Compassionate Use of Concizumab if You Have Haemophilia

Available (expanded access)

Conditions studied: Congenital Haemophilia

In brief

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.

Key facts

Study ID

NCT04921956

Run by

Novo Nordisk A/S

Last updated by the study team

2026-06-15

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

• 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 Cwealth Univ Richmond — Richmond, Virginia, United States
• UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics — Plovdiv, Bulgaria
• Koagulationsmottagningen — Solna, Sweden

Full record on ClinicalTrials.gov

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