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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Hemophilia A
• Intervention / Treatment: Genetic: SPK-8011; Genetic: SPK-8016

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).

• Study Type: Observational
• Enrollment (Actual): 29

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2050
      • Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
  • Victoria
    • Melbourne, Victoria, Australia
      • The Alfred Hospital & Monash Medical Centre
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5B 1W8
      • St. Michael's Hospital
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Mississippi
    • Madison, Mississippi, United States, 39110
      • Mississippi Center for Advanced Medicine
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Truman Medical Centers
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 10733
      • Pennsylvania State University Milton S. Hershey Medical Center
    • Philadelphia, Pennsylvania, United States, 19104
      • The Children's Hospital of Philadelphia
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University
    • Pittsburgh, Pennsylvania, United States, 15213
      • Hemophilia Center of Western Pennsylvania
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)	Up to 9 years
Number of Participants with Adverse Events of Special Interest (AESIs)	Up to 9 years
Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development	Up to 9 years
Number of Participants with Clinical Laboratory Abnormalities	Up to 9 years
Number of Participants with Vital Sign Abnormalities	Up to 9 years
Number of Participants with Physical Exam Abnormalities	Up to 9 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Target Joint Assessment	Investigator assessment of bleeding in target joints (e.g., hip, elbow, wrist, shoulder, knee, and ankle)	4 years
Haem-A-QoL	Quality-of-life (QoL) assessment	4 years
EQ-5D-5L	Quality-of-life (QoL) assessment	4 years
Activities Assessments	Hemophilia Activities List	4 years
Health Economics Assessment	Collection of information including number of hospitalizations, emergency room visits, physician visits and work/school days missed	4 years

Collaborators and Investigators

• Sponsor: Spark Therapeutics, Inc.
• Investigators: Study Director: Clinical Trial Director, Spark Therapeutics, Inc.

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): August 14, 2018
• Primary Completion (Estimated): December 1, 2032
• Study Completion (Estimated): December 1, 2032

Study Registration Dates

• First Submitted: January 24, 2018
• First Submitted That Met QC Criteria: February 7, 2018
• First Posted (Actual): February 14, 2018

Study Record Updates

• Last Update Posted (Actual): November 29, 2024
• Last Update Submitted That Met QC Criteria: November 26, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A
• Other Study ID Numbers: SPK-8011/8016-LTFU

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No