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

Novo Nordisk A/S72 enrolled

Overview

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.

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Interventions

vatreptacog alfa (activated)DRUG

1-3 doses per bleeding episode

eptacog alfa (activated)DRUG

1-3 doses per bleeding episode

Eligibility

Sex: MALEMin age: 12 Years

Medical Language ↔ Plain English

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)

Locations (32)

Outcomes

Primary Outcomes

Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given

Time frame: Within 12 hours of first trial product administration

Secondary Outcomes

Effective and Sustained Bleeding Control

Time frame: Up to 48 hours after first trial product administration

Number of Doses of Trial Product Given for Each Acute Bleed

Time frame: Up to 6 hours after first trial product administration

Number of Adverse Events

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.

Time frame: Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product.

Immunogenicity (Inhibitor Development)

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

Time frame: 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.

Data from ClinicalTrials.gov

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