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New hope for kids with hemophilia: Long-Acting clotting factor shows promise

NCT ID NCT02137850

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 trial tested a long-acting clotting factor called N8-GP in 124 young children (under 6 years old) with severe hemophilia A who had never been treated before. The goal was to see if it safely prevents and treats bleeding episodes. The study found that N8-GP helped control bleeding and was generally safe, though some children developed antibodies that could reduce its effectiveness.

What this could mean

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

Active substance
turoctocog alfa pegol (N8-GP), a lab-made clotting factor given by IV injection
What this could lead to
If successful, this could provide a safe and effective treatment option to prevent and stop bleeding episodes in young children with severe hemophilia A.
What could go wrong
This is a single-arm, non-controlled trial, so results may not compare directly to other treatments. Some children may still develop inhibitors that reduce 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

124 people

The number who actually took part.

Started

Jun 2014

Finished

Jun 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 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 of age at the time of signing informed consent * Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records or central laboratory results * No prior use of purified clotting factor products (5 previous exposures to blood components is acceptable) Exclusion Criteria: * Any history of FVIII inhibitor (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 A * 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')/legally acceptable representative (LAR(s')) 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 A hemorrhagic disease severe hemophilia A

As listed by the trial registrant

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

Congenital bleeding disorder Haemophilia A

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

  • A.O.U. Città della Salute e della Scienza di Torino-Ospedale

    Torino, 10126, Italy

  • Aghia Sophia Childrens' Hospital

    Athens, GR-11527, Greece

  • Ap-Hp-Hopital Necker-1

    Paris, 75015, France

  • Arizona H&T Phoenix Child Hosp

    Phoenix, Arizona, 85016-7710, United States

  • Beni Messous Hospital Issaad Hassani

    Algiers, 16000, Algeria

  • CHU Estaing

    Clermont-Ferrand, 63003, France

  • Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

    Nantes, 44093, 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_Hematology Dept.

    Changhua, 500, Taiwan

  • Children's Hosp Of Orange

    Orange, California, 92868-3835, United States

  • 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 Hosptl Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hsptl Of The Kings

    Norfolk, Virginia, 23507, United States

  • China Medical University Children's Hospital

    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

  • Emergency County Hospital Constanta

    Constanța, 900591, Romania

  • Emory University_Atlanta_1

    Atlanta, Georgia, 30322, United States

  • HZRM Haemophilie-Zentrum Rhein Main GmbH

    Frankfurt am Main, 60596, Germany

  • Hamltn Hth Sci/McMstr Child Hosp

    Hamilton, Ontario, L8N 3Z5, Canada

  • Harbor-UCLA Medical Center

    Torrance, California, 90502-2004, United States

  • Health Science Centre

    St. John's, Newfoundland and Labrador, A1B 3V6, Canada

  • Hemostasis and Thrombosis Center of Nevada

    Las Vegas, Nevada, 89113, United States

  • Hospices Civils de Lyon- Hopital Louis Pradel-1

    Bron, 69500, France

  • Hospital Pulau Pinang_Georgetown, Penang

    George Town, Pulau Pinang, 10450, Malaysia

  • Hospital Sant Joan de Déu

    Esplugues Llobregat, 08950, Spain

  • Hospital Teresa Herrera Materno Infantil . E.O.X.I. A Coruña

    A Coruña, 15006, Spain

  • Hospital Universitario Dr. José Eleuterio González_Monterrey

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Wanita dan Kanak-Kanak Kuala Lumpur

    Kuala Lumpur, 50300, Malaysia

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

    CABA, C1245AAM, Argentina

  • Hyogo prefectural kobe children's hospital

    Hyōgo, 654-0047, Japan

  • IRCCS Meyer Firenze

    Florence, 50139, Italy

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

    Lviv, 79044, Ukraine

  • Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics

    Kaohsiung City, 807, Taiwan

  • Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)

    Klagenfurt, 9020, Austria

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

    Salzburg, 5020, Austria

  • LKH St. Poelten, Kinder-und Jugendheilkunde

    Sankt Pölten, 3100, Austria

  • Lady Cilento Children's Hospital

    South Brisbane, Queensland, 4101, Australia

  • Landes-Frauen und Kinderklinik Linz

    Linz, 4020, Austria

  • Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology

    Chiang Mai, 50200, Thailand

  • Master centre for France

    Paris La Défense, 92936, France

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

    Graz, 8036, Austria

  • Medical College Of Georgia

    Augusta, Georgia, 30912, United States

  • 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 Child Orlando Hem/Onc

    Orlando, Florida, United States

  • Nemours Chld Clnc Jacksonville

    Jacksonville, Florida, 32207, United States

  • North Shore Long Island Jewish Medical Center

    New Hyde Park, New York, 11042, United States

  • Novant Hlth Vasc Ins Charlotte

    Charlotte, North Carolina, 28204, United States

  • Oregon Health & Science University_Portland_5

    Portland, Oregon, 97239, United States

  • Pediatric Hemophilia Program University

    San Juan, 00935, Puerto Rico

  • Ramathibodi Hospital_Paediatrics

    Bangkok, 10400, Thailand

  • Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Rush University Med. Cntr

    Chicago, Illinois, 60612, United States

  • Rutgers-Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 08901, United States

  • 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 Joseph's Hospital Foundation

    Tampa, Florida, 33607, United States

  • St. Luke's Mountain States Tumor Institute

    Boise, Idaho, 83712, United States

  • Sunpasitthiprasong Hospital_Pediatrics Department

    Ubon Ratchathani, Mueang Distirct,, 34000, Thailand

  • The Brooklyn Hsptl Center

    Brooklyn, New York, 11201-5425, United States

  • Torrence Hemby Ped Hem/Onc Ctr

    Charlotte, North Carolina, 28203, United States

  • Tulane University School of Medicine

    New Orleans, Louisiana, 70112, United States

  • 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 Oklahoma Sci Ctr OK City

    Oklahoma City, Oklahoma, 73104, 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

  • Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics

    Kyoto, 602-8566, Japan

  • 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 Virginia Hospitl

    Charlottesville, Virginia, 22908, United States

  • Universitätsklinik Kinder-Jugendheilkunde Innsbruck

    Innsbruck, 6020, Austria

  • Universitätsklinik für Kinder- und Jugendheilkunde

    Vienna, A 1090, Austria

  • Universitätsklinikum des Saarlandes - Pädiatrische Onkologie und Hämatologie

    Homburg/Saar, 66424, Germany

  • Vanderbilt Hemostasis Thrombosis Clinic

    Nashville, Tennessee, 37232, United States

  • Werlhof-Institut

    Hanover, 30159, Germany

  • Werlhof-Institut

    Hanover, Germany

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