E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
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 determine the immunologic and overall safety associated with long-term use of Alphanate® in individuals diagnosed with severe hemophilia A (Factor VIII:C less than 0.01 IU/mL), who have been previously treated with plasma-derived Factor VIII products other than Alphanate® and who have no history of developing either antibody inhibitors to Factor VIII or nonspecific inhibitors of coagulation. |
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E.2.2 | Secondary objectives of the trial |
The frequency, nature, causality and severity of adverse events.2. The occurrence of changes in biochemical parameters which would indicate renal or hepatic impairment.3. The incidence of seroconversion to human immunodeficiency virus type 1 and 2 (HIV-1/HIV-2), hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV) or parvovirus B19 in subjects seronegative for these viruses at the time of enrollment. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.The subject must be male, at least six years of age and not more than 65 years of age. 2.The subject must have signed and dated an Informed Consent Form and Patient Authorization for Release of Information approved by the appropriate Institutional Review Board (IRB) prior to screening and enrollment. If the subject is a minor (i.e., less than 18 years of age) both he and his parent or legal guardian must sign and date the informed consent. 3.The subject must have been diagnosed with severe hemophilia A and must have levels of Factor VIII less than 0.01 IU/mL. 4.The subject must have received treatment with cryoprecipitate, Factor VIII concentrates, and/or whole blood, for at least 150 cumulative exposure days (CEDs) prior to enrollment. 5.The subject must not have received treatment with cryoprecipitate, Factor VIII concentrate, or any other blood product, for at least 72 hours prior to screening. 6.The subject must never have been diagnosed with inhibitors to Factor VIII at any detectable titer. 7.The subject must never have been diagnosed with nonspecific inhibitors of coagulation. 8.The subject must test negative for the presence of Factor VIII inhibitors at screening and enrollment. 9.The subject must have CD4 counts greater than or equal to 400 cells/ µL. 10.The subject must have been vaccinated against hepatitis A and hepatitis B, or have evidence of antibodies against hepatitis A and hepatitis B. However, a subject who has no prior immunity against hepatitis A will be offered a course of vaccination for hepatitis A. 11.The subject must have a Kamofsky Perfomrance Score of at least 50 (Appendix 1).
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E.4 | Principal exclusion criteria |
1.The subject is receiving any immunosuppressive medications including intravenous immunoglobulins at the time of enrollment. 2.The subject has clinical signs or symptoms of an infection, such as fever, chills or nausea during screening or enrollment. 3.The subject has a history of frequent reactions to Factor VIII concentrates (e.g., chills or headaches). 4.The subject has received treatment with Alphanate® (Solvent-Detergent/ Heat-Treated). 5.The subject is immunocompromised (including HIV+ status or has an impaired immune system due to disease or treatment).
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E.5 End points |
E.5.1 | Primary end point(s) |
the incidence of Factor VIII inhibitor after exposure to Alphanate®. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Information not present in EudraCT |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
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) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | 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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 3 |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |