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

NCT ID: NCT03432520

Last Updated: 2024-11-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

29 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-08-14

Study Completion Date

2032-12-31

Brief Summary

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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

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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).

Conditions

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Hemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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SPK-8011

Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study

Intervention Type GENETIC

SPK-8016

Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study

Intervention Type GENETIC

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Spark Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trial Director

Role: STUDY_DIRECTOR

Spark Therapeutics, Inc.

Locations

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Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Mississippi Center for Advanced Medicine

Madison, Mississippi, United States

Site Status

Truman Medical Centers

Kansas City, Missouri, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Pennsylvania State University Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Royal Prince Alfred Hospital Department of Cell & Molecular Therapies

Sydney, New South Wales, Australia

Site Status

The Alfred Hospital & Monash Medical Centre

Melbourne, Victoria, Australia

Site Status

St. Michael's Hospital

Toronto, Ontario, Canada

Site Status

Countries

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United States Australia Canada

References

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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.

Reference Type DERIVED
PMID: 34788507 (View on PubMed)

Other Identifiers

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SPK-8011/8016-LTFU

Identifier Type: -

Identifier Source: org_study_id