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Post-marketing Surveillance (Use Result Surveillance) With Refixia®

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ClinicalTrials.gov Identifier: NCT03875547
Recruitment Status : Not yet recruiting
First Posted : March 14, 2019
Last Update Posted : March 14, 2019
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B. The participants will get Refixia® as prescribed to them by their study doctor. The study will last up to Sep 2025 for the participant. The participants may be asked to fill in the quality of life questionnaires (if they are above age of 15). The blood samples taken from the participants as part of routine clinical practice will also be used to investigate the safety for the long-term use of Refixia®.

Condition or disease Intervention/treatment
Haemophilia B Drug: Refixia®

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Post-marketing Surveillance (Use Result Surveillance) With Refixia®. A Multicentre, Non-interventional Post Marketing Surveillance of Safety and Effectiveness of Refixia® in Routine Clinical Care With Haemophilia B Patients in Japan.
Estimated Study Start Date : March 30, 2019
Estimated Primary Completion Date : September 30, 2025
Estimated Study Completion Date : September 30, 2025

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Patients with haemophilia B
Both patients who have not previously been exposed to Refixia® and patients previously exposed to Refixia® in one of the clinical trials can be included.
Drug: Refixia®
Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician




Primary Outcome Measures :
  1. Number of Adverse Reactions (ARs) [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Count of events


Secondary Outcome Measures :
  1. Number of Serious Adverse Events (SAEs) [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Count of events

  2. Number of Serious Adverse Reactions (SARs) [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Count of events

  3. Number of bleeding episodes as assessed by annualised bleeding rate (ABR) [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Count of bleeding episodes

  4. Number of treatment requiring bleeding episodes as assessed by ABR [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Count of bleeding episodes

  5. Haemostatic response of Refixia® in treatment of bleeds [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Haemostatic response is assessed as success/failure based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure.

  6. Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures [ Time Frame: From baseline (week 0) to end of study (up to 6 years and 10 months) ]
    Haemostatic response is assessed as success/failure based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with haemophilia B
Criteria

Inclusion Criteria:

  • Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
  • The decision to initiate treatment with commercially available Refixia® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. At each site of this study, all patients will be registered consecutively from the first patient after the launch of Refixia® (consecutively registered system).
  • Diagnosis of haemophilia B in males or females, no age limitation. Patients younger than 12 years old will continue to be registered for 3 years of recruitment period regardless of the target number of patients.
  • New patients who have not been previously exposed to Refixia®. Also patients previously exposed to Refixia® in NN7999-3639, -3747, -3774, -3775 or -3895 clinical trial can be enrolled in this study. The patients who have participated in NN7999 -3774 or -3895 clinical trial can be enrolled in this study as continuous cases until 30-September-2024 (one year before planned end of study date).

Exclusion Criteria:

  • Previous participation in this study. Participation is defined as having given informed consent in this study
  • Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation
  • Known or suspected hypersensitivity to study product or related products.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03875547


Contacts
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Contact: Novo Nordisk (+1) 866-867-7178 clinicaltrials@novonordisk.com

Locations
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Denmark
Novo Nordisk Investigational Site
Søborg, Denmark
Sponsors and Collaborators
Novo Nordisk A/S

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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT03875547     History of Changes
Other Study ID Numbers: NN7999-4404
U1111-1198-6270 ( Other Identifier: World Health Organization (WHO) )
First Posted: March 14, 2019    Key Record Dates
Last Update Posted: March 14, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
URL: http://novonordisk-trials.com

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Studies a U.S. FDA-regulated Device Product: No

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