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 5368361, California 5332921, United States
University of California San Francisco UCSF
San Francisco 5391959, California 5332921, United States
Connecticut Children's Medical Center
Hartford 4835797, Connecticut 4831725, United States
Georgetown University Medical Center
Washington D.C. 4140963, District of Columbia 4138106, United States
Augusta University
Augusta 4180531, Georgia 4197000, United States
Memorial Health University Medical Center
Savannah 4221552, Georgia 4197000, United States
St. Luke's Mountain States Tumor Institute
Boise 5586437, Idaho 5596512, United States
Childrens Hospital of Chicago
Chicago 4887398, Illinois 4896861, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis 4259418, Indiana 4921868, United States
Children's Hosp-New Orleans
New Orleans 4335045, Louisiana 4331987, United States
Children's Hospital of Michigan
Detroit 4990729, Michigan 5001836, United States
Southern Specialty Clinic
Flowood 4426822, Mississippi 4436296, United States
Louisiana Ctr for Adv Med-LCAM
Madison 4434663, Mississippi 4436296, United States
The Children's Mercy Hospital
Kansas City 4393217, Missouri 4398678, United States
Children's Nebraska
Omaha 5074472, Nebraska 5073708, United States
ECU Sickle Cell Comp Clinic
Greenville 4469160, North Carolina 4482348, United States
Nationwide Children's Hospital
Columbus 4509177, Ohio 5165418, United States
Penn State Hershey Medical Center
Hershey 5193342, Pennsylvania 6254927, United States
BI-LO Chrt Childn's Cancer Ctr
Greenville 4580543, South Carolina 4597040, United States
Cook Children's Hospital-Hematology-Oncology
Fort Worth 4691930, Texas 4736286, United States
Texas Children's Hospital_Houston
Houston 4699066, Texas 4736286, United States
Univ TX Hlth Sci Ctr Houston
Houston 4699066, Texas 4736286, United States
Virginia Commonwealth University_Richmond_1
Richmond 4781708, Virginia 6254928, United States
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv 728193, Bulgaria
Koagulationsmottagningen
Solna 2675397, Sweden
Novo Nordisk
(+1) 866-867-7178
clinicaltrials@novonordisk.com