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Long-Term Safety and Efficacy of SPK-8011 in Males With Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03432520
Recruitment Status : Enrolling by invitation
First Posted : February 14, 2018
Last Update Posted : December 17, 2018
Sponsor:
Information provided by (Responsible Party):
Spark Therapeutics

Brief Summary:
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 in any Spark-sponsored SPK-8011 study.

Condition or disease Intervention/treatment
Hemophilia A Genetic: SPK-8011

Detailed Description:
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 in any prior Spark-sponsored SPK-8011 study. Subjects will be followed for up to a total of 5 years post infusion (including the time on the dosing study).

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Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Multi-Center Evaluation of the Long-Term Safety and Efficacy of SPK-8011 [Adeno-Associated Viral Vector With B-Domain Deleted Human Factor VIII Gene] in Males With Hemophilia A
Actual Study Start Date : August 14, 2018
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia


Intervention Details:
  • Genetic: SPK-8011
    Observational long-term safety follow-up study of subjects previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study


Primary Outcome Measures :
  1. Incidence of FVIII Inhibitors [ Time Frame: 4 years ]
    Incidence of FVIII Inhibitors

  2. Incidence of all adverse events [ Time Frame: 4 years ]
    Incidence of all adverse events

  3. Incidence(s) of new or exacerbation of adverse events of interest [ Time Frame: 4 years ]
    Such as: malignancies, neurologic, rheumatologic or other autoimmune, or hematologic disorders

  4. Annual bleeding rate [ Time Frame: 4 years ]
    Annual bleeding rate

  5. FVIII activity levels [ Time Frame: 4 years ]
    FVIII activity levels

  6. Total FVIII consumption [ Time Frame: 4 years ]
    Total FVIII consumption

  7. Number of FVIII infusions [ Time Frame: 4 years ]
    Number of FVIII infusions


Secondary Outcome Measures :
  1. Joint Health Assessment [ Time Frame: 4 years ]
    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

  2. Target Joint Assessment [ Time Frame: 4 years ]
    Investigator assessment of bleeding in target joints (e.g., hip, elbow, wrist, shoulder, knee, and ankle)

  3. Haem-A-QoL [ Time Frame: 4 years ]
    Quality-of-life (QoL) assessment

  4. EQ-5D-5L [ Time Frame: 4 years ]
    Quality-of-life (QoL) assessment

  5. Activities Assessments [ Time Frame: 4 years ]
    Hemophilia Activities List

  6. Health Economics Assessment [ Time Frame: 4 years ]
    Collection of information including number of hospitalizations, emergency room visits, physician visits and work/school days missed



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Genetic males
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Males with hemophilia A, who received a single intravenous administration of SPK-8011 in any Spark-sponsored SPK-8011 study.
Criteria

Inclusion Criteria:

  1. Males with hemophilia A, who have received a single intravenous administration of SPK-8011 in any Spark sponsored SPK-8011 study
  2. Individuals who understand the purpose and the risks of the study, and who provide signed and dated informed consent before undergoing any study-specific procedures.

Exclusion Criteria:

1. Individuals who are unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03432520


Locations
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United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
Sponsors and Collaborators
Spark Therapeutics
Investigators
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Study Director: Clinical Trial Director Spark Therapeutics, Inc.

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Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT03432520     History of Changes
Other Study ID Numbers: SPK-8011-LTFU
First Posted: February 14, 2018    Key Record Dates
Last Update Posted: December 17, 2018
Last Verified: December 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants