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Compassionate Use of Concizumab if You Have Haemophilia

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ClinicalTrials.gov Identifier: NCT04921956
Expanded Access Status : Available
First Posted : June 10, 2021
Last Update Posted : March 25, 2022
Information provided by (Responsible Party):
Novo Nordisk A/S

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.

Condition or disease Intervention/treatment
Congenital Haemophilia Drug: Concizumab

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia

Intervention Details:
  • Drug: Concizumab
    Injected under the skin (subcutaneous, sc) once daily, individual dose adjustment.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04921956

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Contact: Novo Nordisk (+1) 866-867-7178 clinicaltrials@novonordisk.com

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United States, Illinois
Novo Nordisk Investigational Site Available
Chicago, Illinois, United States, 60611
United States, Indiana
Novo Nordisk Investigational Site Available
Indianapolis, Indiana, United States, 46260
United States, South Carolina
Novo Nordisk Investigational Site Available
Greenville, South Carolina, United States, 29605
United States, Texas
Novo Nordisk Investigational Site Available
Fort Worth, Texas, United States, 76104
Novo Nordisk Investigational Site Available
Houston, Texas, United States, 77030
United States, Virginia
Novo Nordisk Investigational Site Available
Richmond, Virginia, United States, 23298-0461
Sponsors and Collaborators
Novo Nordisk A/S
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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT04921956    
Other Study ID Numbers: NN7415-4807
First Posted: June 10, 2021    Key Record Dates
Last Update Posted: March 25, 2022
Last Verified: March 2022
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn