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Trial record 1 of 2 for:    ALN-AT3SC
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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

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02554773
First Posted: September 18, 2015
Last Update Posted: September 6, 2017
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.
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
  Purpose
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 Intervention Phase
Hemophilia A Hemophilia B Drug: ALN-AT3SC Phase 1 Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The long-term safety and tolerability of ALN-AT3SC evaluated by the proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation [ Time Frame: Through day 811 ]

Secondary Outcome Measures:
  • Change From Baseline in the Number of Bleeding Events [ Time Frame: Through day 811 ]
  • Change From Baseline in the Amount of Factor VIII (FVIII) or Factor IX (FIX) Administered for the Treatment of Bleeding Episodes (FVIII) or factor IX (FIX) and ALN-AT3SC for treatment of bleeding episodes [ Time Frame: Through day 811 ]
  • Number of Adverse Events in Patients Receiving Concomitantly Administered Factor VIII (FVIII) or Factor IX (FIX) and ALN-AT3SC [ Time Frame: Through day 811 ]
  • The assessment of changes in health-related quality of life (QOL) with long-term dosing of ALN-AT3SC [ Time Frame: Through day 751 ]
  • Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 362 ]
    Cmax

  • Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 362 ]
    tmax

  • Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 362 ]
    AUC

  • Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 362 ]
    t1/2

  • Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 362 ]
    CL/F

  • Profile of Pharmacokinetics (PK) of ALN-AT3SC [ Time Frame: Through day 362 ]
    V/F

  • The effect of ALN-AT3SC on plasma levels of antithrombin (AT) and thrombin generation (TG) [ Time Frame: Through day 811 ]

Estimated Enrollment: 37
Study Start Date: September 2015
Estimated Study Completion Date: September 2021
Estimated Primary Completion Date: July 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ALN-AT3SC Drug: ALN-AT3SC
Multiple doses of ALN-AT3SC by subcutaneous (sc) injection

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

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
  • Agree to use 1 effective method of contraception throughout study participation and until 1 month after administration of the last dose of study drug
  • 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
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, 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
Alnylam Pharmaceuticals
Investigators
Study Director: Kate Madigan, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02554773     History of Changes
Other Study ID Numbers: ALN-AT3SC-002
First Submitted: September 15, 2015
First Posted: September 18, 2015
Last Update Posted: September 6, 2017
Last Verified: September 2017

Keywords provided by Alnylam Pharmaceuticals:
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


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