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Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies 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 : July 18, 2022
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 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.

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 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 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 : 40 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies 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



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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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

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.


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, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Mississippi
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States, 39110
United States, Missouri
Truman Medical Centers
Kansas City, Missouri, United States, 64108
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Pennsylvania State University Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 10733
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Thomas Jefferson University
Philadelphia, Pennsylvania, United States, 19107
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Australia, New South Wales
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
Sydney, New South Wales, Australia, 2050
Sponsors and Collaborators
Spark Therapeutics
Investigators
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Study Director: Clinical Trial Director Spark Therapeutics, Inc.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT03432520    
Other Study ID Numbers: SPK-8011/8016-LTFU
First Posted: February 14, 2018    Key Record Dates
Last Update Posted: July 18, 2022
Last Verified: July 2022

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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