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Clinical Trials in India / NCT03754790
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 · tracked via the Priya Life Science India tracker
Sponsor
Genzyme, a Sanofi Company
Phase
Phase 3
Started
2019-01-09
Last updated
2025-12-17

Condition(s) studied

Hemophilia

Investigational drug(s) / intervention(s)

Fitusiran

Fitusiran: Pharmaceutical form:solution for injection Route of administration: subcutaneous

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

Eligibility

Sex
MALE
Min age
12 Years
Max age
Healthy volunteers
No
Inclusion criteria : * Participant must be at least 12 years of age inclusive, at the time of signing the informed consent * Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial * Male * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative Exclusion criteria: * Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis * Current participation in immune tolerance induction treatment (ITI) * Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period * Use of compounds other than factor concentrates or BPAs for hemophilia treatment * Current or prior participation in a gene therapy trial * Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) \>1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST \> 5 x ULN for patients who were in the fitusiran arm in the parent study * Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort: * Clinically significant liver disease * History of arterial or venous thromboembolism The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Primary outcome measure(s)

Trial sites (79)

FacilityCityRegionStatus
Phoenix Childrens Hospital Site Number : 8400009 Phoenix Arizona
Children's Hospital Los Angeles Site Number : 8400019 Los Angeles California
Center for Inherited Blood Disorders (CIBD) Site Number : 8400016 Orange California
Nemours Children's Clinic Site Number : 8400008 Jacksonville Florida
St Joseph's Children's Hospital of Tampa Site Number : 8400002 Tampa Florida
Rush University Medical Center -1725 W Harrison St Site Number : 8400001 Chicago Illinois
~Massachusetts General Hospital Site Number : 8400011 Boston Massachusetts
University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012 Ann Arbor Michigan
Alliance for Childhood Diseases Site Number : 8400007 Las Vegas Nevada
Childrens Hospital Medical Center of Akron Site Number : 8400006 Akron Ohio
Investigational Site Number : 0360001 Camperdown New South Wales
Investigational Site Number : 0360003 Prahran Victoria
Investigational Site Number : 0360002 Murdoch Western Australia
Investigational Site Number : 1240001 Montreal Quebec
Investigational Site Number : 1560004 Beijing China
Investigational Site Number : 1560007 Beijing China
Investigational Site Number : 1560014 Changsha China
Investigational Site Number : 1560009 Chengdu China
Investigational Site Number : 1560002 Guangzhou China
Investigational Site Number : 1560011 Guiyang China
Investigational Site Number : 1560005 Hangzhou China
Investigational Site Number : 1560008 Jinan China
Investigational Site Number : 1560012 Kunming China
Investigational Site Number : 1560013 Lanzhou China
Investigational Site Number : 1560010 Qingdao China
Investigational Site Number : 1560003 Shanghai China
Investigational Site Number : 1560006 Suzhou China
Investigational Site Number : 2080001 Copenhagen Denmark
Investigational Site Number : 2500002 Lyon France
Investigational Site Number : 2500003 Paris France
Investigational Site Number : 2760001 Berlin Germany
Investigational Site Number : 2760003 Leipzig Germany
Investigational Site Number : 3480002 Budapest Hungary
Investigational Site Number : 3560001 Bangalore India
Investigational Site Number : 3560005 Lucknow India
Investigational Site Number : 3560007 Mumbai India
Investigational Site Number : 3560002 Pune India
Investigational Site Number : 3560010 Pune India
Investigational Site Number : 3560004 Ranipet India
Investigational Site Number : 3720001 Crumlin Dublin

+ 39 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03754790 on ClinicalTrials.gov ↗ ← All trials in India