Phase 3
N=72
Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors
Congenital Bleeding Disorder · Haemophilia A With Inhibitors · Haemophilia B With Inhibitors
Bottom Line
View on ClinicalTrials.gov: NCT01392547 ↗Enrolled (actual)
72
Serious AEs
6.2%
Results posted
Dec 2013
Primary outcome: Primary: Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given — 22; 16; 318; 211 bleeding episodes
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- vatreptacog alfa (activated) (Drug); eptacog alfa (activated) (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Aug 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given |
22; 16; 318; 211 | — |
| SECONDARY Effective and Sustained Bleeding Control |
53; 51; 268; 163 | — |
| SECONDARY Number of Doses of Trial Product Given for Each Acute Bleed |
51; 23; 94; 62; 195; 142 | — |
| SECONDARY Number of Adverse Events |
55; 11; 33; 5; 15; 2 | — |
| SECONDARY Immunogenicity (Inhibitor Development) |
8; 0; 4; 0; 0; 1 | — |
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.
Eligibility Criteria
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)
Data sourced from ClinicalTrials.gov (NCT01392547). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.