E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Following EMEA guidelines NN7008-3545 trial is a prospective clinical phase 3a trial intended at demonstrating safety and efficacy of N8 in previously treated patients < 12 years of age with severe haemophilia A. The trial will continue until at least 50 patients have received at least 50 EDs to the product. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10053753 |
E.1.2 | Term | Hemophilia A without inhibitors |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate safety of N8 in paediatric previously treated patients (PTPs) <12 years of age with haemophilia A |
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E.2.2 | Secondary objectives of the trial |
To evaluate pharmacokinetics of N8 in paediatric PTPs <12 years of age with haemophilia A To evaluate efficacy of N8 in paediatric PTPs <12 years of age with haemophilia A To assess and compare Patient Reported Outcomes from baseline to end of trial in paediatric PTPs <12 years of age with haemophilia |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the patient.) 2. Male patients with severe (baseline FVIII ≤1%) haemophilia A 3. Age <12 years and weight ≥11 kg 4. Documented history of a minimum 50 EDs to FVIII produc prophylaxis/prevention/surgery/on-demand)(Please refer to section 8.4.4.2) 5. No factor VIII inhibitors ( ≥0.6 BU/mL) at screening 6. Documented negative FVIII inhibitor test(s) ( < 0.6 BU/mL ) or documented FVIII recovery tests (within expected normal ranges) within first 50 EDs 7. Immunocompetent, defined as either HIV negative or if HIV positive, CD4 >200 cells/μL according to medical records |
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E.4 | Principal exclusion criteria |
1. Known or suspected allergy to hamster protein or intolerance to trial product or related products 2. Previous participation in this trial defined as withdrawal after administration of trial product 3. Any history of FVIII inhibitors (≥0.6 BU/mL)(Please refer to section 8.4.4.1) 4. Surgery planned to occur during the trial (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures) 5. Platelet count <50,000 platelets/μL 6. Congenital or acquired coagulation disorders other than haemophilia A 7. Ongoing treatment or planned treatment during the trial with chemotherapy, immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids) 8. The receipt of any investigational product within 30 days prior to inclusion in this trial 9. Any disease or condition which, judged by the Investigator, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome 10. Renal dysfunction defined as serum creatinine level ≥ 2 times upper limit of normal or glomerular filtration rate decrease > 50% 11. Liver dysfunction defined as ALT or GGT ≥ 2 times upper limit of normal, or jaundice. 12. Documented diagnosis of obesity (only for patients in the PK part) defined as weight equal to or greater than the 95th percentile for age (refer to Appendix E) 13. Unwillingness, language or other barriers precluding adequate understanding and/or cooperation from parents and child |
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E.5 End points |
E.5.1 | Primary end point(s) |
The incidence rate of FVIII inhibitors (≥0.6 BU/mL) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
2 Coorti di et� pediatrica diversa: una coorte di 0-6 anni e una coorte di 6-11 anni |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
2 coorti di pazienti pediatrici di et� diversa |
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E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |