Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors

Phase 3

Completed

• Conditions: Congenital Bleeding Disorder; Haemophilia A With Inhibitors; Haemophilia B With Inhibitors
• Interventions: Drug: eptacog alfa (activated); Drug: vatreptacog alfa (activated)

• Registration Number: NCT01392547
• Lead Sponsor: Novo Nordisk A/S

Brief Summary

This trial is conducted globally. The purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors.

Detailed Description

Scheduled dose visit in a non-bleeding state. Single dose of NNC 0078-0000-0007 (vatreptocog alfa (activated)) every 3 months.

Study Timeline

• Study Start: 2011-07
• Study First Submitted: 2011-07-08
• Study First Submitted QC: 2011-07-11
• Study First Posted: 2011-07-12
• Primary Completion: 2012-08
• Study Completion: 2012-08
• Disposition First Submitted: 2012-09-20
• Disposition First Submitted QC: 2012-10-17
• Disposition First Posted: 2012-10-18
• Results First Submitted: 2013-09-27
• Results First Submitted QC: 2013-10-08
• Results First Posted: 2013-12-06
• Status Verified: 2015-02
• Last Update Submitted: 2017-03-29
• Last Update Posted: 2017-05-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 72

Inclusion Criteria

• Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX
• Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry

Exclusion Criteria

• Previous participation in this trial defined as withdrawal after administration of trial product
• Patient has received an investigational medicinal product within 30 days prior to this trial
• Congenital or acquired coagulation disorders other than haemophilia A or B
• Any clinical signs or known history of arterial thrombotic events or of deep venous thrombosis or pulmonary embolism (as defined by available medical records)
• Platelet count of less than 50,000 platelets/mcL (at the screening visit)
• ALAT (alanine-transaminase) of more than 3 times the upper normal limit (according to laboratory reference ranges)
• Factor VIII/IX Immune Tolerance Induction regimen planned to occur during the trial
• Ongoing bleeding prophylaxis regimens or planned bleeding prophylaxis to occur during the trial
• HIV (Human Immunodeficiency Virus) positive with current CD4+ count of less than 200/mcL (defined by medical records)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
rFVIIa	eptacog alfa (activated)	-
vatreptocog alfa	vatreptacog alfa (activated)	-

Primary Outcome Measures

Name	Time	Method
Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given	Within 12 hours of first trial product administration

Secondary Outcome Measures

Name	Time	Method
Number of Adverse Events	Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product.	Any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Effective and Sustained Bleeding Control	Up to 48 hours after first trial product administration
Number of Doses of Trial Product Given for Each Acute Bleed	Up to 6 hours after first trial product administration
Immunogenicity (Inhibitor Development)	Adverse events were captured from the time of consent to the end of trial visit 1 month (+14 days) after last administration of trial product.	Immunogenicity was tested by formation of neutralising antibodies towards vatreptacog alfa and/or FVII. Radioimmunoassay using \[125I\]-labelled vatreptacog alfa or rFVIIa was used to screen plasma samples for development of anti-drug antibodies

Trial Locations

Locations (1)

Novo Nordisk Clinical Trial Call Center; 🇵🇷 San Juan, Puerto Rico