E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
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 | 10060612 |
E.1.2 | Term | Hemophilia A |
E.1.2 | System Organ Class | 100000004850 |
|
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 |
Primary objectives is to evaluate and compare the effect on frequency of joint bleeding events in severe and moderate pediatric hemophilia A patients using 3 different prophylactic regimens: once a week prophylactic treatment, twice a week or three times a week prophylactic treatment regimens. |
|
E.2.2 | Secondary objectives of the trial |
Secondary objectives are to assess the effect of prophylactic treatment on the number of all bleeds, the clinical status (Stockholm Hemophilia Joint Score), the number of patients in each treatment group at the end of study, total monthly consumption of rFVIII-FS for each patient and to evaluate the effect of prophylaxis with rFVIII-FS on quality of life in severe and moderate Hemophilia A patients using Haemo-QoL questionnaire. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Male - Severe hemophilia A or moderate hemophilia A - 1-12 years of age - Requiring treatment with FVIII |
|
E.4 | Principal exclusion criteria |
Current or prior inhibitor or familial antecedents of inhibitor - Surgery required during the study (9 months) - Positive for HIV |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Percentage of participants with less than 2 joint bleeds during the 9-month treatment period |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
• Number of Bleeds Per Participant During the 9-month Treatment Period
• Number of Participants With Bleeding Events During the 9-month Treatment Period
• Number of Participants With Joint Bleeds During the 9-month Treatment Period
• Number of Participants in Each Group at the End of the Study
• Actual Monthly rFVIII-FS Consumption
• Change From Baseline in Stockholm Hemophilia Joint Score at 9 Months of Treatment
• Haemo-QoL Standardized Total Score at 9 Months of Treatment (Completed by Participants in the Total Group)
• Haemo-QoL Standardized Total Score (Completed by Parents/Caregivers in the Total Group) at 9 Months of Treatment
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
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) | 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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
different prophylactic regimen: once a week, twice a week or three times a week |
|
E.8.2.4 | Number of treatment arms in the trial | 3 |
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 |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 3 |