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An Open-label Extension Study of an Investigational Drug, ALN-AT3SC, in Patients With Moderate or Severe Hemophilia A or B

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ClinicalTrials.gov Identifier: NCT02554773
Recruitment Status : Active, not recruiting
First Posted : September 18, 2015
Last Update Posted : August 10, 2018
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:
The purpose of this study is to evaluate the long-term safety and tolerability of ALN-AT3SC in male patients with moderate or severe hemophilia A or B.

Condition or disease Intervention/treatment Phase
Hemophilia A Hemophilia B Drug: ALN-AT3SC Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 37 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study of Subcutaneously Administered ALN-AT3SC in Patients With Moderate or Severe Hemophilia A or B Who Have Participated in a Previous Clinical Study With ALN-AT3SC
Actual Study Start Date : September 18, 2015
Estimated Primary Completion Date : July 2021
Estimated Study Completion Date : September 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: ALN-AT3SC Drug: ALN-AT3SC
Multiple doses of ALN-AT3SC by subcutaneous (sc) injection




Primary Outcome Measures :
  1. The long-term safety and tolerability of ALN-AT3SC [ Time Frame: Up to 4 years ]
    • Incidence of treatment-emergent adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation


Secondary Outcome Measures :
  1. Long-term efficacy of ALN-AT3SC [ Time Frame: Up to 4 years ]
    • Annualized bleed rate (ABR)

  2. Assessment of concomitantly administered factor VIII (FVIII), factor IX (FIX), or bypassing agents (BPA) and ALN-AT3SC for treatment of bleeding episodes [ Time Frame: Up to 4 years ]
    • Weight-adjusted consumption of FVIII, FIX, or BPA

  3. Changes in health-related quality of life (QOL) with long-term dosing of ALN-AT3SC [ Time Frame: Up to 4 years ]
  4. Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 722 ]
    Cmax

  5. Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 722 ]
    tmax

  6. Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 722 ]
    AUC

  7. Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 722 ]
    t1/2

  8. Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 722 ]
    CL/F

  9. Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 722 ]
    V/F

  10. The effect of ALN-AT3SC on plasma levels of antithrombin (AT) and thrombin generation (TG) [ Time Frame: Up to 4 years ]


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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
  • 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

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
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
Clinical Trial Site
Varna, 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, SE1 1YR
Clinical Trial Site
London, United Kingdom, SW17 0QT
Clinical Trial Site
Manchester, United Kingdom
Clinical Trial Site
Truro, United Kingdom
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations, MD Sanofi

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02554773     History of Changes
Other Study ID Numbers: LTE14762
ALN-AT3SC-002 ( Other Identifier: Alnylam )
2015-001395-21 ( EudraCT Number )
First Posted: September 18, 2015    Key Record Dates
Last Update Posted: August 10, 2018
Last Verified: June 2018
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://www.clinicalstudydatarequest.com/

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
Hemophilia
RNAi therapeutic

Additional relevant MeSH terms:
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
Factor VIII
Coagulants