Clinical trial

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

Name

SPK-8011/8016-LTFU

Description

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.

Trial arms

Trial start

2018-08-14

Estimated PCD

2032-12-01

Trial end

2032-12-01

Status

Active (not recruiting)

Treatment

SPK-8011

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

Size

Primary endpoint

Incidence of FVIII Inhibitors

4 years

Incidence of all adverse events

4 years

Incidence(s) of new or exacerbation of adverse events of interest

4 years

Annual bleeding rate

4 years

FVIII activity levels

4 years

Total FVIII consumption

4 years

Number of FVIII infusions

4 years

Eligibility criteria

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.

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 29, 'type': 'ACTUAL'}}

Updated at

2024-01-22

1 organization

1 product

1 indication

Organization

Spark Therapeutics

Product

SPK-8011

Indication

Hemophilia A