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New hope for kids with hemophilia b: Long-Acting drug shows promise in preventing bleeds

NCT ID NCT02141074

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a new long-acting clotting factor drug called nonacog beta pegol in 54 young children with hemophilia B who had not been treated before. The drug was given regularly to prevent bleeding episodes and also to treat any bleeds that occurred. The main goal was to see if the drug was safe and if the children's immune systems would develop antibodies that could stop the drug from working. The results help doctors understand how to better manage this bleeding disorder in young patients.

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 clotting factor IX replacement therapy)
What this could lead to
If successful, this could provide a safe and effective way to prevent and treat bleeding episodes in young children with hemophilia B, reducing the risk of joint damage and improving quality of life.
What could go wrong
This is a single-arm, non-controlled trial with only 54 participants, so results may not apply to all patients. There is a risk of developing inhibitory antibodies that can reduce treatment effectiveness.

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

54 people

The number who actually took part.

Started

Jul 2014

Finished

Oct 2022

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 6 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: * Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial * Male, age below 6 years at the time of signing informed consent * Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity level below or equal to 2%) based on medical records or central laboratory results * Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposures to blood components is acceptable) Exclusion Criteria: * Any history of FIX inhibitors (defined by medical records) * Known or suspected hypersensitivity to trial product or related products * Previous participation in this trial. Participation is defined as first dose administered of trial product * Receipt of any investigational medicinal product within 30 days before screening * Congenital or acquired coagulation disorder other than haemophilia B * Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol * Patient's parent(s)/LAR(s) (legally acceptable representative) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

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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

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642, Greece

  • ,,Louis Ţurcanu'' Emergency Hospital for Children

    Timișoara, Timiș County, 300011, Romania

  • 1st Paediatric Department, Fundeni Clinical Institute

    Bucharest, 022328, Romania

  • Aghia Sophia Childrens' Hospital

    Athens, GR-11527, Greece

  • Ap-Hp-Hopital de Bicetre-1

    Le Kremlin-Bicêtre, 94275, France

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Beni Messous Hospital Issaad Hassani

    Algiers, 16000, Algeria

  • Birmingham Children's Hospital

    Birmingham, B4 6NH, United Kingdom

  • CHU Estaing

    Clermont-Ferrand, 63003, France

  • CHU Haute Pierre

    Strasbourg, 67098, France

  • Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

    Nantes, 44093, France

  • Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou

    Rennes, 35033, France

  • Centro Hospitalar Lisboa Norte-HSM

    Lisbon, 1649-035, Portugal

  • Centro Medico Nacional SXXI-Hospital de Pediatria, IMSS

    Mexico City, México, D.F., 06720, Mexico

  • Changhua Christian Hospital

    Changhua, 500, Taiwan

  • Changhua Christian Hospital_Hematology Dept.

    Changhua, 500, Taiwan

  • Children's Hosp-New Orleans

    New Orleans, Louisiana, 70118-5720, United States

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California, 90027, United States

  • Children's Hsptl Of The Kings

    Norfolk, Virginia, 23507, United States

  • China Medical University Children's Hospital

    Taichung, 40447, Taiwan

  • China Medical University Hospital - Children Building

    Taichung, 40447, Taiwan

  • Coagulation Research Center

    Duisburg, 47051, Germany

  • Dayton Children Hemostati Ctr

    Dayton, Ohio, 45404, United States

  • Dipartimento di Ematologia Univ. Firenze

    Florence, 50134, Italy

  • HZRM Haemophilie-Zentrum Rhein Main GmbH

    Frankfurt am Main, 60596, Germany

  • Hamltn Hth Sci/McMstr Child Hosp

    Hamilton, Ontario, L8N 3Z5, Canada

  • Hope for Kids

    Macon, Georgia, 31201, United States

  • Hospital La Fe - Endocrinología y Nutrición

    Valencia, 46026, Spain

  • Hospital Pulau Pinang_Georgetown, Penang

    George Town, Pulau Pinang, 10450, Malaysia

  • Hospital Sant Joan de Déu

    Esplugues Llobregat, 08950, Spain

  • Hospital Tengku Ampuan Afzan

    Kuantan, Pahang, 25100, Malaysia

  • Hospital Tengku Ampuan Rahimah

    Klang, Selangor, 41200, Malaysia

  • Hospital Universitario Dr. José Eleuterio González_Monterrey

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan

    CABA, C1245AAM, Argentina

  • IRCCS Meyer Firenze

    Florence, 50139, Italy

  • Institute for Mother and Child Health Care of Serbia

    Belgrade, 11070, Serbia

  • Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery

    Lviv, 79044, Ukraine

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • Johns Hopkins University_Baltimore_3

    Baltimore, Maryland, 21205, United States

  • Kanagawa Children's Medical Center _ Hematology / Oncology

    Kanagawa, 232-8555, Japan

  • Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics

    Kaohsiung City, 807, Taiwan

  • Kent and Canterbury Hospital - Kent Haemophilia and Thrombosis Centre

    Canterbury, CT1 3NG, United Kingdom

  • King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology

    Bangkok, 10330, Thailand

  • Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)

    Klagenfurt, 9020, Austria

  • LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde

    Salzburg, 5020, Austria

  • Lady Cilento Children's Hospital

    South Brisbane, Queensland, 4101, Australia

  • Landes-Frauen und Kinderklinik Linz

    Linz, 4020, Austria

  • Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

    Leicester, LE1 5WW, United Kingdom

  • Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology

    Chiang Mai, 50200, Thailand

  • Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie

    Graz, 8036, Austria

  • Medical University Of SC

    Charleston, South Carolina, 29425, United States

  • Miller Children's Hospital Long Beach

    Long Beach, California, 90806, United States

  • Nagoya University Hospital_Blood Transfusion

    Aichi, 466-8560, Japan

  • National Blood Centre

    Kuala Lumpur, 50400, Malaysia

  • National Center for Child Health and Development_Hematology

    Tokyo, 157-8535, Japan

  • National Taiwan University Children's Hospital

    Taipei, 100, Taiwan

  • Nemours Chld Clnc Jacksonville

    Jacksonville, Florida, 32207, United States

  • North Shore Long Island Jewish Medical Center

    New Hyde Park, New York, 11040, United States

  • Novant Hlth Vasc Ins Charlotte

    Charlotte, North Carolina, 28204, United States

  • Ogikubo Hospital_Pediatries & Blood

    Tokyo, 167-0035, Japan

  • Ordination Prof. Zwiauer

    Sankt Pölten, A 3100, Austria

  • Penn State MS Hershey Med Ctr

    Hershey, Pennsylvania, 17033, United States

  • Radboudumc

    Nijmegen, 6525 GA, Netherlands

  • Ramathibodi Hospital_Paediatrics

    Bangkok, 10400, Thailand

  • Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Royal Hospital for Children

    Glasgow, G51 4TF, United Kingdom

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • SI Institute of Urgent and Recovery Surgery - Haematology

    Donetsk, 83045, Ukraine

  • Saitama Children's Med Centre_Hematology-Oncology

    Saitama, 330-8777, Japan

  • Sanatorio Mayo Privado S.A

    Córdoba, X5000FAL, Argentina

  • Shands Hospital at the University of Florida

    Gainesville, Florida, 32610, United States

  • Sheba MC - The Israeli National Hemophilia Center

    Tel Litwinsky, 52621, Israel

  • Shizuoka Children's Hospital, Hematology-Oncology

    Shizuoka, 420-8660, Japan

  • St Christopher Hosp for Child

    Philadelphia, Pennsylvania, 19134, United States

  • St Thomas' Hospital - Haemostasis and Thrombosis Centre

    London, SE1 7EH, United Kingdom

  • St. Luke's Mountain States Tumor Institute

    Boise, Idaho, 83712, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • ULS São João, E.P.E.

    Porto, 4200-319, Portugal

  • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

    Plovdiv, 4002, Bulgaria

  • Univ Hosp Cleveland Med Ctr

    Cleveland, Ohio, 44106, United States

  • Univ of NE Med Center_Omaha

    Omaha, Nebraska, 68198-6828, United States

  • Univ of Utah Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • University Children's Hospital Tirsova

    Belgrade, 11000, Serbia

  • University Hospital Saadna Abdenour of Setif

    Sétif, 19000, Algeria

  • University Of Iowa

    Iowa City, Iowa, 52242, United States

  • University of North Carlolina-Chapel Hill

    Chapel Hill, North Carolina, 27514, United States

  • University of Virginia Children's Hospital

    Charlottesville, Virginia, 22903, United States

  • Universitätsklinik Kinder-Jugendheilkunde Innsbruck

    Innsbruck, 6020, Austria

  • Universitätsklinik für Kinder- und Jugendheilkunde

    Vienna, A 1090, Austria

  • Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

    Bonn, 53127, Germany

  • Vanderbilt Hemostasis Thrombosis Clinic

    Nashville, Tennessee, 37232, United States

  • Werlhof-Institut

    Hanover, 30159, Germany

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