Congenital Bleeding DisorderPhase 3pathfinder™6NCT02137850
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
A Phase 3 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 96 sites in 23 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 6 years, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A
- What is being tested?
- turoctocog alfa pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 124 participants
- Where
- 96 sitesin 23 countries
- Study dates
- 26 Jun 2014to 7 Jun 2023
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
96 sites in 23 countries.
96 sites in 23 countries
United States34 sites
Emory University_Atlanta_1
Atlanta, Georgia, 30322
Medical College Of Georgia
Augusta, Georgia, 30912
St. Luke's Mountain States Tumor Institute
Boise, Idaho, 83712
The Brooklyn Hsptl Center
Brooklyn, New York, 11201-5425
Medical University Of SC
Charleston, South Carolina, 29425
Torrence Hemby Ped Hem/Onc Ctr
Charlotte, North Carolina, 28203
Novant Hlth Vasc Ins Charlotte
Charlotte, North Carolina, 28204
University Of Virginia Hospitl
Charlottesville, Virginia, 22908
Rush University Med. Cntr
Chicago, Illinois, 60612
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, 44106
Dayton Children Hemostati Ctr
Dayton, Ohio, 45404
Shands Hospital at the University of Florida
Gainesville, Florida, 32610
University Of Iowa
Iowa City, Iowa, 52242
Nemours Chld Clnc Jacksonville
Jacksonville, Florida, 32207
Hemostasis and Thrombosis Center of Nevada
Las Vegas, Nevada, 89113
Miller Children's Hospital Long Beach
Long Beach, California, 90806
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
Vanderbilt Hemostasis Thrombosis Clinic
Nashville, Tennessee, 37232
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
North Shore Long Island Jewish Medical Center
New Hyde Park, New York, 11042
Tulane University School of Medicine
New Orleans, Louisiana, 70112
Children's Hosp-New Orleans
New Orleans, Louisiana, 70118-5720
Children's Hsptl Of The Kings
Norfolk, Virginia, 23507
Univ Oklahoma Sci Ctr OK City
Oklahoma City, Oklahoma, 73104
Univ of NE Med Center_Omaha
Omaha, Nebraska, 68198-6828
Children's Hosp Of Orange
Orange, California, 92868-3835
Nemours Child Orlando Hem/Onc
Orlando, Florida
Children's Hosptl Philadelphia
Philadelphia, Pennsylvania, 19104
St Christopher Hosp for Child
Philadelphia, Pennsylvania, 19134
Arizona H&T Phoenix Child Hosp
Phoenix, Arizona, 85016-7710
Oregon Health & Science University_Portland_5
Portland, Oregon, 97239
Univ of Utah Primary Children's Hospital
Salt Lake City, Utah, 84113
St Joseph's Hospital Foundation
Tampa, Florida, 33607
Harbor-UCLA Medical Center
Torrance, California, 90502-2004
Austria7 sites
Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie
Graz, 8036
Universitätsklinik Kinder-Jugendheilkunde Innsbruck
Innsbruck, 6020
Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)
Klagenfurt, 9020
Landes-Frauen und Kinderklinik Linz
Linz, 4020
LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde
Salzburg, 5020
LKH St. Poelten, Kinder-und Jugendheilkunde
Sankt Pölten, 3100
Universitätsklinik für Kinder- und Jugendheilkunde
Vienna, A 1090
Japan6 sites
Nagoya University Hospital_Blood Transfusion
Aichi, 466-8560
Hyogo prefectural kobe children's hospital
Hyōgo, 654-0047
Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics
Kyoto, 602-8566
Saitama Children's Med Centre_Hematology-Oncology
Saitama, 330-8777
Shizuoka Children's Hospital, Hematology-Oncology
Shizuoka, 420-8660
National Center for Child Health and Development_Hematology
Tokyo, 157-8535
France5 sites
Hospices Civils de Lyon- Hopital Louis Pradel-1
Bron, 69500
CHU Estaing
Clermont-Ferrand, 63003
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, 44093
Ap-Hp-Hopital Necker-1
Paris, 75015
Master centre for France
Paris La Défense, 92936
Germany5 sites
Coagulation Research Center
Duisburg, 47051
HZRM Haemophilie-Zentrum Rhein Main GmbH
Frankfurt am Main, 60596
Werlhof-Institut
Hanover, 30159
Werlhof-Institut
Hanover
Universitätsklinikum des Saarlandes - Pädiatrische Onkologie und Hämatologie
Homburg/Saar, 66424
Taiwan4 sites
Changhua Christian Hospital_Hematology Dept.
Changhua, 500
Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics
Kaohsiung City, 807
China Medical University Children's Hospital
Taichung, 40447
National Taiwan University Children's Hospital
Taipei, 100
Italy3 sites
Dipartimento di Ematologia Univ. Firenze
Florence, 50134
IRCCS Meyer Firenze
Florence, 50139
A.O.U. Città della Salute e della Scienza di Torino-Ospedale
Torino, 10126
Malaysia3 sites
Hospital Pulau Pinang_Georgetown, Penang
George Town, Pulau Pinang, 10450
Hospital Wanita dan Kanak-Kanak Kuala Lumpur
Kuala Lumpur, 50300
National Blood Centre
Kuala Lumpur, 50400
Romania3 sites
1st Paediatric Department, Fundeni Clinical Institute
Bucharest, 022328
Emergency County Hospital Constanta
Constanța, 900591
,,Louis Ţurcanu'' Emergency Hospital for Children
Timișoara, Timiș County, 300011
Spain3 sites
Hospital Teresa Herrera Materno Infantil . E.O.X.I. A Coruña
A Coruña, 15006
Hospital Sant Joan de Déu
Esplugues Llobregat, 08950
Hospital Universitario La Paz
Madrid, 28046
Thailand3 sites
Ramathibodi Hospital_Paediatrics
Bangkok, 10400
Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology
Chiang Mai, 50200
Sunpasitthiprasong Hospital_Pediatrics Department
Ubon Ratchathani, Mueang Distirct,, 34000
Algeria2 sites
Beni Messous Hospital Issaad Hassani
Algiers, 16000
University Hospital Saadna Abdenour of Setif
Sétif, 19000
Argentina2 sites
Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan
CABA, C1245AAM
Sanatorio Mayo Privado S.A
Córdoba, X5000FAL
Australia2 sites
Royal Children's Hospital
Parkville, Victoria, 3052
Lady Cilento Children's Hospital
South Brisbane, Queensland, 4101
Canada2 sites
Hamltn Hth Sci/McMstr Child Hosp
Hamilton, Ontario, L8N 3Z5
Health Science Centre
St. John's, Newfoundland and Labrador, A1B 3V6
Greece2 sites
Aghia Sophia Childrens' Hospital
Athens, GR-11527
'Ippokrateio' General Hospital of Thessaloniki
Thessaloniki, 54642
Mexico2 sites
Centro Medico Nacional SXXI-Hospital de Pediatria, IMSS
Mexico City, México, D.F., 06720
Hospital Universitario Dr. José Eleuterio González_Monterrey
Monterrey, Nuevo León, 64460
Portugal2 sites
Centro Hospitalar Lisboa Norte-HSM
Lisbon, 1649-035
ULS São João, E.P.E.
Porto, 4200-319
Ukraine2 sites
SI Institute of Urgent and Recovery Surgery - Haematology
Donetsk, 83045
Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery
Lviv, 79044
Bulgaria1 site
UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics
Plovdiv, 4002
Israel1 site
Sheba MC - The Israeli National Hemophilia Center
Tel Litwinsky, 52621
About this study
Official title: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A
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
- Congenital Bleeding Disorder, Haemophilia A
- Being tested
- turoctocog alfa pegol
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
turoctocog alfa pegolDrug
Also called NNC 0129-0000-1003, N8-GP
For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis
| Study group | Role | What they receive |
|---|---|---|
| 50 EDs (exposure days) | Experimental | Not described Receives: Drug: turoctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Number of adverse events including serious adverse events and medical events of special interest reported during the main and extension phase of the trial. An adverse event (AE) is any untoward medical occurrence in a patient administered a product, and which does not necessarily have a causal relationship with this treatment. Serious adverse event (SAE) is an experience that at any dose results in any of the following: Death, a life-threatening experience, in-patient hospitalisation or prolongation of existing hospitalisation, persistent or significant disability or incapacity, congenital anomaly or birth defect and important medical events that may not result in death, be life threatening or require hospitalisation. Medical event of special interest (MESI) is an event which, in the evaluation of safety, has a special focus.
Measured: 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)
Number of participants with confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU) was reported during the main and extension phase of the trial.
Measured: From start of the treatment up to 8.9 years
Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate)
The number of bleeding episodes per year reported during the prophylactic treatment with N8-GP was reported.
Measured: 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")
Haemostatic effect of turoctocog alfa pegol for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hours after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. Number of bleeding episodes in each category is reported.
Measured: From start of the treatment up to 8.9 years
Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg))
Consumption of N8-GP for prophylaxis is reported. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Average dose consumption in IU/kg during main and extension phase is reported.
Measured: From start of the treatment up to 8.9 years
Consumption of N8-GP for Prophylaxis (Number of Injections)
Consumption of N8-GP for prophylaxis is reported. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Number of injections consumption per patient during main and extension phase is reported.
Measured: From start of the treatment up to 8.9 years
Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed))
Consumption of N8-GP for treatment of bleeding episodes (IU/kg/bleed) is reported. Average dose consumption in IU/kg/bleed during main and extension phase is reported.
Measured: From start of the treatment up to 8.9 years
Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections)
Consumption of N8-GP for treatment of bleeding episodes (number of injections) is reported. Number of average injections required for treatment of per bleed is reported.
Measured: From start of the treatment up to 8.9 years
Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) annualised value is presented. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Consumption used for treatment per year per patient (IU/kg/year/patient) is reported.
Measured: From start of the treatment up to 8.9 years
Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other")
The outcome of ITI was evaluated by predefined 4-point outcome of ITI scale ('success', 'partial success', 'failure', 'other'). Success: An inhibitor titre less than (<) 0.6 BU. A normalised FVIII recovery, defined as greater than or equal to (≥) 66 percentage (%) of expected incremental recovery. An N8-GP half-life (t½) ≥9 hours after a 72-hour treatment-free washout period. Partial success: Reduction in inhibitor titre to less than or equal to (≤) 5 BU. Clinical effect of N8-GP therapy as judged by the investigator. Failure: Failure to attain defined success or partial success within 24 months of uninterrupted ITI with N8-GP. Inhibitor decrease less than (<) 20% after one year of ITI treatment. Other: Not fulfilling the above criteria. Number of participants in each category is reported.
Measured: From start of the treatment up to 8.9 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2014 (estimated)
- Study start26 Jun 2014
- Primary completion7 Jun 2023
- Study completion7 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Bleeding DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 124 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02137850
- Sponsor's protocol ID
- NN7088-3908
- Other IDs
- 2013-004025-88, U1111-1148-1897, REec-2014-0898, JapicCTI-142577
- Acronym
- pathfinder™6
- Status last verified
- Dec 2025
- First submitted
- 28 Mar 2014
- Last updated
- 23 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 37597724Male 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.
PubMed 35858373