Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
- Conditions
- Congenital Bleeding DisorderHaemophilia A
- Interventions
- Registration Number
- NCT02137850
- Lead Sponsor
- Novo Nordisk A/S
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 124
- 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)
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description 50 EDs (exposure days) turoctocog alfa pegol -
- Primary Outcome Measures
Name Time Method Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) From start of the treatment up to 7 years Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.
- Secondary Outcome Measures
Name Time Method 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 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.
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 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.
Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate) From start of the treatment up to 8.9 years The number of bleeding episodes per year reported during the prophylactic treatment with N8-GP was reported.
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 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.
Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg)) From start of the treatment up to 8.9 years 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.
Consumption of N8-GP for Prophylaxis (Number of Injections) From start of the treatment up to 8.9 years 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.
Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed)) From start of the treatment up to 8.9 years 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.
Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections) From start of the treatment up to 8.9 years 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.
Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value 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 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.
Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other") From start of the treatment up to 8.9 years 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.
Trial Locations
- Locations (53)
Children's Hospital Los Angeles - Endocrinology
🇺🇸Los Angeles, California, United States
Arizona H&T Phoenix Child Hosp
🇺🇸Phoenix, Arizona, United States
Rush University Med. Cntr
🇺🇸Chicago, Illinois, United States
University Of Iowa
🇺🇸Iowa City, Iowa, United States
North Shore Long Island Jewish Medical Center
🇺🇸New Hyde Park, New York, United States
Torrence Hemby Ped Hem/Onc Ctr
🇺🇸Charlotte, North Carolina, United States
Dayton's Children's Hemostasis and Thrombosis Center
🇺🇸Dayton, Ohio, United States
Univ Oklahoma Sci Ctr OK City
🇺🇸Oklahoma City, Oklahoma, United States
Univ of Utah Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Beni Messous Hospital Issaad Hassani
🇩🇿Algiers, Algeria
University Hospital Saadna Abdenour of Setif
🇩🇿Setif, Algeria
Sanatorio Mayo Privado S.A
🇦🇷Cordoba, Argentina
Lady Cilento Children's Hospital
🇦🇺South Brisbane, Queensland, Australia
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
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, Austria
UMHAT "Sveti Georgi" Clinica of Pediatric
🇧🇬Plovdiv, Bulgaria
Hamltn Hth Sci/McMstr Child Hosp
🇨🇦Hamilton, Ontario, Canada
Hôpital Cardiologique Louis Pradel
🇫🇷Bron Cedex, France
Centre régional de traitement de l'Hémophilie
🇫🇷Nantes Cedex 1, France
Hopital Necker
🇫🇷Paris, France
Werlhof-Institut
🇩🇪Hannover, Germany
Uniklinikum des Saarlandes
🇩🇪Homburg/Saar, Germany
Aghia Sophia Childrens' Hospital
🇬🇷Athens, Greece
'Ippokrateio' General Hospital of Thessaloniki
🇬🇷Thessaloniki, Greece
Sheba MC The Israeli National Hemophilia Center
🇮🇱Tel-Hashomer, Israel
Dipartimento di Ematologia Univ. Firenze
🇮🇹Firenze, Italy
AOU Meyer
🇮🇹Firenze, Italy
A.O.U. Città della Salute e della Scienza di Torino-Ospedale
🇮🇹Torino, Italy
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, Japan
Hospital Pulau Pinang_Georgetown, Penang
🇲🇾Georgetown, Penang, Malaysia
Hospital Wanita dan Kanak-Kanak Kuala Lumpur
🇲🇾Kuala Lumpur, Malaysia
National Blood Centre
🇲🇾Kuala Lumpur, Malaysia
NTU Hospital - Children and Women Hospital
🇨🇳Taipei, Taiwan
,,Louis Ţurcanu'' Emergency Hospital for Children
🇷🇴Timisoara, Timis, Romania
1st Paediatric Department, Fundeni Clinical Institute
🇷🇴Bucharest, Romania
Emergency County Hospital Constanta
🇷🇴Constanta, Romania
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, Spain
Sunpasitthiprasong Hospital
🇹🇭Ubon Ratchathani, Mueang Distirct,, Thailand
Ramathibodi Hospital_Bangkok
🇹🇭Bangkok, Thailand
Hematology and Oncology, Dept.of Pediatrics, CMU
🇹🇭Chiang Mai, Thailand