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 |
Bleeding disorder, inherited deficiency in clotting factor VIII |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018938 |
E.1.2 | Term | Haemophilia A (Factor VIII) |
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 assess safety of turoctocog alfa during treatment and prophylaxis of bleeding episodes in previously treated patients with moderate or severe Haemophilia A in India |
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E.2.2 | Secondary objectives of the trial |
To assess efficacy outcomes with turoctocog alfa during treatment and prophylaxis of bleeding episodes in previously treated patients with moderate or severe Haemophilia A in India |
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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 procedures that are carried out as part of the trial, including activities to determine suitability for the trial. 2. Male, age above or equal to 12 years at the time of signing informed consent. 3. Patients with the diagnosis of congenital moderate or severe Haemophilia A based on medical records. (FVIII ≤ 5%). 4. Documented history of at least 150 EDs to FVIII containing products. |
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E.4 | Principal exclusion criteria |
1. Confirmed inhibitors to FVIII (≥ 0.6 BU) at screening as assessed by central laboratory. 2. History of FVIII inhibitors. 3. Known or suspected hypersensitivity to trial product(s) or related products. 4. Previous participation in this trial. Participation is defined as signed informed consent. 5. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 1 month before screening (visit 1). 6. Any disorder, except for conditions associated with haemophilia A, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol. 7. Immunocompromised patients due to HIV infection (defined as viral load ≥400.000 copies/mL and/or CD4+ lymphocyte count ≤200/μL). HIV status and CD4+ lymphocyte count /viral load results may be obtained at screening or from available medical records; results must be not older than 6 months. 8. Known congenital or acquired coagulation disorders other than haemophilia A. 9. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Occurrence of confirmed FVIII inhibitor development (≥ 0.6 BU) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
During 8 weeks of treatment |
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E.5.2 | Secondary end point(s) |
1. Frequency of adverse drug reactions (AR) and serious adverse reactions (SAR) 2. Successful haemostatic effect of turoctocog alfa in the treatment of bleeding episodes 3. Total annualised consumption of turoctocog alfa 4. Frequency of allergic or infusion reactions related to the trial product |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Reported until follow-up, 12 weeks after first treatment 2. During 8 weeks of treatment 3. Measured during the 8 weeks of treatment 4. Reported until follow-up, 12 weeks after first treatment |
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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 | 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) | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |