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RNAi Therapeutics

Fitusiran (SAR439774) for Hemophilia A

Phase 1 & 2

Waitlist Available

Research Sponsored by Genzyme, a Sanofi Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 7 years

Awards & highlights

Study Summary

This trial is investigating the long-term safety and efficacy of fitusiran, a new drug for hemophilia A or B. The objectives are to assess safety, investigate efficacy, and study how the drug affects quality of life, clotting function, and thrombin generation.

Eligible Conditions

Hemophilia A
Hemophilia B

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 7 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 7 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 7 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation

Secondary outcome measures

Annualized bleed rate (ABR)

Changes in health-related quality of life (QOL) with long-term dosing of fitusiran

Pharmacokinetics (PK) of fitusiran: AUC

+7 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: FitusiranExperimental Treatment1 Intervention

Patients will be administered subcutaneous (SC) fitusiran once monthly or every 2 months according to the dose selection rules defined in protocol.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fitusiran (SAR439774)

2015

Completed Phase 2

~40

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Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor

524 Previous Clinical Trials

85,592 Total Patients Enrolled

6 Trials studying Hemophilia A

586 Patients Enrolled for Hemophilia A

Clinical Sciences & Operations, MDStudy DirectorSanofi

4 Previous Clinical Trials

203 Total Patients Enrolled

2 Trials studying Hemophilia A

180 Patients Enrolled for Hemophilia A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants is this experiment recruiting at maximum?

"At the moment, this clinical trial is not accepting applications. The study was first published on September 18th 2015 and last updated on August 4th 2022. If you are searching for alternative trials, there are currently 89 studies recruiting patients with hemophilia A and 3 trials for Fitusiran (SAR439774) enrolling participants."

Answered by AI

Is there any availability for participants in this experiment?

"According to information published on clinicaltrials.gov, this medical trial has ended its recruitment efforts since the last update made on August 4th 2022. Initially posted in September 2015, it is not longer accepting applicants; however there are still 92 other studies actively searching for participants."

Answered by AI

Is this an original research study?

"Research into Fitusiran (SAR439774) began in 2015, with the first trial sponsored by Genzyme. Subsequent Phase 1 and 2 trials confirmed its efficacy, leading to 3 simultaneous research projects being conducted in 24 countries across 34 cities."

Answered by AI

What other investigations have been conducted with regards to Fitusiran (SAR439774)?

"Currently, there are 3 research initiatives in progress for Fitusiran (SAR439774), two of which have arrived at Phase 3. Manchester, Michigan is hosting many of the studies but overall 184 different medical sites worldwide are running clinical trials related to this medication."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2015. "An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02554773.