E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Disease in which clotting factor VIII (FVIII), is deficient or inactive. Subjects with this condition have an increased risk to bleed compared to unaffected individuals. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018937 |
E.1.2 | Term | Haemophilia A |
E.1.2 | System Organ Class | 100000004850 |
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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 describe the safety and clinical efficacy of Xyntha in previously treated Chinese subjects with hemophilia A. |
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E.2.2 | Secondary objectives of the trial |
To describe FVIII recovery after exposure to Xyntha in previously treated Chinese subjects with hemophilia A.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia A (FVIII activity: more than 5 percentage (%), 1-5%, or less than 1%, respectively).
2. Subjects with previous exposure to FVIII replacement therapy.
3. If human immunodeficiency virus (HIV) positive, documented cluster of differentiation (CD4) count more than 200 per microliter (mcL) within 6 months of study entry.
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E.4 | Principal exclusion criteria |
1. Diagnosed with any bleeding disorder in addition to hemophilia A.
2. Current FVIII inhibitor or history of FVIII inhibitor (defined as positive result of the reporting laboratory).
3. Subject has no history of exposure to FVIII products (previously untreated patient [PUP]).
4. Subject is currently utilizing primary FVIII prophylaxis.
5. Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry.
6. Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation.
7. Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation.
8. Subjects with a known hypersensitivity to hamster protein.
9. Significant hepatic or renal impairment (alanine aminotransferase [ALT] and aspartate aminotransferase [AST] >5*upper limit of normal [ULN], bilirubin greater than (>) 2 microgram per deciliter (mg/dL) or serum creatinine >1.25 *ULN).
10. Prothrombin Time >1.5 * ULN.
11. Platelet count less than (<) 80,000/mcL.
12. Pregnant or breastfeeding women.
13. Unwilling or unable to follow the terms of the protocol.
14. Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation, in the opinion of the Investigator or Sponsor.
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E.5 End points |
E.5.1 | Primary end point(s) |
1) Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
2) Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
3) Number of Participants With Factor VIII (FVIII) Inhibitor Development
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1) 8 hours post infusion
2) 24 hours post infusion
3) Day 1 and Month 6 or Early Termination Visit |
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E.5.2 | Secondary end point(s) |
1) FVIII Recovery : Change From Baseline in FVIII Concentration
2) Number of Participants With Less Than Expected Therapeutic Effect (LETE)
3) Number of Participants With Thrombosis Allergic-Type Reactions
4) Number of Participants With Thrombosis |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) Day 1 and Month 6 or Early Termination Visit
2) 24 hours after each of 2 successive infusion, up to 6 months
3) Baseline up to 6 months
4) Baseline up to 6 months |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
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) | 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 | 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.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA 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.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 17 |