Verified date: January 2024
• Source: Spark Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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. Subjects will be followed for up to a total of 5 years post infusion (including the time on the dosing study).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 29
• Est. completion date: December 2032
• Est. primary completion date: December 2032
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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 any Spark-sponsored gene therapy study; and

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Genetic:
• SPK-8011
Observational long-term safety follow-up study of subjects previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study

Locations

Country	Name	City	State
Australia	The Alfred Hospital & Monash Medical Centre	Melbourne	Victoria
Australia	Royal Prince Alfred Hospital Department of Cell & Molecular Therapies	Sydney	New South Wales
Canada	St. Michael's Hospital	Toronto	Ontario
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Pennsylvania State University Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	Truman Medical Centers	Kansas City	Missouri
United States	Mississippi Center for Advanced Medicine	Madison	Mississippi
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Thomas Jefferson University	Philadelphia	Pennsylvania
United States	Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Virginia Commonwealth University	Richmond	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Spark Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of FVIII Inhibitors	Incidence of FVIII Inhibitors	4 years
Primary	Incidence of all adverse events	Incidence of all adverse events	4 years
Primary	Incidence(s) of new or exacerbation of adverse events of interest	Such as: malignancies, neurologic, rheumatologic or other autoimmune, or hematologic disorders	4 years
Primary	Annual bleeding rate	Annual bleeding rate	4 years
Primary	FVIII activity levels	FVIII activity levels	4 years
Primary	Total FVIII consumption	Total FVIII consumption	4 years
Primary	Number of FVIII infusions	Number of FVIII infusions	4 years
Secondary	Joint Health Assessment	Assessment of joint health for six index joints using a modified Hemophilia Joint Health Score (HJHS). The HJHS is a physical examination assessment tool, that measures joint health in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in hemophilia: the knees, ankles, and elbows	4 years
Secondary	Target Joint Assessment	Investigator assessment of bleeding in target joints (e.g., hip, elbow, wrist, shoulder, knee, and ankle)	4 years
Secondary	Haem-A-QoL	Quality-of-life (QoL) assessment	4 years
Secondary	EQ-5D-5L	Quality-of-life (QoL) assessment	4 years
Secondary	Activities Assessments	Hemophilia Activities List	4 years
Secondary	Health Economics Assessment	Collection of information including number of hospitalizations, emergency room visits, physician visits and work/school days missed	4 years