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Weekly shot could help kids with hemophilia b avoid bleeding

NCT ID NCT01467427

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

25 people

The number who actually took part.

Started

May 2012

Finished

Nov 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 12 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male patients with moderately severe or severe congenital haemophilia B with a Factor IX activity level below or equal to 2% according to medical records * Age below or equal to 12 years (until patient turns 13 years, at time of inclusion) * Body weight above or equal to 10 kg * History of at least 50 exposure days (EDs) to other FIX products * The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an electronic diary (eDiary), capable of conducting home treatment and otherwise able to follow trial procedures Exclusion Criteria: * Known history of FIX inhibitors * Current FIX inhibitors above or equal to 0.6 Bethesda Units (BU) * Congenital or acquired coagulation disorder other than haemophilia B * Platelet count below 50,000/mcL at screening * Alanine aminotransferase (ALT) above 3 times the upper limit of normal reference ranges at screening * Creatinine level above or equal to 1.5 times above the upper normal limit of normal reference ranges at screening * Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count below or equal to 200/mcL * Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids) * Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)

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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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