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ACTIVE NOT 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

NCT03754790 · View on ClinicalTrials.gov ↗

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

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

FieldValue
Enrollment Target 281 participants
Start Date 2019-01-09
Est. Completion 2026-11-05
Phase Phase 3

Sponsor

Genzyme, a Sanofi Company

52 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03754790

The ClinicalTrials.gov registry entry for NCT03754790 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 281 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 52 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03754790 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Other, California, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03754790?

The interventions under investigation include: Fitusiran (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03754790?

This trial is sponsored by Genzyme, a Sanofi Company, which has 52 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial