ACTIVE_NOT_RECRUITING

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

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Conditions

Haemophilia A and B With and Without Inhibitors

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use. Participants will have to inject the study medicine every day under the skin with a pen-injector. The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

Official Title

Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

Quick Facts

Study Start:2022-03-24
Study Completion:2029-11-02
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05135559

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
  2. * Diagnosis of congenital severe haemophilia A (FVIII below 1%) or moderate/severe congenital haemophilia B (FIX (coagulation factor IX) below or equal to 2%), or congenital haemophilia with inhibitors.
  3. * For arm 1 only: Male aged below 12 years of age at the time of signing informed consent.
  4. * For arm 1 only: Patients with inhibitors (haemophilia A with inhibitors or haemophilia B with inhibitors)
  5. 1. Patients with HAwI (haemophilia A with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
  6. 2. Patients with HBwI (haemophilia B with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
  7. 3. Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products)
  8. * For arm 1 only: Patients without inhibitors (haemophilia A or haemophilia B)
  9. 1. Patients with historical medical records of at least 52 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products; Surgery related PPX or short-term PPX (e.g., in relation to a severe bleed) is not allowed) during the last year prior to enrolment and with at least 3 documented treated bleeds (For participants less than (\<) 2 years of age there is no limitation for number of documented treated bleeds in the medical history) during this period
  10. 2. Patients with historical medical records of a total of at least 26 weeks of PPX (prophylaxis) treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia \<1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available)
  11. * For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.
  1. * Known or suspected hypersensitivity to study intervention or related products.
  2. * Known inherited or acquired coagulation disorder other than congenital haemophilia.
  3. * Ongoing or planned Immune Tolerance Induction treatment.
  4. * History of thromboembolic disease (aIncludes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events).

Contacts and Locations

Principal Investigator

Clinical Transparency (dept. 2834)
STUDY_DIRECTOR
Novo Nordisk A/S

Study Locations (Sites)

Rady Childrens Hosp San Diego
San Diego, California, 92123
United States
Arnold Palmer Children's Hospital
Orlando, Florida, 32806
United States
Memorial Health University Medical Center
Savannah, Georgia, 31404
United States
Childrens Hospital of Chicago
Chicago, Illinois, 60611
United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260
United States
Children's Hosp-New Orleans
New Orleans, Louisiana, 70118
United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108
United States
Children's Nebraska
Omaha, Nebraska, 68114
United States
ECU Sickle Cell Comp Clinic
Greenville, North Carolina, 27834
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
St Christopher Hosp for Child
Philadelphia, Pennsylvania, 19134
United States
Vanderbilt Hemostasis Treatment Clinic
Nashville, Tennessee, 37212
United States
Cook Children's Hospital-Hematology-Oncology
Fort Worth, Texas, 76104
United States
Texas Children's Hospital_Houston
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Novo Nordisk A/S

  • Clinical Transparency (dept. 2834), STUDY_DIRECTOR, Novo Nordisk A/S

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-03-24
Study Completion Date2029-11-02

Study Record Updates

Study Start Date2022-03-24
Study Completion Date2029-11-02

Terms related to this study

Additional Relevant MeSH Terms

  • Haemophilia A and B With and Without Inhibitors