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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02554773
Recruitment Status : Completed
First Posted : September 18, 2015
Last Update Posted : April 4, 2023
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief 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

Condition or disease Intervention/treatment Phase
Hemophilia A Hemophilia B Drug: Fitusiran (SAR439774) Phase 1 Phase 2

Detailed Description:
It is anticipated that patients in this study will receive treatment with open label fitusiran for approximately 7 years or until fitusiran becomes commercially available, whichever occurs first.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study of Subcutaneously Administered Fitusiran in Patients With Moderate or Severe Hemophilia A or B Who Have Participated in a Previous Clinical Study With Fitusiran
Actual Study Start Date : September 18, 2015
Actual Primary Completion Date : March 21, 2023
Actual Study Completion Date : March 21, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Fitusiran
Patients will be administered subcutaneous (SC) fitusiran once monthly or every 2 months according to the dose selection rules defined in protocol.
 Drug: Fitusiran (SAR439774)
Pharmaceutical form: solution for injection Route of administration : subcutaneous (sc)


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation [ Time Frame: Up to 7 years ]
    Incidence of treatment-emergent adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation


Secondary Outcome Measures :
  1. Annualized bleed rate (ABR) [ Time Frame: Up to 7 years ]
  2. Time intervals between bleeding episodes [ Time Frame: Up to 7 years ]
  3. Weight-adjusted consumption of FVIII, FIX, or BPA [ Time Frame: Up to 7 years ]
    BPA Assessment of concomitantly administered factor VIII (FVIII), factor IX (FIX), or bypassing agents (BPA) and fitusiran for treatment of bleeding

  4. Changes in health-related quality of life (QOL) with long-term dosing of fitusiran [ Time Frame: Up to 7 years ]
    QOL assessed by an EQ-5D questionnaire and HAEM-A-QoL

  5. Pharmacokinetics (PK) of fitusiran: Cmax [ Time Frame: Day 1, Month 12, Month 24 ]
  6. Pharmacokinetics (PK) of fitusiran: AUC [ Time Frame: Day 1, Month 12, Month 24 ]
  7. Pharmacokinetics (PK) of fitusiran: t1/2 [ Time Frame: Day 1, Month 12, Month 24 ]
  8. Pharmacokinetics (PK) of fitusiran: CL/F [ Time Frame: Day 1, Month 12, Month 24 ]
  9. Pharmacokinetics (PK) of fitusiran: V/F [ Time Frame: Day 1, Month 12, Month 24 ]
  10. The effect of fitusiran on plasma levels of antithrombin (AT) and thrombin generation (TG) [ Time Frame: Up to 7 years ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02554773


Locations
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United States, Michigan
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
United States, Pennsylvania
Clinical Trial Site
Pittsburgh, Pennsylvania, United States
Bulgaria
Clinical Trial Site
Plovdiv, Bulgaria
Clinical Trial Site
Sofia, Bulgaria
Russian Federation
Clinical Trial Site
Kirov, Russian Federation
Clinical Trial Site
Moscow, Russian Federation
Switzerland
Clinical Trial Site
Zurich, Switzerland
United Kingdom
Clinical Trial Site
Basingstoke, United Kingdom
Clinical Trial Site
Glasgow, United Kingdom
Clinical Trial Site
London, United Kingdom, NW3 2QG
Clinical Trial Site
London, United Kingdom, SW17 0QT
Clinical Trial Site
Truro, United Kingdom
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Clinical Sciences & Operations, MD Sanofi
More Information
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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02554773    
Other Study ID Numbers: LTE14762
ALN-AT3SC-002 ( Other Identifier: Alnylam )
2015-001395-21 ( EudraCT Number )
U1111-1251-5204 ( Registry Identifier: ICTRP )
First Posted: September 18, 2015    Key Record Dates
Last Update Posted: April 4, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
Hemophilia
RNAi therapeutic
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked