Compassionate Use of Concizumab if You Have Haemophilia

June 11, 2026 updated by: Novo Nordisk A/S

Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Available

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Bulgaria
      • Plovdiv, Bulgaria, 4002
        • Available
        • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
  • Sweden
      • Solna, Sweden, 171 64
        • Available
        • Koagulationsmottagningen
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Available
        • Children's Hospital Los Angeles - Endocrinology
      • San Francisco, California, United States, 94158
        • Available
        • University of California San Francisco UCSF
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Available
        • Connecticut Children's Medical Center
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20007
        • Available
        • Georgetown University Medical Center
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Available
        • Augusta University
      • Savannah, Georgia, United States, 31404
        • Available
        • Memorial Health University Medical Center
    • Idaho
      • Boise, Idaho, United States, 83712
        • Available
        • St. Luke's Mountain States Tumor Institute
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Available
        • Childrens Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Available
        • Indiana Hemophilia-Thromb Ctr
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Available
        • Children's Hosp-New Orleans
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Available
        • Children's Hospital of Michigan
    • Mississippi
      • Flowood, Mississippi, United States, 39232
        • Available
        • Southern Specialty Clinic
      • Madison, Mississippi, United States, 39110
        • Available
        • Louisiana Ctr for Adv Med-LCAM
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Available
        • The Children's Mercy Hospital
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Available
        • Children's Nebraska
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • Available
        • ECU Sickle Cell Comp Clinic
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Available
        • Nationwide Children's Hospital
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Available
        • Penn State Hershey Medical Center
    • South Carolina
      • Greenville, South Carolina, United States, 29605
        • Available
        • BI-LO Chrt Childn's Cancer Ctr
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Available
        • Cook Children's Hospital-Hematology-Oncology
      • Houston, Texas, United States, 77030
        • Available
        • Texas Children's Hospital_Houston
      • Houston, Texas, United States, 77030
        • Available
        • Univ TX Hlth Sci Ctr Houston
    • Virginia
      • Richmond, Virginia, United States, 23298-0461
        • Available
        • Virginia Cwealth Univ Richmond

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 4, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 4, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 10, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 15, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 11, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NN7415-4807

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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