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NCT05135559 | Recruiting | Haemophilia A and B With and Without Inhibitors


A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
Sponsor:

Novo Nordisk A/S

Brief Summary:

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use. Participants will have to inject the study medicine every day under the skin with a pen-injector. The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

Condition or disease

Haemophilia A and B With and Without Inhibitors

Intervention/treatment

Concizumab

Phase

Phase 3

Study Type : Interventional
Estimated Enrollment : 90 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors
Actual Study Start Date : March 24, 2022
Estimated Primary Completion Date : December 1, 2024
Estimated Study Completion Date : November 2, 2029
Arm Intervention/treatment

Experimental: Concizumab-naïve patients

Concizumab-naïve participants below 12 years of age at the time of consent/assent

Drug: Concizumab

Experimental: Patients coming from compassionate use

Patients previously treated with concizumab via compassionate use, either on an individual patient basis or through the concizumab compassionate use programme NN7415-4807

Drug: Concizumab

Ages Eligible for Study:
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria
  • Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
  • Diagnosis of congenital severe haemophilia A (FVIII below 1%) or moderate/severe congenital haemophilia B (FIX (coagulation factor IX) below or equal to 2%), or congenital haemophilia with inhibitors.
  • For arm 1 only: Male aged below 12 years of age at the time of signing informed consent.
  • For arm 1 only: Patients with inhibitors (haemophilia A with inhibitors or haemophilia B with inhibitors)
  • Patients with HAwI (haemophilia A with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia <1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
  • Patients with HBwI (haemophilia B with inhibitors) with historical medical records of a total of at least 26 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia <1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available).
  • Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products)
  • For arm 1 only: Patients without inhibitors (haemophilia A or haemophilia B)
  • Patients with historical medical records of at least 52 weeks of on-demand treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products; Surgery related PPX or short-term PPX (e.g., in relation to a severe bleed) is not allowed) during the last year prior to enrolment and with at least 3 documented treated bleeds (For participants less than (<) 2 years of age there is no limitation for number of documented treated bleeds in the medical history) during this period
  • Patients with historical medical records of a total of at least 26 weeks of PPX (prophylaxis) treatment (On-demand or PPX treatment qualifying for this study is understood as patient-treatment solely for bleeds with intravenous coagulation factor-containing products) within the last 52 weeks prior to enrolment (For patients below 1 year of age that have been diagnosed with haemophilia <1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available)
  • For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.
Exclusion Criteria
  • Known or suspected hypersensitivity to study intervention or related products.
  • Known inherited or acquired coagulation disorder other than congenital haemophilia.
  • Ongoing or planned Immune Tolerance Induction treatment.
  • History of thromboembolic disease (aIncludes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events).

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

Location Details


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A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations


Recruiting

United States, California

Rady Childrens Hosp San Diego

San Diego, California, United States, 92123

Recruiting

United States, Florida

Arnold Palmer Children's Hospital

Orlando, Florida, United States, 32806

Not yet recruiting

United States, Florida

Nemours Child Orlando Hem/Onc.

Orlando, Florida, United States, 32827

Not yet recruiting

United States, Georgia

Augusta Univ/Childrens Hosp-GA

Augusta, Georgia, United States, 30912

Recruiting

United States, Georgia

Memorial Health University Medical Center

Savannah, Georgia, United States, 31404

Recruiting

United States, Indiana

Indiana Hemophilia-Thromb Ctr

Indianapolis, Indiana, United States, 46260

Recruiting

United States, Louisiana

Children's Hosp-New Orleans

New Orleans, Louisiana, United States, 70118

Not yet recruiting

United States, Missouri

The Children's Mercy Hospital

Kansas City, Missouri, United States, 64108

Recruiting

United States, North Carolina

ECU Sickle Cell Comp Clinic

Greenville, North Carolina, United States, 27834

Not yet recruiting

United States, Ohio

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Recruiting

United States, Pennsylvania

St Christopher Hosp for Child

Philadelphia, Pennsylvania, United States, 19134

Recruiting

United States, Tennessee

Vanderbilt Hemostasis Treatment Clinic

Nashville, Tennessee, United States, 37212

Not yet recruiting

United States, Texas

Cook Children's Hospital-Hematology-Oncology

Fort Worth, Texas, United States, 76104

Recruiting

United States, Virginia

Pediatrics Hematology/Oncology Clinic Battle Building

Charlottesville, Virginia, United States, 22908

Not yet recruiting

Algeria, British Columbia

Haematology and Blood Bank Department

Algiers, British Columbia, Algeria, 16000

Not yet recruiting

Algeria, Ontario

CHU Constantine IBN BADIS/ Hematology department

Constantine, Ontario, Algeria, 25000

Not yet recruiting

Bosnia and Herzegovina, Gujarat

University Clinical Center of Republic Srpska (545)

Banja Luka, Gujarat, Bosnia and Herzegovina, 78000

Recruiting

Bulgaria, Maharashtra

UMHAT "Sveti Georgi" Clinica of Pediatric

Plovdiv, Maharashtra, Bulgaria, 4002

Not yet recruiting

Canada, Rajasthan

BC Children's Hospital

Vancouver, Rajasthan, Canada, V6H 3V4

Recruiting

Canada, Uttar Pradesh

McMaster Children's Hospital

Hamilton, Uttar Pradesh, Canada, L8N 3Z5

Not yet recruiting

Estonia, Kuala Lumpur

Tallinn Children's Hospital

Tallinn, Kuala Lumpur, Estonia, 13419

Recruiting

France, Penang

Centre Hospitalier Metropole Savoie

Chambery, Penang, France, 73000

Not yet recruiting

France, Terengganu

Ap-Hp-Hopital de Bicetre-1

Le Kremlin Bicetre Cedex, Terengganu, France, 94275

Recruiting

France, Dolnoslaskie

Ap-Hp-Hopital Necker-1

Paris, Dolnoslaskie, France, 75015

Recruiting

Greece, Dolnoslaskie

Aghia Sophia Childrens' Hospital

Athens, Dolnoslaskie, Greece, GR-11527

Not yet recruiting

Greece, Gauteng

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, Gauteng, Greece, GR 54642

Recruiting

Greece, Beşevler/Ankara

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, Beşevler/Ankara, Greece, GR-54642

Recruiting

India,

Nirmal Hospital Pvt. Ltd.

Surat, India, 395002

Recruiting

India,

Sahyadri Super Speciality Hospital

Pune, India, 411004

Recruiting

India,

J K Lon Hospital

Jaipur, India, 302004

Recruiting

India,

Post Graduate Institute of Child Health

Noida, India, 201303

Not yet recruiting

Italy,

A.O.U policlinico "G. Rodolico-San Marco"

Catania, Italy, 95123

Recruiting

Italy,

Dipartimento di Ematologia Univ. Firenze

Firenze, Italy, 50134

Recruiting

Italy,

Azienda Ospedaliera di Padova

Padova, Italy, 35128

Recruiting

Italy,

Azienda Ospedaliera-Universitaria Parma

Parma, Italy, 43126

Recruiting

Japan,

St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, Japan, 216-8511

Recruiting

Japan,

Saitama Children's Med Centre_Hematology-Oncology

Saitama, Japan, 330-8777

Not yet recruiting

Lebanon,

Saint George Hospital University Medical Center

Beirut, Lebanon, 961

Not yet recruiting

Lebanon,

Hospital Nini

Tripoli, Lebanon, 1434

Recruiting

Lithuania,

Centre of Oncology and Hematology, Vilnius University

Vilnius, Lithuania, LT-08406

Not yet recruiting

Malaysia,

Hospital Tunku Azizah

Kampung Baru, Malaysia, 50300

Not yet recruiting

Malaysia,

Hospital Pulau Pinang

George Town, Malaysia, 10450

Not yet recruiting

Malaysia,

Hospital Sultanah Nur Zahirah

Kuala Terengganu, Malaysia, 20400

Not yet recruiting

Malaysia,

Sarawak General Hospital

Kuching, Malaysia, 93586

Recruiting

North Macedonia,

PHI University Clinic for Children's Diseases Skopje

Skopje, North Macedonia, 1000

Recruiting

Norway,

Klinisk forskningspost

Oslo, Norway, 0372

Recruiting

Poland,

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

Wroclaw, Poland, 50-556

Not yet recruiting

Poland,

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

Wroclaw, Poland, 50-556

Not yet recruiting

Poland,

Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

Lublin, Poland, 20-093

Not yet recruiting

Poland,

Uniwersyteckie Centrum Kliniczne WUM

Warszawa, Poland, 02-091

Not yet recruiting

Romania,

Clinic of Haematology, Fundeni Clinical Institute

Bucharest, Romania, 022328

Not yet recruiting

Romania,

Spitalul Clinic de Urgenta pentru Copii Cluj Napoca

Cluj-Napoca, Romania, 400177

Not yet recruiting

Romania,

Spitalul Clinic Judetean De Urgenta Bihor

Oradea, Romania, 410469

Withdrawn

Russian Federation,

Children Regional Clinical Hospital

Krasnodar, Russian Federation, 350007

Withdrawn

Russian Federation,

Morozovskaya municipal children hospital

Moscow, Russian Federation, 119049

Withdrawn

Russian Federation,

City out-patient clinic 37, City Hemophilia Centre

Saint-Petersburg, Russian Federation, 191186

Not yet recruiting

South Africa,

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, South Africa, 2193

Recruiting

Spain,

Hospital Vall d'Hebron

Barcelona, Spain, 08035

Recruiting

Spain,

Hospital Virgen de la Arrixaca - Hematología

El Palmar, Spain, 30120

Recruiting

Spain,

Hospital Universitario La Paz

Madrid, Spain, 28046

Recruiting

Spain,

Hospital Universitario Regional de Málaga

Málaga, Spain, 29009

Recruiting

Sweden,

Koagulationscentrum

Göteborg, Sweden, 413 46

Recruiting

Thailand,

King Chulalongkorn Memorial Hospital_Bangkok_0

Bangkok, Thailand, 10330

Recruiting

Thailand,

Ramathibodi Hospital

Bangkok, Thailand, 10400

Recruiting

Thailand,

Siriraj Hospital - Hematology and Oncology

Bangkok, Thailand, 10700

Recruiting

Thailand,

Sunpasitthiprasong Hospital

Ubon Ratchathani, Thailand, 34000

Recruiting

Turkey,

Gazi University

Ankara, Turkey, 06500

Recruiting

Turkey,

Acibadem Adana Hastanesi

Adana, Turkey, 01130

Recruiting

Turkey,

Ege Universitesi Tip Fakultesi

Izmir, Turkey, 35100

Recruiting

Turkey,

Ondokuz Mayis University Medical Faculty Ped. Haematology

Samsun, Turkey, 55139

Recruiting

United Kingdom,

Birmingham Children's Hospital

Birmingham, United Kingdom, B4 6NH

Recruiting

United Kingdom,

University Hospitals Bristol & Weston NHS Foundation Trust

Bristol, United Kingdom, BS2 8BJ

Recruiting

United Kingdom,

Great Ormond Street Hospital for Children

London, United Kingdom, WC1N 3HR

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