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NCT03974113 · ClinicalTrials.gov registry record · Phase 3
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
A Phase 3 study of Hemophilia, sponsored by Genzyme, a Sanofi Company.
- Active
- Registry status
- Phase 3
- Development phase
- 32
- Enrollment target
- 16
- Study locations
NCT03974113: Active Phase 3 study of Hemophilia, sponsored by Genzyme, a Sanofi Company.
NCT03974113 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 32 participants, below the 407-participant average among 9 other Hemophilia trials with a reported enrollment target (92% lower). The trial reports 16 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03974113, 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
- 32 participants
- Enrollment target
- 16
- Study locations
Study Summary
Primary Objective: \- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B Secondary Objectives: * To characterize the safety and tolerability * To determine fitusiran plasma concentrations at selected time points
Primary Outcome
Characterize the AT activity at the optimal therapeutic dose
Conditions Studied
Interventions
- DRUG Fitusiran
Study Locations (16)
Other
- Investigational Site Number : 3560006 - Bangalore
- Investigational Site Number : 3560002 - Mumbai
- Investigational Site Number : 3560001 - Pune-411011
- Investigational Site Number : 3560004 - Vellore
- Investigational Site Number : 3800002 - Florence
- Investigational Site Number : 3800001 - Milan
- Investigational Site Number : 7920001 - Adana
- Investigational Site Number : 7920002 - Istanbul
- Investigational Site Number : 7920003 - Izmir
Ontario
- Investigational Site Number : 1240001 - Hamilton
- Investigational Site Number : 1240002 - Ottawa
California
- Children's Hospital Los Angeles Site Number : 8400002 - Los Angeles
New Jersey
- Hackensack University Medical Center- Site Number : 8400008 - Hackensack
Ohio
- University Hospitals of Cleveland- Site Number : 8400007 - Cleveland
Pennsylvania
- Penn State Milton S. Hershey Medical Center- Site Number : 8400006 - Hershey
Madrid, Comunidad de
- Investigational Site Number : 7240002 - Madrid
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2020-01-28 |
| Est. Completion | 2026-12-15 |
| Phase | Phase 3 |
What the registry record for NCT03974113 still lists
NCT03974113 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 407-participant average among 9 other Hemophilia trials with a reported enrollment target (92% 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.
NCT03974113 lists 16 locations in 7 states (Other, Ontario, California).
Frequently Asked Questions
What is clinical trial NCT03974113 about?
NCT03974113 is a clinical study titled "Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B". Primary Objective: \- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B Secondary Objectives: * To characterize the safety and tolerability * To determine fitusiran plasma concentrations at ...
What is the current status of trial NCT03974113?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 32 participants. The study started on 2020-01-28. Estimated completion is 2026-12-15.
What conditions does trial NCT03974113 study?
This clinical trial studies the following conditions: Hemophilia.
What interventions are being tested in trial NCT03974113?
The interventions under investigation include: Fitusiran (DRUG).
Who is sponsoring clinical trial NCT03974113?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03974113 being conducted?
This trial has 16 study locations across California, New Jersey, Ohio, Pennsylvania, Ontario. 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 NCT03974113's enrollment target sits among peer trials
32 8th of 9 higher than 2 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
RECRUITING · 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
ACTIVE NOT RECRUITING · Phase 3
-
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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-
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The Use of Cyclokapron for Treatment and Management of Women With Bleeding Disorders
COMPLETED · NA
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