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NCT Number: NCT03432520

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

18 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Royal Prince Alfred Hospital Department of Cell & Molecular Therapies, Sydney, New South Wales, Australia

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Participants are eligible to be included in the study only if all of the following criteria apply:

  • Have received a single intravenous administration of SPK-8011 or SPK-8016 in either Study SPK-8011-101 or SPK-8016-101, respectively.
  • 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.

Treatment and study plan

SPK-8011

Genetic

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

SPK-8016

Genetic

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

Primary outcomes

  1. Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Up to 9 years

  2. Number of Participants with Adverse Events of Special Interest (AESIs)

    Time frame: Up to 9 years

  3. Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development

    Time frame: Up to 9 years

  4. Number of Participants with Clinical Laboratory Abnormalities

    Time frame: Up to 9 years

  5. Number of Participants with Vital Sign Abnormalities

    Time frame: Up to 9 years

  6. Number of Participants with Physical Exam Abnormalities

    Time frame: Up to 9 years

Sponsors and collaborators

Lead sponsor

Spark Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2018
Primary completion
2032
Study completion
2032
First posted
Feb 14, 2018
Registry last updated
Nov 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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