Compassionate Use of Concizumab if You Have Haemophilia
Public ClinicalTrials.gov record NCT04921956. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 identification
- NCT ID
- NCT04921956
- Recruitment status
- Available
- Study type
- Expanded access
- Phase
- Not listed
- Enrollment
- Not listed
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- Jun 14, 2026
United States locations
- U.S. sites
- 23
- U.S. states
- 18
- U.S. cities
- 22
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Los Angeles - Endocrinology | Los Angeles | California | 90027 | Available |
| University of California San Francisco UCSF | San Francisco | California | 94158 | Available |
| Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | Available |
| Georgetown University Medical Center | Washington D.C. | District of Columbia | 20007 | Available |
| Augusta University | Augusta | Georgia | 30912 | Available |
| Memorial Health University Medical Center | Savannah | Georgia | 31404 | Available |
| St. Luke's Mountain States Tumor Institute | Boise | Idaho | 83712 | Available |
| Childrens Hospital of Chicago | Chicago | Illinois | 60611 | Available |
| Indiana Hemophilia-Thromb Ctr | Indianapolis | Indiana | 46260 | Available |
| Children's Hosp-New Orleans | New Orleans | Louisiana | 70118 | Available |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | Available |
| Southern Specialty Clinic | Flowood | Mississippi | 39232 | Available |
| Louisiana Ctr for Adv Med-LCAM | Madison | Mississippi | 39110 | Available |
| The Children's Mercy Hospital | Kansas City | Missouri | 64108 | Available |
| Children's Nebraska | Omaha | Nebraska | 68114 | Available |
| ECU Sickle Cell Comp Clinic | Greenville | North Carolina | 27834 | Available |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Available |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | Available |
| BI-LO Chrt Childn's Cancer Ctr | Greenville | South Carolina | 29605 | Available |
| Cook Children's Hospital-Hematology-Oncology | Fort Worth | Texas | 76104 | Available |
| Texas Children's Hospital_Houston | Houston | Texas | 77030 | Available |
| Univ TX Hlth Sci Ctr Houston | Houston | Texas | 77030 | Available |
| Virginia Cwealth Univ Richmond | Richmond | Virginia | 23298-0461 | Available |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04921956, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 14, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04921956 live on ClinicalTrials.gov.