Have you or your loved ones been diagnosed with congenital bleeding disorder?

You may be eligible to participate in a congenital bleeding disorder clinical trial.

Have you or your loved ones been diagnosed with congenital bleeding disorder? You may be eligible to participate in a congenital bleeding disorder clinical trial.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Congenital Bleeding Disorder Clinical Trial

NCT02141074 | Phase 3 | Interventional

Have you or your loved ones been diagnosed with congenital bleeding disorder?

You may be eligible to participate in a congenital bleeding disorder clinical trial.

Have you or your loved ones been diagnosed with congenital bleeding disorder? You may be eligible to participate in a congenital bleeding disorder clinical trial.

Completed

Male

Up to 6

Years old

This study is looking to recruit 60 Participants

This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

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Details for the study

Brief Title

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

Official Title

An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)

Brief Summary

This trial is conducted globally. The aim of the trial is to investigate the safety and <br /> efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

Treatments and/or Procedures

Nonacog beta pegol

For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.

Study Criteria

Inclusion Criteria:

  -  Informed consent obtained before any trial-related activities. Trial-related
     activities are any procedures that are carried out as part of the trial, including
     activities to determine suitability for the trial

  -  Male, age below 6 years at the time of signing informed consent

  -  Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity
     level below or equal to 2%) based on medical records or central laboratory results

  -  Previously untreated or exposed to FIX containing products less than or equal to 3
     exposure days (5 previous exposures to blood components is acceptable)

Exclusion Criteria:

  -  Any history of FIX inhibitors (defined by medical records)

  -  Known or suspected hypersensitivity to trial product or related products

  -  Previous participation in this trial. Participation is defined as first dose
     administered of trial product

  -  Receipt of any investigational medicinal product within 30 days before screening

  -  Congenital or acquired coagulation disorder other than haemophilia B

  -  Any chronic disorder or severe disease which, in the opinion of the Investigator,
     might jeopardise the patient's safety or compliance with the protocol

  -  Patient's parent(s)/LAR(s) (legally acceptable representative) mental incapacity,
     unwillingness to cooperate, or a language barrier precluding adequate understanding
     and cooperation