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Free daily shot offers hope for hemophilia patients

NCT ID NCT04921956

Disease control Sponsor: Novo Nordisk A/S Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access This study
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 11, 2026 · Updated 4 times

Summary

This program provides concizumab, a daily injection under the skin, for free to people with congenital hemophilia who need better treatment. Participants will be monitored for safety and how well the medicine works over several years. The program is for those with severe hemophilia A or moderate/severe hemophilia B, with or without inhibitors.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Concizumab (a daily injection under the skin)
What this could lead to
If successful, this could provide a long-term treatment option for hemophilia patients who have limited alternatives, helping to prevent bleeding episodes.
What could go wrong
This is an early-access program, not a full clinical trial, so long-term safety and effectiveness are still being studied. The drug is not yet approved and may have unknown risks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

hemophilia hemophilia A hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congenital haemophilia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    25 sites in 3 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Augusta University

    AVAILABLE

    Augusta, Georgia, 30912, United States

  • BI-LO Chrt Childn's Cancer Ctr

    AVAILABLE

    Greenville, South Carolina, 29605, United States

  • Children's Hosp-New Orleans

    AVAILABLE

    New Orleans, Louisiana, 70118, United States

  • Children's Hospital Los Angeles - Endocrinology

    AVAILABLE

    Los Angeles, California, 90027, United States

  • Children's Hospital of Michigan

    AVAILABLE

    Detroit, Michigan, 48201, United States

  • Children's Nebraska

    AVAILABLE

    Omaha, Nebraska, 68114, United States

  • Childrens Hospital of Chicago

    AVAILABLE

    Chicago, Illinois, 60611, United States

  • Connecticut Children's Medical Center

    AVAILABLE

    Hartford, Connecticut, 06106, United States

  • Cook Children's Hospital-Hematology-Oncology

    AVAILABLE

    Fort Worth, Texas, 76104, United States

  • ECU Sickle Cell Comp Clinic

    AVAILABLE

    Greenville, North Carolina, 27834, United States

  • Georgetown University Medical Center

    AVAILABLE

    Washington D.C., District of Columbia, 20007, United States

  • Indiana Hemophilia-Thromb Ctr

    AVAILABLE

    Indianapolis, Indiana, 46260, United States

  • Koagulationsmottagningen

    AVAILABLE

    Solna, 171 64, Sweden

  • Louisiana Ctr for Adv Med-LCAM

    AVAILABLE

    Madison, Mississippi, 39110, United States

  • Memorial Health University Medical Center

    AVAILABLE

    Savannah, Georgia, 31404, United States

  • Nationwide Children's Hospital

    AVAILABLE

    Columbus, Ohio, 43205, United States

  • Penn State Hershey Medical Center

    AVAILABLE

    Hershey, Pennsylvania, 17033, United States

  • Southern Specialty Clinic

    AVAILABLE

    Flowood, Mississippi, 39232, United States

  • St. Luke's Mountain States Tumor Institute

    AVAILABLE

    Boise, Idaho, 83712, United States

  • Texas Children's Hospital_Houston

    AVAILABLE

    Houston, Texas, 77030, United States

  • The Children's Mercy Hospital

    AVAILABLE

    Kansas City, Missouri, 64108, United States

  • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

    AVAILABLE

    Plovdiv, 4002, Bulgaria

  • Univ TX Hlth Sci Ctr Houston

    AVAILABLE

    Houston, Texas, 77030, United States

  • University of California San Francisco UCSF

    AVAILABLE

    San Francisco, California, 94158, United States

  • Virginia Cwealth Univ Richmond

    AVAILABLE

    Richmond, Virginia, 23298-0461, United States

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