E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Autistic disorder in children and adolescents |
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E.1.1.1 | Medical condition in easily understood language |
Autistic disorder, being irritable |
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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 | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10003808 |
E.1.2 | Term | Autistic disorder |
E.1.2 | System Organ Class | 100000004852 |
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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 |
The purpose of this study is to evaluate the efficacy of risperidone compared with placebo in children and adolescents with irritability associated with autistic disorder. |
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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 |
- Diagnostic for autistic disorder
- A Clinical Global Impression - Severity (CGI-S) score of =>4 and an Aberrant Behavior Checklist - Japanese Version (ABC-J) Irritability Subscale score of =>18
- Patients with mental age of >18 months as measured by appropriate developmental or mental scales
- Patients who have an appropriate caregiver, eg, parent or study-site personnel, who is able to observe the patient’s condition, provide information, and evaluate the patient’s response appropriately |
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E.4 | Principal exclusion criteria |
- Patients with previous or current psychotic disorder (eg, schizophrenia, bipolar disorder, or other psychiatric disorders) or with pervasive developmental disorder not otherwise specified, Asperger’s disorder, Rett’s disorder, pediatric destructive behavior disorder, or substance dependence
- Patients with a clinically significant endocrine, metabolic, cardiac, hepatic, renal, or pulmonary disorder, or hypertension
- Weight of <15 kg at the time of screening and baseline
- Patients with QTc>450 msec in the standard 12-lead electrocardiogram (ECG) at the time of screening
- Patients with known hypersensitivity to risperidone or paliperidone |
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E.5 End points |
E.5.1 | Primary end point(s) |
The change from baseline in the Aberrant Behavior Checklist−Japanese Version (ABC-J) Irritability Subscale scores |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- The changes from baseline in the subscale scores of Aberrant Behavior Checklist-Japanese Version (ABC-J) at each evaluation of the double-blind phase
- The changes from baseline in the subscale scores of Aberrant Behavior Checklist-Japanese Version (ABC-J) at each evaluation of the open-label phase
- The changes from baseline in scores of the Clinical Global Impression – Severity (CGI-S) at each evaluation time point of the double-blind phase
- The changes from baseline in scores of the Clinical Global Impression – Severity (CGI-S) at each evaluation time point of the open-label-phase
- The changes from baseline in scores of the Children's Global Assessment Scale (C-GAS) at each evaluation time point of the double-blind phase and open-label-phase
- The changes from baseline in scores of the Children's Global Assessment Scale (C-GAS) at each evaluation time point of open-label-phase
- The Clinical Global Impression−Change (CGI-C) at each evaluation time point of the double-blind phase
- The Clinical Global Impression−Change (CGI-C) at each evaluation time point of the open label-phase
- The Parent Satisfaction Questionnaire (PSQ) at each evaluation time point of the double-blind phase
- The Parent Satisfaction Questionnaire (PSQ) at each evaluation time point of the open label-phase |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Baseline, Week 2, Week 4, Week 6
- Baseline, Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48
- Baseline, Week 1, Week 2, Week 3, Week 4, Week 6, Week 8
- Baseline, Week 1, Week 2, Week 3, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48
- Baseline, Week 4, Week 8
- Baseline, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48
- Week 1, Week 2, Week 3, Week 4, Week 6, Week 8
- Week 1, Week 2, Week 3, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48
- Week 1, Week 2, Week 3, Week 4, Week 6, Week 8
- Week 1, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, Week 28, Week 32, Week 36, Week 40, Week 44, Week 48 |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | 2 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial months | 1 |