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

Phase 3

Completed

Conditions: Congenital Bleeding Disorder
Haemophilia A

Interventions: Drug: turoctocog alfa pegol

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

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

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
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Phoenix, Arizona, United States
Rush University Med. Cntr
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Chicago, Illinois, United States
University Of Iowa
🇺🇸
Iowa City, Iowa, United States
North Shore Long Island Jewish Medical Center
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New Hyde Park, New York, United States
Torrence Hemby Ped Hem/Onc Ctr
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Charlotte, North Carolina, United States
Dayton's Children's Hemostasis and Thrombosis Center
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Dayton, Ohio, United States
Univ Oklahoma Sci Ctr OK City
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Oklahoma City, Oklahoma, United States
Univ of Utah Primary Children's Hospital
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Salt Lake City, Utah, United States
Beni Messous Hospital Issaad Hassani
🇩🇿
Algiers, Algeria
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Children's Hospital Los Angeles - Endocrinology
🇺🇸Los Angeles, California, United States