Clinical trial NCT04921956: Compassionate Use of Concizumab if You Have Haemophilia
Official title: Concizumab Compassionate Use Programme for Patients With Congenital Haemophilia
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Congenital Haemophilia
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2021-06-10
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no

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.


Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles - Endocrinology
state: California
city: San Francisco
country: United States
facility: University of California San Francisco UCSF
state: California
city: Hartford
country: United States
facility: Connecticut Children's Medical Center
state: Connecticut
city: Washington
country: United States
facility: Georgetown University Medical Center
state: District of Columbia
city: Savannah
country: United States
facility: Memorial Health University Medical Center
state: Georgia
city: Chicago
country: United States
facility: Childrens Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia-Thromb Ctr
state: Indiana
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Flowood
country: United States
facility: Southern Specialty Clinic
state: Mississippi
city: Madison
country: United States
facility: Louisiana Ctr for Adv Med-LCAM
state: Mississippi
city: Kansas City
country: United States
facility: The Children's Mercy Hospital
state: Missouri
city: Omaha
country: United States
facility: Children's Nebraska
state: Nebraska
city: Greenville
country: United States
facility: ECU Sickle Cell Comp Clinic
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Greenville
country: United States
facility: BI-LO Chrt Childn's Cancer Ctr
state: South Carolina
city: Fort Worth
country: United States
facility: Cook Children's Hospital-Hematology-Oncology
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital_Houston
state: Texas
city: Houston
country: United States
facility: Univ TX Hlth Sci Ctr Houston
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University_Richmond_1
state: Virginia
city: Plovdiv
country: Bulgaria
facility: UMHAT "Sveti Georgi" Clinica of Pediatric
city: Solna
country: Sweden
facility: Koagulationsmottagningen
Study officials:
References: