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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03432520
Other study ID # SPK-8011/8016-LTFU
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
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