Clinical trial NCT02137850: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2014-05-14
Enrollment: 124
Sex: MALE
Minimum age (years): 0
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)
timeFrame: From start of the treatment up to 7 years
Secondary outcomes:
measure: Number of Adverse Events Including Serious Adverse Events and Medical Events of Special Interest
timeFrame: From start of the treatment up to 8.9 years
measure: Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU)
timeFrame: From start of the treatment up to 8.9 years
measure: Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate)
timeFrame: From start of the treatment up to 8.9 years
measure: Haemostatic Effect of N8-GP in Treatment of Bleeding Episodes, Assessed by a Predefined 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "None")
timeFrame: From start of the treatment up to 8.9 years
measure: Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg))
timeFrame: From start of the treatment up to 8.9 years
measure: Consumption of N8-GP for Prophylaxis (Number of Injections)
timeFrame: From start of the treatment up to 8.9 years
measure: Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed))
timeFrame: From start of the treatment up to 8.9 years
measure: Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections)
timeFrame: From start of the treatment up to 8.9 years
measure: Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value
timeFrame: From start of the treatment up to 8.9 years
measure: Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other")
timeFrame: From start of the treatment up to 8.9 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Arizona H&T Phoenix Child Hosp
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles - Endocrinology
state: California
city: Chicago
country: United States
facility: Rush University Med. Cntr
state: Illinois
city: Iowa City
country: United States
facility: University Of Iowa
state: Iowa
city: New Hyde Park
country: United States
facility: North Shore Long Island Jewish Medical Center
state: New York
city: Charlotte
country: United States
facility: Torrence Hemby Ped Hem/Onc Ctr
state: North Carolina
city: Dayton
country: United States
facility: Dayton's Children's Hemostasis and Thrombosis Center
state: Ohio
city: Oklahoma City
country: United States
facility: Univ Oklahoma Sci Ctr OK City
state: Oklahoma
city: Salt Lake City
country: United States
facility: Univ of Utah Primary Children's Hospital
state: Utah
city: Algiers
country: Algeria
facility: Beni Messous Hospital Issaad Hassani
city: Setif
country: Algeria
facility: University Hospital Saadna Abdenour of Setif
city: Cordoba
country: Argentina
facility: Sanatorio Mayo Privado S.A
city: South Brisbane
country: Australia
facility: Lady Cilento Children's Hospital
state: Queensland
city: Parkville
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Graz
country: Austria
facility: Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie
city: Innsbruck
country: Austria
facility: Universitätsklinik Kinder-Jugendheilkunde
city: Klagenfurt
country: Austria
facility: Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)
city: Linz
country: Austria
facility: Landes-Frauen und Kinderklinik Linz
city: Salzburg
country: Austria
facility: LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde
city: St. Poelten
country: Austria
facility: LKH St. Poelten, Kinder-und Jugendheilkunde
city: Wien
country: Austria
facility: Universitätsklinik für Kinder- und Jugendheilkunde
city: Plovdiv
country: Bulgaria
facility: UMHAT "Sveti Georgi" Clinica of Pediatric
city: Hamilton
country: Canada
facility: Hamltn Hth Sci/McMstr Child Hosp
state: Ontario
city: Bron Cedex
country: France
facility: Hôpital Cardiologique Louis Pradel
city: Nantes Cedex 1
country: France
facility: Centre régional de traitement de l'Hémophilie
city: Paris
country: France
facility: Hopital Necker
city: Hannover
country: Germany
facility: Werlhof-Institut
city: Homburg/Saar
country: Germany
facility: Uniklinikum des Saarlandes
city: Athens
country: Greece
facility: Aghia Sophia Childrens' Hospital
city: Thessaloniki
country: Greece
facility: 'Ippokrateio' General Hospital of Thessaloniki
city: Tel-Hashomer
country: Israel
facility: Sheba MC The Israeli National Hemophilia Center
city: Firenze
country: Italy
facility: Dipartimento di Ematologia Univ. Firenze
city: Firenze
country: Italy
facility: AOU Meyer
city: Torino
country: Italy
facility: A.O.U. Città della Salute e della Scienza di Torino-Ospedale
city: Aichi
country: Japan
facility: Nagoya University Hospital_Blood Transfusion
city: Hyogo
country: Japan
facility: Hyogo prefectural kobe children's hospital
city: Kyoto
country: Japan
facility: Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics
city: Saitama
country: Japan
facility: Saitama Children's Med Centre_Hematology-Oncology
city: Shizuoka
country: Japan
facility: Shizuoka Children's Hospital, Hematology-Oncology
city: Tokyo
country: Japan
facility: National Center for Child Health and Development_Hematology
city: Georgetown, Penang
country: Malaysia
facility: Hospital Pulau Pinang_Georgetown, Penang
city: Kuala Lumpur
country: Malaysia
facility: Hospital Wanita dan Kanak-Kanak Kuala Lumpur
city: Kuala Lumpur
country: Malaysia
facility: National Blood Centre
city: Timisoara
country: Romania
facility: ,,Louis Ţurcanu'' Emergency Hospital for Children
state: Timis
city: Bucharest
country: Romania
facility: 1st Paediatric Department, Fundeni Clinical Institute
city: Constanta
country: Romania
facility: Emergency County Hospital Constanta
city: A Coruña
country: Spain
facility: Hospital Teresa Herrera Materno Infantil . E.O.X.I. A Coruña
city: Esplugues Llobregat
country: Spain
facility: Hospital Sant Joan de Déu
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Taipei
country: Taiwan
facility: NTU Hospital - Children and Women Hospital
city: Ubon Ratchathani
country: Thailand
facility: Sunpasitthiprasong Hospital
state: Mueang Distirct,
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital_Bangkok
city: Chiang Mai
country: Thailand
facility: Hematology and Oncology, Dept.of Pediatrics, CMU
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: