Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
| Status: | Recruiting |
|---|---|
| Conditions: | Blood Cancer, Women's Studies, Anemia, Hematology, Hematology, Hematology, Hematology |
| Therapuetic Areas: | Hematology, Oncology, Reproductive |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 3/17/2019 |
| Start Date: | January 21, 2019 |
| End Date: | July 2020 |
| Contact: | Clinical Director |
| Email: | clinicaltrials@sparktx.com |
| Phone: | 215-220-9300 |
A Multi-Center, Observational Study in Males With Hemophilia A
The aim of this prospective, observational study is to establish a dataset on the frequency
of bleeding events, as well as other characteristics of bleeding events and FVIII infusions,
in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement
therapy as standard of care. The data collected from this study may assist in providing
baseline information for comparison to the Spark's investigational hemophilia A gene therapy
in future Phase 3 studies.
of bleeding events, as well as other characteristics of bleeding events and FVIII infusions,
in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement
therapy as standard of care. The data collected from this study may assist in providing
baseline information for comparison to the Spark's investigational hemophilia A gene therapy
in future Phase 3 studies.
Inclusion Criteria:
1. Able and willing to provide written informed consent.
2. Males ≥18 years of age.
3. Clinically severe hemophilia A
4. Previous exposure to FVIII therapy
5. No prior history of hypersensitivity or anaphylaxis associated with an FVIII or
intravenous immunoglobulin administration.
6. No measurable inhibitor against FVIII
7. Willing to participate and receive treatment in a future Spark hemophilia A gene
therapy study.
Exclusion Criteria:
1. Documented active hepatitis B or C within the past 12 months of Screening
2. Currently on antiviral therapy to treat hepatitis B or C;
3. Documented significant liver disease within the past 6 months of Screening
4. Have serological evidence of HIV-1 or HIV-2
5. Anti-AAV-Spark 200 neutralizing titers ≥1:1
6. Previously received SPK-8011;
7. Previously dosed with any investigational or approved gene therapy product at any time
or treated with an investigational drug within the last 12 weeks;
8. Planned surgical procedure in the next 12 months requiring FVIII prophylactic
treatment.
9. Any history of chronic infection or other chronic disease, concurrent clinically
significant major disease (such as liver abnormalities or type I diabetes) including
active malignancy, except for non-melanoma skin cancer, any other condition or any
other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the
participant unsuitable for participation and dosing in a future clinical study for
Spark's hemophilia A gene therapy.
10. Unable or unwilling to comply with the schedule of visits and/or study assessments
described in the protocol.
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