CompletedPhase 1/2

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

United States, Bulgaria, Russia34 participantsStarted 2015-09-18

Plain-language summary

Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: * To investigate the long-term efficacy of fitusiran * To characterize the safety and efficacy of concomitantly administered Factor VIII (FVIII), Factor IX (FIX) or bypassing agents (BPA) and fitusiran for treatment of bleeding episodes * To assess changes in health-related quality of life (QOL) over time * To characterize antithrombin (AT) reduction and thrombin generation (TG) increase * To characterize the pharmacokinetics (PK) of fitusiran

Who can participate

Age range18 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Completed and tolerated study drug dosing in study TDR14767 (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% * Willing and able to comply with the study requirements and provide written informed consent Exclusion Criteria: * Clinically significant liver disease * Patients known to be human immunodeficiency virus seropositive and have a CD4 count \<200 cells/μL * 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 * 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

What they're measuring

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)

Timeframe: From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months)

Number of Participants With Potentially Clinically Significant Abnormality (PCSA): Hematology

Timeframe: From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months)

Number of Participants With Potentially Clinically Significant Abnormality: Clinical Chemistry

Timeframe: From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months)

Number of Participants With Potentially Clinically Significant Abnormality: Urinalysis

Timeframe: From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months)

Number of Participants With Potentially Clinically Significant Abnormality: Vital Signs

Timeframe: From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months)

Number of Participants With Potentially Clinically Significant Abnormality: Electrocardiogram (ECG)

Timeframe: From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months)

Trial details

NCT IDNCT02554773

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-03-21

Results submitted2024-03-14

View on ClinicalTrials.gov More Hemophilia A trials