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Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients (paradigm™ 2)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01333111
First received: April 8, 2011
Last updated: April 15, 2014
Last verified: April 2014
  Purpose

This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of NNC-0156-0000-0009 when used for treatment and prophylaxis of bleeding episodes in patients with haemophilia B.


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

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title: A Multi-centre, Single-blind Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC-0156-0000-0009 When Used for Treatment and Prophylaxis of Bleeding Episodes 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 factor IX defined as titre equal to or above 0.6 BU (Bethesda Units) [ Time Frame: 52 weeks after treatment start for patients on prophylaxis ] [ Designated as safety issue: No ]
  • Incidence of inhibitory antibodies against factor IX defined as titre equal to or above 0.6 BU (Bethesda Units) [ Time Frame: 28 weeks after treatment start on on-demand treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Haemostatic effect of NNC-0156-0000-0009 when used for prophylaxis of bleeding episodes, assessed as success/failure based on a four-point scale for haemostatic response [ Time Frame: 52 weeks after treatment start for patients on prophylaxis ] [ Designated as safety issue: No ]
  • 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 [ Time Frame: 28 weeks after treatment start on on-demand treatment ] [ Designated as safety issue: No ]
  • Number of bleeding episodes per patient during routine prophylaxis [ Time Frame: 52 weeks after treatment start for patients on prophylaxis ] [ Designated as safety issue: No ]
  • Factor IX trough levels [ Time Frame: 52 weeks after treatment start for patients on prophylaxis ] [ Designated as safety issue: No ]
  • Incidence of adverse events (AEs) [ Time Frame: at 56 weeks ±2 weeks for patients on prophylaxis ] [ Designated as safety issue: No ]
  • Incidence of adverse events (AEs) [ Time Frame: at 32 weeks ±2 weeks for patients on on-demand treatment ] [ Designated as safety issue: No ]
  • Incidence of serious adverse events (SAEs) [ Time Frame: at 56 weeks ±2 weeks for patients on prophylaxis ] [ Designated as safety issue: No ]
  • Incidence of serious adverse events (SAEs) [ Time Frame: at 32 weeks ±2 weeks for patients on on-demand treatment ] [ Designated as safety issue: No ]
  • Host Cell Proteins (HCP) antibodies [ Time Frame: 52 weeks after treatment start for patients on prophylaxis ] [ Designated as safety issue: No ]
  • Host Cell Proteins (HCP) antibodies [ Time Frame: 28 weeks after treatment start on on-demand treatment ] [ Designated as safety issue: No ]

Enrollment: 74
Study Start Date: April 2011
Study Completion Date: April 2013
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prophylaxis, high dose (trial duration 52 weeks) 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 (trial duration 52 weeks) 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 (trial duration 28 weeks) Drug: NNC-0156-0000-0009
Patients will treat themselves with either a low or a high dose dependent on the severity of the bleeding episode

  Eligibility

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

Inclusion Criteria:

  • Male patients with moderately severe or severe congenital haemophilia B with a factor IX activity of 2% or below according to medical records
  • History of at least 150 exposure days to other factor IX products
  • Patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months, or patients currently on prophylaxis

Exclusion Criteria:

  • Known history of factor IX inhibitors based on existing medical records, laboratory report reviews and patient and legally acceptable representative (LAR) interviews
  • HIV (Human immunodeficiency virus) positive, with a viral load equal to or above 400,000 copies/mL and/or CD4+ lymphocyte count equal to or below 200/microL
  • 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)
  • Immune modulating or chemotherapeutic medication
  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: NCT01333111

  Show 25 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: NCT01333111     History of Changes
Other Study ID Numbers: NN7999-3747, 2010-023069-24, U1111-1119-6415, JapicCTI-111644
Study First Received: April 8, 2011
Last Updated: April 15, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medicinal Devices (BfarM)
Hungary: Ministry of Health, Social and Family Affairs
Italy: The Italian Medicines Agency
Japan: Ministry of Health, Labor and Welfare
Macedonia, The Former Yugoslav Republic of: Ministry of Health of Republic of Macedonia
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Russia: Federal Service for Control of Health Care and Social Development
South Africa: Medicines Control Council
Thailand: Thai FDA
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Regulatory Authority (MHRA)
United States: Food and Drug Administration
Malaysia: Ministry of Health

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

ClinicalTrials.gov processed this record on November 27, 2014