Congenital HaemophiliaNCT04921956
Compassionate Use of Concizumab if You Have Haemophilia
A study testing Concizumab in people with Congenital Haemophilia, run by Novo Nordisk A/S at 25 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Haemophilia
- What is being tested?
- Concizumab
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 25 sitesin 3 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
25 sites in 3 countries.
25 sites in 3 countries
United States23 sites
Augusta University
Augusta, Georgia, 30912
St. Luke's Mountain States Tumor Institute
Boise, Idaho, 83712
Childrens Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Hospital of Michigan
Detroit, Michigan, 48201
Southern Specialty Clinic
Flowood, Mississippi, 39232
Cook Children's Hospital-Hematology-Oncology
Fort Worth, Texas, 76104
ECU Sickle Cell Comp Clinic
Greenville, North Carolina, 27834
BI-LO Chrt Childn's Cancer Ctr
Greenville, South Carolina, 29605
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital_Houston
Houston, Texas, 77030
Univ TX Hlth Sci Ctr Houston
Houston, Texas, 77030
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260
The Children's Mercy Hospital
Kansas City, Missouri, 64108
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
Louisiana Ctr for Adv Med-LCAM
Madison, Mississippi, 39110
Children's Hosp-New Orleans
New Orleans, Louisiana, 70118
Children's Nebraska
Omaha, Nebraska, 68114
Virginia Cwealth Univ Richmond
Richmond, Virginia, 23298-0461
University of California San Francisco UCSF
San Francisco, California, 94158
Memorial Health University Medical Center
Savannah, Georgia, 31404
Georgetown University Medical Center
Washington D.C., District of Columbia, 20007
Bulgaria1 site
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv, 4002
About this study
Official title: 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.
- Conditions
- Congenital Haemophilia
- Being tested
- Concizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ConcizumabDrug
Injected under the skin (subcutaneous, sc) once daily, individual dose adjustment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital HaemophiliaStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04921956
- Sponsor's protocol ID
- NN7415-4807
- Status last verified
- Oct 2026
- First submitted
- 4 Jun 2021
- Last updated
- 28 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia
- ClinicalTrials.gov ID
- NCT04921956
- Acronym / sponsor ID
- NN7415-4807
- Conditions
- Congenital Haemophilia
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- First posted
- 10 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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:
- The potential benefit for the individual patient justifies the potential risks of treatment.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ConcizumabInjected under the skin (subcutaneous, sc) once daily, individual dose adjustment. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
Related trials for Congenital Haemophilia
RecruitingObservational study
Severe Congenital Hemostatic Defects, Cerebral MIcrobleeds and COGnition
Cerebral Microbleeds, Congenital Haemophilia, Congenital Von Willebrand Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
25 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Concizumab in people with Congenital Haemophilia, run by Novo Nordisk A/S at 25 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Bulgaria, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2026.