Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773 (paradigm™ 4)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01395810
First received: June 30, 2011
Last updated: September 3, 2014
Last verified: September 2014
  Purpose

This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of NNC-0156-0000-0009 after long-term exposure in patients with haemophilia B.

This trial is an extension to trials NN7999-3747 (NCT01333111/paradigm™ 2) and NN7999-3773 (NCT01386528/paradigm™ 3).


Condition Intervention Phase
Congenital Bleeding Disorder
Haemophilia B
Drug: NNC-0156-0000-0009
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • Incidence of inhibitory antibodies against FIX defined as titre above or equal to 0.6 BU (Bethesda Units) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion (up to 2 years) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Haemostatic effect of NNC-0156-0000-0009 when used for treatment of bleeding episodes, assessed as success/failure based on a four-point scale for haemostatic response (excellent, good moderate, poor) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion (up to 2 years) ] [ Designated as safety issue: No ]
  • Number of bleeding episodes during routine prophylaxis [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion (up to 2 years) ] [ Designated as safety issue: No ]
  • FIX trough levels [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion (up to 2 years) ] [ Designated as safety issue: No ]
  • Incidence of Adverse Events (AEs) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion (up to 2 years) ] [ Designated as safety issue: No ]
  • Incidence of Serious Adverse Events (SAEs) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion (up to 2 years) ] [ Designated as safety issue: No ]

Enrollment: 71
Study Start Date: April 2012
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prophylaxis, high dose (once weekly) Drug: NNC-0156-0000-0009
One single dose administered intravenously (into the vein) once weekly. Patients will receive instruction on how to treat any bleeding episode they may experience.
Experimental: Prophylaxis, low dose (once weekly) Drug: NNC-0156-0000-0009
One single dose administered intravenously (into the vein) once weekly. Patients will receive instruction on how to treat any bleeding episode they may experience.
Experimental: On-demand Drug: NNC-0156-0000-0009
One single dose administered intravenously (into the vein). Patients will treat themselves with either a low or a high dose dependent on the severity of the bleeding episode.
Experimental: Prophylaxis, high dose (every second week) Drug: NNC-0156-0000-0009
One single dose administered intravenously (into the vein) every second week. Patients will receive instruction on how to treat any bleeding episode they may experience.

  Eligibility

Ages Eligible for Study:   13 Years to 70 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Previous participation in NN7999-3747 (NCT01333111) and/or NN7999-3773

Exclusion Criteria:

  • Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
  • Current FIX inhibitors above or equal to 0.6 BU (Bethesda Units)
  • Congenital or acquired coagulation disorders other than haemophilia B
  • Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
  • Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's (trial physician) judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01395810

  Show 28 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
No publications provided

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01395810     History of Changes
Other Study ID Numbers: NN7999-3775, 2010-023072-17, U1111-1121-5408, JapicCTI-121812
Study First Received: June 30, 2011
Last Updated: September 3, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Vaccines and Biomedicines; Paul-Ehrlich-Institut
Greece: National Organization of Medicines
Italy: AIFA, National Medicines Agency
Japan: Ministry of Health, Labour and Welfare (MHLW)
Macedonia, The Former Yugoslav Republic of: Ministry of Health of Republic of Macedonia
Malaysia: Drug Control Authority (DCA)
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Romania: National Medicines Agency
Russia: Ministry of Health of the Russian Federation
South Africa: Medicines Control Council
Taiwan: Department of Health
Thailand: THFDA
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders
Hemostatic Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on October 01, 2014