An Extension Study in Patients with Moderate or Severe Hemophilia A or B who have Participated in a Previous Clinical Study with Fitusira

Phase 1

• Conditions: Hemophilia A or Hemophilia B; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]; MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850

• Registration Number: EUCTR2015-001395-21-GB
• Lead Sponsor: Genzyme Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-07-17
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 48

Inclusion Criteria

Completed and tolerated study drug dosing in study ALN-AT3SC-001.
Male aged =18 years.
Moderate or severe, clinically stable hemophilia A or B as evidenced by a laboratory FVIII or FIX level =5% at screening. Patients with a FVIII or FIX level >5% at screening will be eligible on provision of a historic laboratory report indicating a trough level =5%.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 44
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion Criteria

Liver disease defined as clinically significant cirrhosis as determined by the Investigator, INR >1.5 at Screening, ALT/AST >3× upper limit of the normal reference range (ULN), platelet count =120,000/mL and/or other complete blood count test results that are considered clinically significant and unacceptable by the Investigator, or known hepatitis C virus currently requiring treatment with ribavirin or interferon.
Patients known to be human immunodeficiency virus seropositive and have a CD4 count <200 cells/µL at screening.
History of venous thromboembolism.
Current serious mental illness that, in the judgment of the Investigator, may compromise patient safety, ability to participate in all study assessments, or study integrity.
Uncontrolled hypertension.
Clinically relevant history or presence of cardiovascular, respiratory, gastrointestinal, renal, neurological, inflammatory, or other diseases that, in the judgment of the Investigator, precludes study participation.
If using nonsteroidal anti-inflammatory drugs intermittently or chronically, must tolerate them with no previous side effects.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified