NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

CompletedPhase 3

Sponsor: Genzyme, a Sanofi Company

Conditions: Hemophilia A, Hemophilia B

Interventions: fitusiran, Bypassing agents

Countries: United States, Australia, Canada, China, France, Germany, India, Italy

The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

Eligibility overview

Sex: MALE

Age: 12 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

Inclusion Criteria:

* Males, greater than or equal to (\>=) 12 years of age.
* Severe hemophilia A or B with inhibitors.

  * (Severity confirmed by a central laboratory where coagulation factor VIII (FVIII) level was less than (\<)1% or factor IX (FIX) level was less than or equal to \[\<=\]2% at Screening; Inhibitors defined as inhibitor titer of \>=0.6 Bethesda units per milliliter \[BU/mL\] or as evidenced by medical records).
* A minimum of 6 bleeding episodes requiring BPA treatment within the last 6 months prior to screening.
* Willing and able to comply with the study requirements and to provide written informed consent and assent.

Exclusion Criteria:

* Known co-existing bleeding disorders other than hemophilia A or B.
* Antithrombin (AT) activity \<60% at Screening.
* Co-existing thrombophilic disorder.
* Clinically significant liver disease.
* Active hepatitis C virus infection.
* HIV positive with a cluster of differentiation-4 count of \<200 cells/microliter.
* History of arterial or venous thromboembolism.
* Inadequate renal function.
* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine.
* History of intolerance to SC injection(s).
* Any other conditions or comorbidities that would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement.

Locations (58)

Phoenix, Arizona, United States
Los Angeles, California, United States
Orange, California, United States
San Diego, California, United States
Gainesville, Florida, United States
Jacksonville, Florida, United States
Miami, Florida, United States
Tampa, Florida, United States
New Orleans, Louisiana, United States
Baltimore, Maryland, United States
Las Vegas, Nevada, United States
Philadelphia, Pennsylvania, United States
Camperdown, Australia
Clayton, Australia
Montreal, Canada
Beijing, China
Guangzhou, China
Hangzhou, China
Shanghai, China
Tianjin, China
Lyon, France
Rouen, France
Frankfurt am Main, Germany
Leipzig, Germany
Bangalore, India
India, India
Jaipur, India
Lucknow, India
Pune, India
Pune, India
+ 28 more on CT.gov