Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
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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 : February 10, 2022
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Condition or disease | Intervention/treatment |
---|---|
Hemophilia A | Genetic: SPK-8011 |
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 |

- Genetic: SPK-8011
Observational long-term safety follow-up study of subjects previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study
- Incidence of FVIII Inhibitors [ Time Frame: 4 years ]Incidence of FVIII Inhibitors
- Incidence of all adverse events [ Time Frame: 4 years ]Incidence of all adverse events
- 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
- Annual bleeding rate [ Time Frame: 4 years ]Annual bleeding rate
- FVIII activity levels [ Time Frame: 4 years ]FVIII activity levels
- Total FVIII consumption [ Time Frame: 4 years ]Total FVIII consumption
- Number of FVIII infusions [ Time Frame: 4 years ]Number of FVIII infusions
- 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
- Target Joint Assessment [ Time Frame: 4 years ]Investigator assessment of bleeding in target joints (e.g., hip, elbow, wrist, shoulder, knee, and ankle)
- Haem-A-QoL [ Time Frame: 4 years ]Quality-of-life (QoL) assessment
- EQ-5D-5L [ Time Frame: 4 years ]Quality-of-life (QoL) assessment
- Activities Assessments [ Time Frame: 4 years ]Hemophilia Activities List
- Health Economics Assessment [ Time Frame: 4 years ]Collection of information including number of hospitalizations, emergency room visits, physician visits and work/school days missed

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.
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 |
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 any Spark-sponsored gene therapy study; and
- 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.

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
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 |
Study Director: | Clinical Trial Director | Spark Therapeutics, Inc. |
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: | February 10, 2022 |
Last Verified: | February 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |