A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
Public ClinicalTrials.gov record NCT03587116. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, LEAD-IN STUDY TO EVALUATE AT LEAST 6 MONTHS OF PROSPECTIVE EFFICACY AND SAFETY DATA OF FACTOR IX OR FACTOR VIII PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR-SPARK100 AND MODERATELY SEVERE TO SEVERE HEMOPHILIA A ADULT SUBJECTS (FVIII:C≤1%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR SB-525 CAPSID (AAV6), PRIOR TO THE RESPECTIVE THERAPEUTIC PH 3 GENE THERAPY STUDIES (See Detailed Description Section for Official Protocol Title)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study. The enrollment for hemophilia A participants is completed. At this time participants are only being enrolled for hemophilia B cohort.
Study identification
- NCT ID
- NCT03587116
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 212 participants
Conditions and interventions
Conditions
Interventions
Drug
Eligibility (public fields only)
- Age range
- 18 Years to 64 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 26, 2018 (Actual)
- Primary completion
- Dec 13, 2024 (Actual)
- Completion
- Dec 13, 2024 (Actual)
- Last update posted
- Nov 6, 2025
2018 – 2024
United States locations
- U.S. sites
- 13
- U.S. states
- 8
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Clinical and Translational Research Unit (CTRU) | Palo Alto | California | 94304 | — |
| Lucile Packard Childrens Hospital | Palo Alto | California | 94304 | — |
| University of California, San Francisco - Outpatient Hematology Clinic | San Francisco | California | 94143 | — |
| Stanford Health Care | Stanford | California | 94305 | — |
| Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | — |
| Emory University Hospital Midtown | Atlanta | Georgia | 30308 | — |
| Emory University Hospital | Atlanta | Georgia | 30322 | — |
| Investigational Drug Service | Atlanta | Georgia | 30322 | — |
| Indiana Hemophilia & Thrombosis Center, Inc. | Indianapolis | Indiana | 46260 | — |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | — |
| Alliance for Childhood Diseases | Las Vegas | Nevada | 89135 | — |
| Penn Blood Disorder Center | Philadelphia | Pennsylvania | 19104 | — |
| Bloodworks NW | Seattle | Washington | 98104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 62 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03587116, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 6, 2025 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03587116 live on ClinicalTrials.gov.