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NCT03754790 · ClinicalTrials.gov registry record · Phase 3
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
A Phase 3 study of Hemophilia, sponsored by Genzyme, a Sanofi Company.
- Active
- Registry status
- Phase 3
- Development phase
- 281
- Enrollment target
- 20
- Study locations
NCT03754790: Active Phase 3 study of Hemophilia, sponsored by Genzyme, a Sanofi Company.
NCT03754790 is a Phase 3 study of Hemophilia that is active but no longer recruiting, run by Genzyme, a Sanofi Company. The registered enrollment target is 281 participants, below the 379-participant average among 9 other Hemophilia trials with a reported enrollment target (26% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03754790, a Phase 3 study of Hemophilia, is active but no longer recruiting, sponsored by Genzyme, a Sanofi Company.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 281 participants
- Enrollment target
- 20
- Study locations
Study Summary
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: * To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: * Bleeding episodes * Spontaneous bleeding episodes * Joint bleeding episodes * To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Primary Outcome
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
Conditions Studied
Interventions
- DRUG Fitusiran
Study Locations (20)
Other
- Investigational Site Number : 1560004 - Beijing
- Investigational Site Number : 1560007 - Beijing
- Investigational Site Number : 1560014 - Changsha
- Investigational Site Number : 1560009 - Chengdu
- Investigational Site Number : 1560002 - Guangzhou
- Investigational Site Number : 1560011 - Guiyang
California
- Children's Hospital Los Angeles Site Number : 8400019 - Los Angeles
- Center for Inherited Blood Disorders (CIBD) Site Number : 8400016 - Orange
Florida
- Nemours Children's Clinic Site Number : 8400008 - Jacksonville
- St Joseph's Children's Hospital of Tampa Site Number : 8400002 - Tampa
Arizona
- Phoenix Childrens Hospital Site Number : 8400009 - Phoenix
Illinois
- Rush University Medical Center -1725 W Harrison St Site Number : 8400001 - Chicago
Massachusetts
- ~Massachusetts General Hospital Site Number : 8400011 - Boston
Michigan
- University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012 - Ann Arbor
Nevada
- Alliance for Childhood Diseases Site Number : 8400007 - Las Vegas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 281 participants |
| Start Date | 2019-01-09 |
| Est. Completion | 2026-11-05 |
| Phase | Phase 3 |
What the registry record for NCT03754790 still lists
NCT03754790 is an interventional study that assigns participants to a tested intervention. The registered 281 participants enrollment target is mid-sized for trials with a published cap, below the 379-participant average among 9 other Hemophilia trials with a reported enrollment target (26% lower).
The record links to 1 condition, with Hemophilia appearing as the primary indexed condition, and to 1 intervention - of which Fitusiran is the first listed.
NCT03754790 names 20 study sites across 13 states, led by Other, California, Florida.
Frequently Asked Questions
What is clinical trial NCT03754790 about?
NCT03754790 is a clinical study titled "Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX". Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: * To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: * Bleeding episodes * Spontaneous bleeding episodes * Joint bleeding episodes * To...
What is the current status of trial NCT03754790?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 281 participants. The study started on 2019-01-09. Estimated completion is 2026-11-05.
What conditions does trial NCT03754790 study?
This clinical trial studies the following conditions: Hemophilia.
What interventions are being tested in trial NCT03754790?
The interventions under investigation include: Fitusiran (DRUG).
Who is sponsoring clinical trial NCT03754790?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03754790 being conducted?
This trial has 20 study locations across Arizona, California, Florida, Illinois, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Hemophilia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03754790's enrollment target sits among peer trials
281 2nd of 9 the highest of 9 other Hemophilia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
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-
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
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-
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
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ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
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-
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The Use of Cyclokapron for Treatment and Management of Women With Bleeding Disorders
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