E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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) |
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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 |
Describe the histamine release patterns produced by activated basophils obtained from peripheral blood of subjects who meet the protocol entry criteria, using the methodology described in the protocol. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Study Group: 1. Written informed consent or assent, as applicable. 2. Subjects with moderate to severe hemophilia B (FIX: C <5%), who have reported class II or class III allergic manifestations within 24 hours of a BeneFIX infusion.
Control Group (subjects with no known history of class ll or class lll allergic reactions to any FIX product): 1. Written informed consent or assent, as applicable. 2. Subjects with moderate to severe hemophilia B (FIX: C <5%). |
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E.4 | Principal exclusion criteria |
Study Group: 1. Subjects who had NO reaction when rechallenged with BeneFIX, in the absence of prophylactic immunomodulating therapy. 2. Subjects whose most recent allergic manifestations with BeneFIX have occurred > 36 months prior to providing written consent for this study. 3. Subjects who have taken antihistamine, steroidal, or other immunosuppressive therapy within 72 hours of the scheduled visit for this study. 4. Subjects with immune disorders (e.g. HIV, myeloma, lymphoma). 5. Subjects unable to comply with a minimum 5-day FIX washout requirement.
Control Group (subjects with no known history of class ll or class lll allergic reactions to any FIX product): 1. Subjects with documented evidence of prior class ll or class lll allergic reaction to any FIX product. 2. Subjects with documented evidence of prior or current FIX inhibitor 3. (BU ≥ 0.6) 4. Subjects who have taken antihistamine, steroidal, or other immunosuppressive therapy within 72 hours of the scheduled study blood draw. 5. Subjects with immune disorder (e.g. HIV, myeloma, lymphoma). 6. Subjects unable to comply with a minimum 5-day FIX washout requirement. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Descriptive presentation of the results of the Basophil histamine release assay for both study and control groups. |
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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 | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
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) | 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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Control group of haemophilia B patients |
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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 |
Control group of haemophilia B patients |
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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.8.6 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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Last patient last visit.
This study will remain open for 36 months in order to recruit 18 or more subjects (9 or more subjects with allergic reactions to BeneFIX, 9 or more control subjects). |
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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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |