E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients diagnosed with infantile spasms |
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E.1.1.1 | Medical condition in easily understood language |
Patients diagnosed with infantile spasms |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10021750 |
E.1.2 | Term | Infantile spasms |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 investigate the efficacy of orally-administered vigabatrin in patients with infantile spasms, using changes in spasm frequency as an endpoint. Also to investigate safety and pharmacokinetics. |
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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 |
Patients (aged >=4 weeks, <2 years) diagnosed with infantile spasms manifested by spasms and hypsarrhythmia on electroencephalography. Using classification of ILAE 1989 |
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E.4 | Principal exclusion criteria |
- Patients with concurrent severe liver/renal disease, cardiac disease, serious gastrointestinal disorder.
- Patients who received adrenocorticotropic hormone (ACTH) products or steroids within 4 weeks (28 days prior to the formal registration of this study.
- Patients previously or currently treated with vigabatrin.
- Patients who have concurrent or a history of ophthalmological complication in whom possible aggravation of the symptoms with vigabatrin.
- Patients with concomitant Lennox-Gastaut syndrome. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Changes in frequency of spasms before and after administration of the investigational drug (percentage of subjects with reduction in frequency of spasms of at least 50% from baseline on the primary spasms assessment date |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
2 days prior to the maintenance administration start date |
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E.5.2 | Secondary end point(s) |
- Changes in frequency of spasms
- Disappearance of spasms
- Complete disappearance of infantile spasms and hypsarrhythmia
- Brainwave findings
- Comprehensive evaluation of efficacy by the Investigators including guardians' opinion. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
2 days prior to the maintenance administration start date |
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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 | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Measurement of plasma taurine concentration |
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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 | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
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 | Yes |
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 |