Safety and Efficacy of 3 Different Doses of Long Acting Factor VII in Haemophilia A or B Patients With Inhibitors

NCT ID: NCT00951405

Last Updated: 2018-09-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-09-01

Study Completion Date

2011-03-29

Brief Summary

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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.

Detailed Description

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Conditions

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Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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A

Group Type EXPERIMENTAL

activated recombinant human factor VII, long acting

Intervention Type DRUG

After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 25 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months

B

Group Type EXPERIMENTAL

activated recombinant human factor VII, long acting

Intervention Type DRUG

After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 100 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months

C

Group Type EXPERIMENTAL

activated recombinant human factor VII, long acting

Intervention Type DRUG

After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 200 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months

Interventions

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activated recombinant human factor VII, long acting

After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 25 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months

Intervention Type DRUG

activated recombinant human factor VII, long acting

After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 100 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months

Intervention Type DRUG

activated recombinant human factor VII, long acting

After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 200 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months

Intervention Type DRUG

Other Intervention Names

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NN7128 LA-rFVIIa NN7128 LA-rFVIIa NN7128 LA-rFVIIa

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Novo Nordisk Investigational Site

Little Rock, Arkansas, United States

Site Status

Novo Nordisk Investigational Site

Los Angeles, California, United States

Site Status

Novo Nordisk Investigational Site

Orange, California, United States

Site Status

Novo Nordisk Investigational Site

Tampa, Florida, United States

Site Status

Novo Nordisk Investigational Site

Boston, Massachusetts, United States

Site Status

Novo Nordisk Investigational Site

Portland, Oregon, United States

Site Status

Novo Nordisk Investigational Site

Hershey, Pennsylvania, United States

Site Status

Novo Nordisk Investigational Site

Rio de Janeiro, , Brazil

Site Status

Novo Nordisk Investigational Site

Le Kremlin-Bicêtre, , France

Site Status

Novo Nordisk Investigational Site

Paris, , France

Site Status

Novo Nordisk Investigational Site

Kashihara-shi, Nara, , Japan

Site Status

Novo Nordisk Investigational Site

Kitakyusyu, Fukuoka, , Japan

Site Status

Novo Nordisk Investigational Site

Nishinomiya-shi, , Japan

Site Status

Novo Nordisk Investigational Site

Shinjuku-ku, Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Kuala Lumpur, , Malaysia

Site Status

Novo Nordisk Investigational Site

Belgrade, , Serbia and Montenegro

Site Status

Novo Nordisk Investigational Site

Belgrade, , Serbia

Site Status

Novo Nordisk Investigational Site

Parktown Johannesburg, Gauteng, South Africa

Site Status

Novo Nordisk Investigational Site

Durban, KwaZulu-Natal, South Africa

Site Status

Novo Nordisk Investigational Site

Malmo, , Sweden

Site Status

Novo Nordisk Investigational Site

Ankara, , Turkey (Türkiye)

Site Status

Novo Nordisk Investigational Site

Istanbul, , Turkey (Türkiye)

Site Status

Novo Nordisk Investigational Site

London, , United Kingdom

Site Status

Novo Nordisk Investigational Site

London, , United Kingdom

Site Status

Novo Nordisk Investigational Site

Oxford, , United Kingdom

Site Status

Countries

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United States Brazil France Japan Malaysia Serbia and Montenegro Serbia South Africa Sweden Turkey (Türkiye) United Kingdom

References

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Ljung R, Karim FA, Saxena K, Suzuki T, Arkhammar P, Rosholm A, Giangrande P; Pioneer1 Investigators. 40K glycoPEGylated, recombinant FVIIa: 3-month, double-blind, randomized trial of safety, pharmacokinetics and preliminary efficacy in hemophilia patients with inhibitors. J Thromb Haemost. 2013 Jul;11(7):1260-8. doi: 10.1111/jth.12237.

Reference Type DERIVED
PMID: 23578227 (View on PubMed)

Related Links

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http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

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2008-006424-54

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

JapicCTI-090860

Identifier Type: REGISTRY

Identifier Source: secondary_id

U1111-1111-8584

Identifier Type: OTHER

Identifier Source: secondary_id

NN7128-1907

Identifier Type: -

Identifier Source: org_study_id

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