Weekly shot could help kids with hemophilia b avoid bleeding

NCT ID NCT01467427

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

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a new long-acting factor IX drug (nonacog beta pegol) in 25 children with hemophilia B. The drug is given as a weekly injection to prevent bleeding episodes. The main goal was to check safety and whether the body develops antibodies that block the treatment. Results showed it was generally safe and effective at reducing bleeds.

What this could mean

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

Active substance: nonacog beta pegol (a long-acting factor IX replacement given as a weekly injection)
What this could lead to: If successful, this could provide a more convenient once-weekly treatment to prevent bleeding episodes in children with hemophilia B.
What could go wrong: This is a small Phase 3 trial (25 children) and results may not apply to all patients. Some children may still develop inhibitors that block the treatment's effect.

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

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Conditions

The condition(s) this trial relates to.

hemophilia B hemorrhagic disease

As listed by the trial registrant

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

CONGENITAL BLEEDING DISORDER HAEMOPHILIA B

Contacts and locations

Locations

Ap-Hp-Hopital Necker-1

Paris, 75015, France
Ap-Hp-Hopital de Bicetre-1

Le Kremlin-Bicêtre, 94275, France
Augusta University

Augusta, Georgia, 30912, United States
Basingstoke & North Hampshire Hospital - Centre for Haemophilia, Haemostasis and Thrombosis

Basingstoke, RG24 9NA, United Kingdom
Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom
Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States
Children's Hospital of Michigan

Detroit, Michigan, 48201, United States
Children's Hospitals And Clinics Of Minnesota

Minneapolis, Minnesota, 55404, United States
Children's Hosptl Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Medical Center_Dallas

Dallas, Texas, 75235, United States
Childrens National Medical Ctr

Washington D.C., District of Columbia, 20010-2978, United States
Clinical Hospital Centre Split, Firule, Paediatric Haem. Dpt

Split, 21 000, Croatia
Coagulation Research Center

Duisburg, 47051, Germany
Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, 80045, United States
Dayton Children Hemostati Ctr

Dayton, Ohio, 45404, United States
Emory University_Atlanta_1

Atlanta, Georgia, 30322, United States
Hemorio-Fundarj

Rio de Janeiro, 20211-030, Brazil
Hospital das Clínicas da Faculdade de Medicina da USP

São Paulo, São Paulo, 05403-000, Brazil
Hôpital de la Timone

Marseille, 13385, France
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano

Milan, MI, 20124, Italy
John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom
Johns Hopkins University_Baltimore_3

Baltimore, Maryland, 21205, United States
Leicester Royal Infirmary

Leicester, LE1 5WW, United Kingdom
Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

Leicester, LE1 5WW, United Kingdom
Maimonides Medical Center

Brooklyn, New York, 11220, United States
Medizinische Hochschule Hannover - Pädiatrische Hämato-, Onko- und Hämostasiologie

Hanover, 30625, Germany
Mount Sinai Medical Center

New York, New York, 10029, United States
National Blood Centre

Kuala Lumpur, 50400, Malaysia
National Taiwan University Children's Hospital

Taipei, 100, Taiwan
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Necmettin Erbakan University Pediatric Hematology

Konya, 42090, Turkey (Türkiye)
Nucleo de Pesquisa Instituto Pele Pequeno Principe

Curitiba, Paraná, 80250-060, Brazil
Ogikubo Hospital_Pediatries & Blood

Tokyo, 167-0035, Japan
Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States
Shizuoka Children's Hospital, Hematology-Oncology

Shizuoka, 420-8660, Japan
St Thomas' Hospital

London, SE1 7EH, United Kingdom
St Thomas' Hospital - Haemostasis and Thrombosis Centre

London, SE1 7EH, United Kingdom
St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, 216-8511, Japan
Texas Children's Hospital_Houston

Houston, Texas, 77030, United States
The Children's Mercy Hospital

Kansas City, Missouri, 64108-4619, United States
The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada
UC Davis Medical Center

Sacramento, California, 95817, United States
Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, 44106, United States
Univ Oklahoma Sci Ctr OK City

Oklahoma City, Oklahoma, 73104, United States
Univ TX Hlth Sci Ctr Houston

Houston, Texas, 77030, United States
Univ of NE Med Center_Omaha

Omaha, Nebraska, 68198, United States
Universidade Estadual de Campinas

Campinas, São Paulo, 13081-970, Brazil
University Of Iowa

Iowa City, Iowa, 52242, United States
University Of Louisville_Louisville_0

Louisville, Kentucky, 40202, United States
University of Miami Hospital & Clinics

Miami, Florida, 33136, United States
Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, 37232, United States

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