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NCT03417245 · ClinicalTrials.gov registry record · Phase 3
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
A Phase 3 study of Hemophilia A and Hemophilia B, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 3
- Development phase
- 120
- Enrollment target
- 20
- Study locations
NCT03417245: Completed Phase 3 study of Hemophilia A and Hemophilia B, sponsored by Genzyme, a Sanofi Company.
NCT03417245 is a Phase 3 study of Hemophilia A and Hemophilia B that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 120 participants, above the 89-participant average among 42 other Hemophilia A trials with a reported enrollment target (35% higher). The trial reports 20 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03417245, a Phase 3 study of Hemophilia A and Hemophilia B, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 120 participants
- Enrollment target
- 20
- Study locations
Study Summary
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: * To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by: * The frequency of spontaneous bleeding episodes. * The frequency of joint bleeding episodes. * Health-related quality of life (HRQOL) in participants \>=17 years of age. * To determine the frequency of bleeding episodes during the onset period. * To determine the safety and tolerability of fitusiran.
Primary Outcome
ABR for a participant during efficacy period (EP) was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. Treated Bleeding episode: any occurrence of hemorrhage that required administration of by-passing agents (BPA) or factor. It started from first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at same location or injections less than or equal to (\<=) 72 hours apart were cons
Conditions Studied
Interventions
- DRUG fitusiran
- DRUG factor concentrates
Study Locations (20)
Other
- Investigational Site Number 6101 - Camperdown
- Investigational Site Number 6103 - Murdoch
- Investigational Site Number 6104 - Prahran
- Investigational Site Number 8604 - Beijing
- Investigational Site Number 8602 - Guangzhou
- Investigational Site Number 8605 - Hangzhou
- Investigational Site Number 8603 - Shanghai
- Investigational Site Number 8601 - Tianjin
- Investigational Site Number 4501 - Copenhagen
- Investigational Site Number 3303 - Lyon
- Investigational Site Number 3305 - Paris
- Investigational Site Number 3301 - Rouen
Florida
- Investigational Site Number 128 - Gainesville
- Investigational Site Number 103 - Tampa
Arkansas
- Investigational Site Number 0140 - Little Rock
Illinois
- Investigational Site Number 102 - Chicago
Louisiana
- Investigational Site Number 119 - New Orleans
Michigan
- Investigational Site Number 125 - Ann Arbor
Nevada
- Investigational Site Number 111 - Las Vegas
Ohio
- Investigational Site Number 110 - Akron
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 120 participants |
| Start Date | 2018-03-01 |
| Est. Completion | 2021-07-14 |
| Phase | Phase 3 |
What the finished NCT03417245 record still lists
NCT03417245 is an interventional study that assigns participants to a tested intervention. The registered 120 participants enrollment target is mid-sized for trials with a published cap, above the 89-participant average among 42 other Hemophilia A trials with a reported enrollment target (35% higher).
The record links to 2 conditions, with Hemophilia A appearing as the primary indexed condition, and to 2 interventions - of which fitusiran is the first listed.
NCT03417245 names 20 study sites across 8 states, led by Other, Florida, Arkansas.
Frequently Asked Questions
What is clinical trial NCT03417245 about?
NCT03417245 is a clinical study titled "A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors". Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: * To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as deter...
What is the current status of trial NCT03417245?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 120 participants. The study started on 2018-03-01. Estimated completion is 2021-07-14.
What conditions does trial NCT03417245 study?
This clinical trial studies the following conditions: Hemophilia A, Hemophilia B.
What interventions are being tested in trial NCT03417245?
The interventions under investigation include: fitusiran (DRUG), factor concentrates (DRUG).
Who is sponsoring clinical trial NCT03417245?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03417245 being conducted?
This trial has 20 study locations across Arkansas, Florida, Illinois, Louisiana, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Hemophilia A
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 NCT03417245's enrollment target sits among peer trials
120 10th of 42 higher than 32 of 42 other Hemophilia A trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia A 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
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