Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A

NCT02137850 | Completed Phase 3 Results

• 5 other identifiers: NN7088-39082013-004025-88U1111-1148-1897REec-2014-0898JapicCTI-142577
• Study Type: interventional
• Target: 124
• Locations: 22 countries
• Sites: 94

Brief Summary

This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.

Conditions

Haemophilia A; Congenital Bleeding Disorder

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)

  Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.

  From start of the treatment up to 7 years

Secondary Outcomes (10)

• Number of Adverse Events Including Serious Adverse Events and Medical Events of Special Interest

  From start of the treatment up to 8.9 years

• Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU)

  From start of the treatment up to 8.9 years

• Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate)

  From start of the treatment up to 8.9 years

• Haemostatic Effect of N8-GP in Treatment of Bleeding Episodes, Assessed by a Predefined 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "None")

  From start of the treatment up to 8.9 years

• Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg))

  From start of the treatment up to 8.9 years

Study Arms (1)

50 EDs (exposure days)

EXPERIMENTAL

Drug: turoctocog alfa pegol

Interventions

turoctocog alfa pegol DRUG

For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis

Also known as: NNC 0129-0000-1003, N8-GP

50 EDs (exposure days)

Eligibility Criteria

• Age: 0 Years - 6 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (96)

Arizona H&T Phoenix Child Hosp

Phoenix, Arizona, 85016-7710, United States

Location

Miller Children's Hospital Long Beach

Long Beach, California, 90806, United States

Location

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

Location

Children's Hosp Of Orange

Orange, California, 92868-3835, United States

Location

Harbor-UCLA Medical Center

Torrance, California, 90502-2004, United States

Location

Shands Hospital at the University of Florida

Gainesville, Florida, 32610, United States

Location

Nemours Chld Clnc Jacksonville

Jacksonville, Florida, 32207, United States

Location

Nemours Child Orlando Hem/Onc

Orlando, Florida, United States

Location

St Joseph's Hospital Foundation

Tampa, Florida, 33607, United States

Location

Emory University_Atlanta_1

Atlanta, Georgia, 30322, United States

Location

Medical College Of Georgia

Augusta, Georgia, 30912, United States

Location

St. Luke's Mountain States Tumor Institute

Boise, Idaho, 83712, United States

Location

Rush University Med. Cntr

Chicago, Illinois, 60612, United States

Location

University Of Iowa

Iowa City, Iowa, 52242, United States

Location

Tulane University School of Medicine

New Orleans, Louisiana, 70112, United States

Location

Children's Hosp-New Orleans

New Orleans, Louisiana, 70118-5720, United States

Location

Univ of NE Med Center_Omaha

Omaha, Nebraska, 68198-6828, United States

Location

Hemostasis and Thrombosis Center of Nevada

Las Vegas, Nevada, 89113, United States

Location

Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

The Brooklyn Hsptl Center

Brooklyn, New York, 11201-5425, United States

Location

North Shore Long Island Jewish Medical Center

New Hyde Park, New York, 11042, United States

Location

Torrence Hemby Ped Hem/Onc Ctr

Charlotte, North Carolina, 28203, United States

Location

Novant Hlth Vasc Ins Charlotte

Charlotte, North Carolina, 28204, United States

Location

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, 44106, United States

Location

Dayton Children Hemostati Ctr

Dayton, Ohio, 45404, United States

Location

Univ Oklahoma Sci Ctr OK City

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health & Science University_Portland_5

Portland, Oregon, 97239, United States

Location

Children's Hosptl Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

St Christopher Hosp for Child

Philadelphia, Pennsylvania, 19134, United States

Location

Medical University Of SC

Charleston, South Carolina, 29425, United States

Location

Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, 37232, United States

Location

Univ of Utah Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

University Of Virginia Hospitl

Charlottesville, Virginia, 22908, United States

Location

Children's Hsptl Of The Kings

Norfolk, Virginia, 23507, United States

Location

Beni Messous Hospital Issaad Hassani

Algiers, 16000, Algeria

Location

University Hospital Saadna Abdenour of Setif

Sétif, 19000, Algeria

Location

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

CABA, C1245AAM, Argentina

Location

Sanatorio Mayo Privado S.A

Córdoba, X5000FAL, Argentina

Location

Lady Cilento Children's Hospital

South Brisbane, Queensland, 4101, Australia

Location

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

Location

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

Graz, 8036, Austria

Location

Universitätsklinik Kinder-Jugendheilkunde Innsbruck

Innsbruck, 6020, Austria

Location

Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)

Klagenfurt, 9020, Austria

Location

Landes-Frauen und Kinderklinik Linz

Linz, 4020, Austria

Location

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

Salzburg, 5020, Austria

Location

LKH St. Poelten, Kinder-und Jugendheilkunde

Sankt Pölten, 3100, Austria

Location

Universitätsklinik für Kinder- und Jugendheilkunde

Vienna, A 1090, Austria

Location

UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

Plovdiv, 4002, Bulgaria

Location

Health Science Centre

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

Location

Hamltn Hth Sci/McMstr Child Hosp

Hamilton, Ontario, L8N 3Z5, Canada

Location

Hospices Civils de Lyon- Hopital Louis Pradel-1

Bron, 69500, France

Location

CHU Estaing

Clermont-Ferrand, 63003, France

Location

Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

Nantes, 44093, France

Location

Ap-Hp-Hopital Necker-1

Paris, 75015, France

Location

Master centre for France

Paris La Défense, 92936, France

Location

Coagulation Research Center

Duisburg, 47051, Germany

Location

HZRM Haemophilie-Zentrum Rhein Main GmbH

Frankfurt am Main, 60596, Germany

Location

Werlhof-Institut

Hanover, 30159, Germany

Location

Werlhof-Institut

Hanover, Germany

Location

Universitätsklinikum des Saarlandes - Pädiatrische Onkologie und Hämatologie

Homburg/Saar, 66424, Germany

Location

Aghia Sophia Childrens' Hospital

Athens, GR-11527, Greece

Location

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

Location

Sheba MC - The Israeli National Hemophilia Center

Tel Litwinsky, 52621, Israel

Location

Dipartimento di Ematologia Univ. Firenze

Florence, 50134, Italy

Location

IRCCS Meyer Firenze

Florence, 50139, Italy

Location

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

Torino, 10126, Italy

Location

Nagoya University Hospital_Blood Transfusion

Aichi, 466-8560, Japan

Location

Hyogo prefectural kobe children's hospital

Hyōgo, 654-0047, Japan

Location

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

Kyoto, 602-8566, Japan

Location

Saitama Children's Med Centre_Hematology-Oncology

Saitama, 330-8777, Japan

Location

Shizuoka Children's Hospital, Hematology-Oncology

Shizuoka, 420-8660, Japan

Location

National Center for Child Health and Development_Hematology

Tokyo, 157-8535, Japan

Location

Hospital Pulau Pinang_Georgetown, Penang

George Town, Pulau Pinang, 10450, Malaysia

Location

Hospital Wanita dan Kanak-Kanak Kuala Lumpur

Kuala Lumpur, 50300, Malaysia

Location

National Blood Centre

Kuala Lumpur, 50400, Malaysia

Location

Centro Medico Nacional SXXI-Hospital de Pediatria, IMSS

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

Location

Hospital Universitario Dr. José Eleuterio González_Monterrey

Monterrey, Nuevo León, 64460, Mexico

Location

Centro Hospitalar Lisboa Norte-HSM

Lisbon, 1649-035, Portugal

Location

ULS São João, E.P.E.

Porto, 4200-319, Portugal

Location

Pediatric Hemophilia Program University

San Juan, 00935, Puerto Rico

Location

,,Louis Ţurcanu'' Emergency Hospital for Children

Timișoara, Timiș County, 300011, Romania

Location

1st Paediatric Department, Fundeni Clinical Institute

Bucharest, 022328, Romania

Location

Emergency County Hospital Constanta

Constanța, 900591, Romania

Location

University Children's Hospital Tirsova

Belgrade, 11000, Serbia

Location

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

A Coruña, 15006, Spain

Location

Hospital Sant Joan de Déu

Esplugues Llobregat, 08950, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Changhua Christian Hospital_Hematology Dept.

Changhua, 500, Taiwan

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics

Kaohsiung City, 807, Taiwan

Location

China Medical University Children's Hospital

Taichung, 40447, Taiwan

Location

National Taiwan University Children's Hospital

Taipei, 100, Taiwan

Location

Sunpasitthiprasong Hospital_Pediatrics Department

Ubon Ratchathani, Mueang Distirct,, 34000, Thailand

Location

Ramathibodi Hospital_Paediatrics

Bangkok, 10400, Thailand

Location

Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology

Chiang Mai, 50200, Thailand

Location

SI Institute of Urgent and Recovery Surgery - Haematology

Donetsk, 83045, Ukraine

Location

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

Lviv, 79044, Ukraine

Location

Related Publications (2)

• Kenet G, Young G, Chuansumrit A, Matsushita T, Yadav V, Zak M, Male C. The immunogenicity, safety, and efficacy of N8-GP in previously untreated patients with severe hemophilia A: pathfinder6 end-of-trial results. J Thromb Haemost. 2023 Nov;21(11):3109-3116. doi: 10.1016/j.jtha.2023.07.030. Epub 2023 Aug 18.

  PMID: 37597724 DERIVED

• Male C, Konigs C, Dey S, Matsushita T, Millner AH, Zak M, Young G, Kenet G. The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A. Blood Adv. 2023 Feb 28;7(4):620-629. doi: 10.1182/bloodadvances.2022007529.

  PMID: 35858373 DERIVED

Related Links

• Clinical Trials at Novo Nordisk

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Clinical Reporting Office (2834)
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

866-867-7178

Study Officials

• Global Clinical Registry (GCR, 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2014
• First Posted: May 14, 2014
• Study Start: June 26, 2014
• Primary Completion: June 7, 2023
• Study Completion: June 7, 2023
• Last Updated: December 23, 2025
• Results First Posted: October 31, 2024

Record last verified: 2025-12

Locations

AL (2); ES (3); RO (3); IL (1); AU (7); TW (4); TH (3); JP (6); IT (3); PT (2); CA (2); GR (2); FR (5); US (34); SE (1); UA (2); PR (1); AR (2); ME (2); DE (5); BU (1); MY (3)