E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
HEREDITARY HAEMOPHILIA B
One single pharmacokinetic to compare the pharmacokinetic profile of BeneFIX with that of AlphaNine used by the same patients in a previous trial. |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018939 |
E.1.2 | Term | Haemophilia B (Factor IX) |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
• To compare AlphaNine® with BeneFIX® in terms of theirtioned pharmacokinetic profile.
Pharmacokinetic parameters of FIX: in vivo recovery, half-life, area under the curve (AUC), mean residence time (MRT) and clearance, in at least 15 patients. |
|
E.2.2 | Secondary objectives of the trial |
Safety Immunogenicity Clinical safety (thrombogenicity and tolerance to infusion) Viral safety (HIV, HCV).
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• patients having participated in the previous study “Efficacy and safety of factor IX (FIX) contained in ALPHANINE® in patients with severe hereditary haemophilia B” |
|
E.4 | Principal exclusion criteria |
Patients will be deemed ineligible if they:
• have received a dose of FIX in the 7 days previous to the infusion • have a FIX inhibitor level of >0.5 Bethesda units or clinically relevant presence in the past (>5 BU), • have active bleeding at that moment of infusion, • CD4 lymphocyte count < 400/l, • have known allergic reaction to any Benefix® component, • exhibits symptoms of any intercurrent infection (i.e. fever, chills, nausea, etc.) at the time of the first infusion, • have any disease that might affect the distribution or metabolism of FIX and which could affect interpretation of the study (such as non-controlled diabetes mellitus), • have non-controlled arterial hypertension, • have abnormal renal function (creatinine >1.5 mg/dl), • have documented liver cirrhosis or any hepatic disorder with ALT levels 2.5 times or more than the normal upper limit, • are simultaneously participating in other clinical studies, • prevision to be concomitantly treated with other FIX-containing products, • have conditions that might affect patient compliance (survival-limiting [in 2 year time] diseases, alcohol or other drug abuse, etc.), • any patient unable to provide a storage plasma sample before the first dose of Benefix®.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy variables: FIX:C activity before and at different times after infusion of FIX concentrate. Consumption of FIX (IU/kg) Achievement of haemostasis Requirements of other blood derivative products Loss of blood in surgery
Safety Variables: FIX inhibitor status Thrombogenicity markers: prothrombin fragments 1+2 (F1+2), thrombin-antithrombin complex (TAT) and d-dimer Changes in vital signs and occurrence of adverse events Seroconversions (HIV, HCV)
|
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
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 | No |
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) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
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 | No |
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 | 3 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | |