Compassionate Use of Concizumab if You Have Haemophilia
Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia
1 other identifier
expanded_access
N/A
3 countries
25
Brief Summary
The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that participants are safe and that the medicine works for them. The programme may last for years. Participants will take one injection under their skin every day. Participants will have 4-5 visits with the study doctor for the first half year. After that they will have 1 visit every half year. At all clinic visits participants will have blood samples taken. Participants will fill in a diary between the visits. 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
25 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 4, 2021
CompletedFirst Posted
Study publicly available on registry
June 10, 2021
CompletedFebruary 13, 2026
February 1, 2026
June 4, 2021
February 10, 2026
Conditions
Interventions
Injected under the skin (subcutaneous, sc) once daily, individual dose adjustment.
Eligibility Criteria
You may qualify if:
- Informed consent obtained before any programme-related activities. Programme-related activities are any procedures that are carried out as part of the programme.
- Patients with congenital haemophilia:
- 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
- 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.
You may not qualify if:
- Known or suspected hypersensitivity to investigational medicinal product or related products.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Novo Nordisk A/Slead
Study Sites (25)
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027, United States
University of California San Francisco UCSF
San Francisco, California, 94158, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Georgetown University Medical Center
Washington D.C., District of Columbia, 20007, United States
Augusta University
Augusta, Georgia, 30912, United States
Memorial Health University Medical Center
Savannah, Georgia, 31404, United States
St. Luke's Mountain States Tumor Institute
Boise, Idaho, 83712, 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
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Southern Specialty Clinic
Flowood, Mississippi, 39232, United States
Louisiana Ctr for Adv Med-LCAM
Madison, Mississippi, 39110, 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
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
BI-LO Chrt Childn's Cancer Ctr
Greenville, South Carolina, 29605, United States
Cook Children's Hospital-Hematology-Oncology
Fort Worth, Texas, 76104, United States
Texas Children's Hospital_Houston
Houston, Texas, 77030, United States
Univ TX Hlth Sci Ctr Houston
Houston, Texas, 77030, United States
Virginia Commonwealth University_Richmond_1
Richmond, Virginia, 23298-0461, United States
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv, 4002, Bulgaria
Koagulationsmottagningen
Solna, 171 64, Sweden