The compassionate use programme will give participants concizumab for free, even thoughit is not yet approved by health authorities. This is because participants need thismedicine to treat their haemophilia properly. The programme will check that participantsare 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 have4-5 visits with the study doctor for the first half year. After that they will have 1visit 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 followingcriteria occurred first: 1) when the patient is included in a clinical trial withconcizumab or 2) up to 6 months after concizumab is commercially available in thepatient's country and approved for the patient (The time span of 6 months should provideample time for the patient to obtain concizumab commercially) or 3) the sponsor decidesto discontinue concizumab clinical development for the patient's population.
Drug: Concizumab
Injected under the skin (subcutaneous, sc) once daily, individual dose adjustment.
Inclusion Criteria:
- 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:
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:
- 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.
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Georgetown University Medical Center
Washington, District of Columbia, United States
Memorial Health University Medical Center
Savannah, Georgia, United States
Childrens Hospital of Chicago
Chicago, Illinois, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 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
Nationwide Children's Hospital
Columbus, Ohio, 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" Clinica of Pediatric
Plovdiv, Bulgaria
Koagulationsmottagningen
Solna, Sweden
Novo Nordisk
(+1) 866-867-7178
clinicaltrials@novonordisk.com