Compassionate Use of Concizumab if You Have Haemophilia
NCT ID: NCT04921956
Last Updated: 2025-12-29
Study Results
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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Brief Summary
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A patient is considered to have completed the programme when any of the following criteria occurred first: 1) when the patient is included in a clinical trial with concizumab or 2) up to 6 months after concizumab is commercially available in the patient's country and approved for the patient (The time span of 6 months should provide ample time for the patient to obtain concizumab commercially) or 3) the sponsor decides to discontinue concizumab clinical development for the patient's population.
Detailed Description
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Conditions
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Interventions
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Concizumab
Injected under the skin (subcutaneous, sc) once daily, individual dose adjustment.
Eligibility Criteria
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Inclusion Criteria
* Patients with congenital haemophilia:
1. severe haemophilia A (coagulation factor VIII (FVIII) less than 1%) or moderate/severe haemophilia B (coagulation factor IX (FIX) less than or equal to 2%) without inhibitors or
2. any haemophilia severity with documented history of inhibitors (more than or equal to 0.6 bethesda unit (BU)) who cannot be treated satisfactorily with authorised and marketed medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access), and who are not able to enrol in clinical trials designed to support the development and registration of concizumab medicines (example: due to inhibitors or allergic reactions to factor-containing products, or due to poor venous access) as per investigator and Novo Nordisk assessment.
* The potential benefit for the individual patient justifies the potential risks of treatment.
Exclusion Criteria
* Any condition (current or medical history), which in the investigator's or Novo Nordisk's opinion might jeopardise patient's safety or compliance with the protocol.
ALL
No
Sponsors
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Novo Nordisk A/S
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
University of California San Francisco UCSF
San Francisco, California, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Georgetown University Medical Center
Washington D.C., District of Columbia, United States
Augusta University
Augusta, Georgia, United States
Memorial Health University Medical Center
Savannah, Georgia, United States
St. Luke's Mountain States Tumor Institute
Boise, Idaho, United States
Childrens Hospital of Chicago
Chicago, Illinois, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, United States
Children's Hosp-New Orleans
New Orleans, Louisiana, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Southern Specialty Clinic
Flowood, Mississippi, United States
Louisiana Ctr for Adv Med-LCAM
Madison, Mississippi, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Children's Nebraska
Omaha, Nebraska, United States
ECU Sickle Cell Comp Clinic
Greenville, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Penn State Hershey Medical Center
Hershey, Pennsylvania, United States
BI-LO Chrt Childn's Cancer Ctr
Greenville, South Carolina, United States
Cook Children's Hospital-Hematology-Oncology
Fort Worth, Texas, United States
Texas Children's Hospital_Houston
Houston, Texas, United States
Univ TX Hlth Sci Ctr Houston
Houston, Texas, United States
Virginia Commonwealth University_Richmond_1
Richmond, Virginia, United States
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv, , Bulgaria
Koagulationsmottagningen
Solna, , Sweden
Countries
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Central Contacts
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Other Identifiers
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NN7415-4807
Identifier Type: -
Identifier Source: org_study_id