Congenital Bleeding Disorder Clinical Trial
Official title:
An Exploratory Multi-Centre, Multi-National, Randomised, Double Blinded, Parallel Arm Trial Evaluating Safety, Pharmacokinetics and Dose-finding of Prophylactic Administration of Long Acting rFVIIa (LA-rFVIIa) in Haemophilia A or B Patients With Inhibitors
Verified date | September 2018 |
Source | Novo Nordisk A/S |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This trial is conducted in Asia, Europe, Japan and North America. The aim of this clinical trial is to investigate the safety and the efficacy of a prophylactic treatment option with long acting coagulation factor VII (LA-rFVIIa) for haemophilia patients with inhibitors.
Status | Completed |
Enrollment | 23 |
Est. completion date | March 29, 2011 |
Est. primary completion date | March 29, 2011 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 12 Years to 65 Years |
Eligibility |
Inclusion Criteria: - Male haemophilia A or B patients with inhibitors - Willing to undergo a bleeding preventive regimen of 3 months' duration and a total trial length of approximately 8 months - Historical or ongoing high titre inhibitor (more than or equal to 5 BU) based on either medical records, laboratory report reviews, patient and/or care provider interviews - At least 2 bleeding episodes requiring bypassing haemostatic-drug-based treatment within the last month or 12 bleeding episodes within the last 6 months prior to observation period - Body weight between 30 and 100 kg (both inclusive) Exclusion Criteria: - Body Mass Index (BMI) above 30 kg/m2 - Immune tolerance induction therapy within the last month prior to entering observation phase period - Known active pseudo tumours - Platelet count less than 50,000 platelets/microL (based on local laboratory value at screening visit) - Congenital or acquired coagulation disorders other than haemophilia A or B - Surgery within one month prior to the observation period. Catheter, stents and dental extractions do not count as surgeries, i.e. they will not exclude the patient. Port insertion is classified as surgery - Scheduled major and/or orthopaedic surgery, during the trial period until Follow up visit. Catheter, stents and dental extractions do not count as surgeries and will not exclude the patient. Port insertion is classified as surgery - Advanced atherosclerotic disease (i.e. known history of ischemic heart disease, or ischemic stroke) - Any clinical signs or known history of thromboembolic events incl. known deep vein thrombosis (DVT) - Known or clinically suspected allergy to activated recombinant human factor VII (NovoSeven®/NovoSeven RT®/Niastase®) - Prothrombin Time (PT) prolongation (30% above normal limits, or more than 5 seconds compared to control or International Normalised Range (INR) more than 1.7 as defined by local laboratory ranges at screening visit - Severe liver disease (ALAT more than 4 times of the upper limit of normal reference range) (as defined by local laboratory ranges) within a year of enrolment or at the screening - Clinical signs of renal dysfunction (dialysis) and/or creatinine levels more than or equal to 20% above upper normal limit (according to local laboratory range at the screening visit) - Dosing of any investigational drug within the last 30 days prior to the present trial - Any disease or condition which, according to the investigator's judgement, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome - HIV positive patients who either have low CD4+ lymphocyte count ( 200/microL or less based on medical records within 6 months or laboratory screening at screening visit), or who are HCV-PCR positive (based on medical records), or who both have low CD4+ lymphocyte count (200/microL or less) and are HCV-PCR positive. If HCV-PCR testing is not locally available, a HIV positive patient who is HCV antibody positive cannot be included - Need to use other PEGylated pharmaceutical drug during the trial period |
Country | Name | City | State |
---|---|---|---|
Brazil | Novo Nordisk Investigational Site | Rio de Janeiro | |
Former Serbia and Montenegro | Novo Nordisk Investigational Site | Belgrade | |
France | Novo Nordisk Investigational Site | Le Kremlin Bicetre | |
France | Novo Nordisk Investigational Site | Paris | |
Japan | Novo Nordisk Investigational Site | Kashihara-shi, Nara | |
Japan | Novo Nordisk Investigational Site | Kitakyusyu, Fukuoka | |
Japan | Novo Nordisk Investigational Site | Nishinomiya-shi | |
Japan | Novo Nordisk Investigational Site | Shinjuku-ku, Tokyo | |
Malaysia | Novo Nordisk Investigational Site | Kuala Lumpur | |
Serbia | Novo Nordisk Investigational Site | Belgrade | |
South Africa | Novo Nordisk Investigational Site | Durban | KwaZulu-Natal |
South Africa | Novo Nordisk Investigational Site | Parktown Johannesburg | Gauteng |
Sweden | Novo Nordisk Investigational Site | Malmö | |
Turkey | Novo Nordisk Investigational Site | Ankara | |
Turkey | Novo Nordisk Investigational Site | Istanbul | |
United Kingdom | Novo Nordisk Investigational Site | London | |
United Kingdom | Novo Nordisk Investigational Site | London | |
United Kingdom | Novo Nordisk Investigational Site | Oxford | |
United States | Novo Nordisk Investigational Site | Boston | Massachusetts |
United States | Novo Nordisk Investigational Site | Hershey | Pennsylvania |
United States | Novo Nordisk Investigational Site | Little Rock | Arkansas |
United States | Novo Nordisk Investigational Site | Los Angeles | California |
United States | Novo Nordisk Investigational Site | Orange | California |
United States | Novo Nordisk Investigational Site | Portland | Oregon |
United States | Novo Nordisk Investigational Site | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
Novo Nordisk A/S |
United States, Brazil, Former Serbia and Montenegro, France, Japan, Malaysia, Serbia, South Africa, Sweden, Turkey, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Thrombogenecity | at all scheduled visits (1 - 9) | ||
Primary | Immunogenecity: Neutralising Antibody Development | at all scheduled visits (1 - 9) | ||
Secondary | AUC(0-48h) and AUC: Area under the FVIIa activity-time profile in the given time period, which is a measure of total blood exposure | at visit 2 and visit 7 until 48 hours after trial product administration | ||
Secondary | Annualized bleeding rates | During observation period; from visit 1 until visit 2 and treatment period; from visit 2 until visit 7. In total a period of 6 to 8 months |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00978380 -
Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
|
Phase 3 | |
Completed |
NCT02568202 -
Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S)
|
N/A | |
Completed |
NCT01949792 -
A Trial Investigating the Pharmacokinetics and Pharmacodynamics of rFVIIa in Patients With Haemophilia A or B With or Without Inhibitors
|
Phase 1 | |
Completed |
NCT01205724 -
Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A
|
Phase 1 | |
Completed |
NCT01562587 -
Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State
|
Phase 1 | |
Completed |
NCT00108797 -
Trial of NovoSeven® in Haemophilia - Joint Bleeds
|
Phase 4 | |
Completed |
NCT01493778 -
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
|
Phase 3 | |
Completed |
NCT02490787 -
Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects
|
Phase 1 | |
Completed |
NCT01876745 -
A Study to Evaluate Safety and Efficacy of NovoSeven® in Patients With Glanzmann's Thrombasthenia in Japan
|
N/A | |
Completed |
NCT02920398 -
A Multi-centre, Comparative, Double Blind, Randomised Cross-over Trial Investigating Single Dose Pharmacokinetics and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia A
|
Phase 1 | |
Completed |
NCT00984126 -
Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015
|
Phase 3 | |
Completed |
NCT01228669 -
Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B
|
Phase 1 | |
Completed |
NCT01988532 -
Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia
|
N/A | |
Completed |
NCT01234545 -
Observational Study Describing the Usual Clinical Practice Use of NovoSeven® in the Home Treatment of Joint Bleeds in Patients With Haemophilia A or B and Inhibitors
|
N/A | |
Completed |
NCT01779921 -
Treatment of Congenital Factor VII Deficiency
|
N/A | |
Completed |
NCT01563471 -
Safety and Tolerability of Intravenous Doses of Activated Recombinant Human Factor VII in Healthy Volunteers
|
Phase 1 | |
Completed |
NCT02941354 -
Evaluating the Pharmacokinetics of NovoEight® (Turoctocog Alfa) in Relation to BMI in Subjects With Haemophilia A
|
Phase 1 | |
Completed |
NCT02241694 -
To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily Lives
|
N/A | |
Completed |
NCT01230021 -
Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency
|
Phase 3 | |
Completed |
NCT01238367 -
A Single Dose Trial of Recombinant Factor VIII (N8) in Japanese Subjects With Haemophilia A: An Extension to Trial NN7008-3543
|
Phase 1 |