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

Congenital Bleeding Disorder Clinical Trial

— pathfinder™6  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02137850
• Other study ID #: NN7088-3908
• Secondary ID: 2013-004025-88U1
• Status: Completed
• Phase: Phase 3
• Start date: June 26, 2014
• Est. completion date: June 7, 2023

Study information

• Verified date: April 2024
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 125
• Est. completion date: June 7, 2023
• Est. primary completion date: June 7, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 0 Years to 6 Years

Eligibility

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

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Haemophilia A
• Hemophilia A
• Hemorrhage
• Hemostatic Disorders

Intervention

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

Locations

Country	Name	City	State
Algeria	Beni Messous Hospital Issaad Hassani	Algiers
Algeria	University Hospital Saadna Abdenour of Setif	Setif
Argentina	Sanatorio Mayo Privado S.A	Cordoba
Australia	Royal Children's Hospital	Parkville	Victoria
Australia	Lady Cilento Children's Hospital	South Brisbane	Queensland
Austria	Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie	Graz
Austria	Universitätsklinik Kinder-Jugendheilkunde	Innsbruck
Austria	Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)	Klagenfurt
Austria	Landes-Frauen und Kinderklinik Linz	Linz
Austria	LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde	Salzburg
Austria	LKH St. Poelten, Kinder-und Jugendheilkunde	St. Poelten
Austria	Universitätsklinik für Kinder- und Jugendheilkunde	Wien
Bulgaria	UMHAT "Sveti Georgi" Clinica of Pediatric	Plovdiv
Canada	Hamltn Hth Sci/McMstr Child Hosp	Hamilton	Ontario
France	Hôpital Cardiologique Louis Pradel	Bron Cedex
France	Centre régional de traitement de l'Hémophilie	Nantes Cedex 1
France	Hopital Necker	Paris
Germany	Werlhof-Institut	Hannover
Germany	Uniklinikum des Saarlandes	Homburg/Saar
Greece	Aghia Sophia Childrens' Hospital	Athens
Greece	'Ippokrateio' General Hospital of Thessaloniki	Thessaloniki
Israel	Sheba MC The Israeli National Hemophilia Center	Tel-Hashomer
Italy	AOU Meyer	Firenze
Italy	Dipartimento di Ematologia Univ. Firenze	Firenze
Italy	A.O.U. Città della Salute e della Scienza di Torino-Ospedale	Torino
Japan	Nagoya University Hospital_Blood Transfusion	Aichi
Japan	Hyogo prefectural kobe children's hospital	Hyogo
Japan	Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics	Kyoto
Japan	Saitama Children's Med Centre_Hematology-Oncology	Saitama
Japan	Shizuoka Children's Hospital, Hematology-Oncology	Shizuoka
Japan	National Center for Child Health and Development_Hematology	Tokyo
Malaysia	Hospital Pulau Pinang_Georgetown, Penang	Georgetown, Penang
Malaysia	Hospital Wanita dan Kanak-Kanak Kuala Lumpur	Kuala Lumpur
Malaysia	National Blood Centre	Kuala Lumpur
Romania	1st Paediatric Department, Fundeni Clinical Institute	Bucharest
Romania	Emergency County Hospital Constanta	Constanta
Romania	,,Louis Turcanu'' Emergency Hospital for Children	Timisoara	Timis
Spain	Hospital Teresa Herrera Materno Infantil . E.O.X.I. A Coruña	A Coruña
Spain	Hospital Sant Joan de Déu	Esplugues Llobregat
Spain	Hospital Universitario La Paz	Madrid
Taiwan	NTU Hospital - Children and Women Hospital	Taipei
Thailand	Ramathibodi Hospital_Bangkok	Bangkok
Thailand	Hematology and Oncology, Dept.of Pediatrics, CMU	Chiang Mai
Thailand	Sunpasitthiprasong Hospital	Ubon Ratchathani
United States	Torrence Hemby Ped Hem/Onc Ctr	Charlotte	North Carolina
United States	Rush University Med. Cntr	Chicago	Illinois
United States	Dayton's Children's Hemostasis and Thrombosis Center	Dayton	Ohio
United States	University Of Iowa	Iowa City	Iowa
United States	Children's Hosp-Los Angeles	Los Angeles	California
United States	North Shore Long Island Jewish Medical Center	New Hyde Park	New York
United States	Univ Oklahoma Sci Ctr OK City	Oklahoma City	Oklahoma
United States	Arizona H&T Phoenix Child Hosp	Phoenix	Arizona
United States	Univ of Utah Primary Children's Hospital	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Algeria,  Argentina,  Australia,  Austria,  Bulgaria,  Canada,  France,  Germany,  Greece,  Israel,  Italy,  Japan,  Malaysia,  Romania,  Spain,  Taiwan,  Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)		When the first 50 PUP have reached at least 50 exposure dates. (Expected to reach between 6 - 18 months)
Primary	Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)		At the end of the trial. End of trial will be up to 4 years after the patient has reached 100 exposure dates. (Expected to reach between 12 - 60 months)
Secondary	Frequency of adverse events including serious adverse events and medical events of special interest		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU)		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate)		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none")		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year)		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode)		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days
Secondary	Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other")		When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days

External Links

•   Clinical Trials at Novo Nordisk