Clinical trial NCT02141074: Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Official title: An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2014-05-19
Enrollment: 54
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 nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.


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 at the time of signing informed consent
* Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity level below or equal to 2%) based on medical records or central laboratory results
* Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposures to blood components is acceptable)

Exclusion Criteria:

* Any history of FIX inhibitors (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 B
* 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)/LAR(s) (legally acceptable representative) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

Primary outcomes:
measure: Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)
timeFrame: When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (ED) (up to 156 weeks)
measure: Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)
timeFrame: When minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)
measure: Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)
timeFrame: At end of trial (up to 434 weeks)
Secondary outcomes:
measure: Number of Adverse Events
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Frequency of Adverse Events
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Number of Serious Adverse Events
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Frequency of Serious Adverse Events
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Number of Medical Events of Special Interest
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Frequency of Medical Events of Special Interest
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate)
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Incremental Recovery at 30 Minutes (IR30min)
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)
measure: FIX Activity at 30 Minutes (C30min)
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)
measure: FIX Trough Levels
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Amount of Drug Administered to Treat a Bleeding Episode
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
measure: Number of Injections Needed to Treat a Bleeding Episode
timeFrame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles - Endocrinology
state: California
city: Boise
country: United States
facility: St. Luke's Mountain States Tumor Institute
state: Idaho
city: New Orleans
country: United States
facility: Children's Hosp-New Orleans
state: Louisiana
city: Omaha
country: United States
facility: Univ of NE Med Center_Omaha
state: Nebraska
city: Charlotte
country: United States
facility: St.Jude Clin at Novant Hlth Hemby Cld Hosp
state: North Carolina
city: Cleveland
country: United States
facility: Univ Hosp Cleveland Med Ctr
state: Ohio
city: Dayton
country: United States
facility: Children's Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: St Christopher Hosp for Child
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Hemostasis Thrombosis Clinic
state: Tennessee
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: Caba
country: Argentina
facility: Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan
city: Cordoba
country: Argentina
facility: Sanatorio Mayo Privado S.A
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: Ordination Prof. Zwiauer
city: Wien
country: Austria
facility: Universitätsklinik für Kinder- und Jugendheilkunde
city: Hamilton
country: Canada
facility: Hamltn Hth Sci/McMstr Child Hosp
state: Ontario
city: Clermont Ferrand
country: France
facility: CHU Estaing
city: Kremlin-Bicêtre
country: France
facility: Hopital de Bicetre
city: Nantes Cedex 1
country: France
facility: Centre régional de traitement de l'Hémophilie
city: Tel-Hashomer
country: Israel
facility: Sheba MC The Israeli National Hemophilia Center
city: Saitama
country: Japan
facility: Saitama Children's Med Centre_Hematology-Oncology
city: Kuantan
country: Malaysia
facility: Hospital Tengku Ampuan Afzan
state: Pahang
city: Georgetown, Penang
country: Malaysia
facility: Hospital Pulau Pinang_Georgetown, Penang
city: Klang, Selangor
country: Malaysia
facility: Hospital Tengku Ampuan Rahimah
city: Kuala Lumpur
country: Malaysia
facility: National Blood Centre
city: Esplugues Llobregat
country: Spain
facility: Hospital Sant Joan de Déu
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Valencia
country: Spain
facility: Hospital La Fe - Endocrinología y Nutrición
city: Changhua
country: Taiwan
facility: Changhua Christian Hospital
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Chung-Ho Memorial Hospital_Kaohsiung
city: Taichung
country: Taiwan
facility: China Medical University Hospital - Children Building
city: Taipei
country: Taiwan
facility: NTU Hospital - Children and Women Hospital
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital_Bangkok
city: Chiang Mai
country: Thailand
facility: Hematology and Oncology, Dept.of Pediatrics, CMU
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary
city: London
country: United Kingdom
facility: St Thomas' Hospital
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.