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 |
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E.1.1.2 | Therapeutic area | Psychiatry and Psychology [F] - Mental Disorders [F03] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10017324 |
E.1.2 | Term | Fragile X syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 evaluate the safety and tolerability of AFQ056 in adult patients with FXS as assessed by:
• Incidence and severity of adverse events and serious adverse events
• Changes in vital signs, laboratory assessments, and ECGs
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E.2.2 | Secondary objectives of the trial |
To evaluate the efficacy of AFQ056 treatment in both Fully Methylated and Partially Methylated patients with FXS as assessed by:
• Change from baseline in the Aberrant Behavior Checklist – Community edition (ABC-C) total score and subscale scores
• Rating of global improvement of symptoms in Fragile X patients using the Clinical Global Impression - Improvement (CGI-I) scale.
• Change from baseline in the Repetitive Behavior Symptom – Research version (RBS) total score and subscale scores
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Must have been enrolled in Studies CAFQ056A2204 or CAFQ056A2212
- Has a caregiver who spends, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.
Other protocol-defined inclusion criteria may apply |
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E.4 | Principal exclusion criteria |
- Any advanced, severe or unstable disease
- History of severe self-injurious behavior
- History of uncontrolled seizure disorder or resistant to therapy within the past 2 years (Patients who are clinically stable under anti-convulsant therapy for the past 2 years are not excluded)
- History of clinically significant allergies requiring hospitalization or non-inhaled corticosteroid therapy (asthma, anaphylaxis, etc.)
- Any treatment regimen, including psychotropic and/or anticonvulsant therapy that has not been stable for ≥ 6 weeks prior to randomization
- Current treatment with more than two psychoactive medications, excluding medication used specifically for seizure control
- Using (or used within 6 weeks before randomization) concomitant medications that are potent inhibitors or inducers of CYP3A4
- Using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of randomization
- Planning to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study
Other protocol-defined exclusion criteria may apply |
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E.5 End points |
E.5.1 | Primary end point(s) |
Evaluation of the safety and tolerability of AFQ056 in adult patients with FXS as assessed by:
Incidence and severity of adverse events and serious adverse events and change in vital signs, laboratory assessments, and ECGs. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At multiple visits:
weekly during first month and monthly afterwards
Time Frame: 9 months |
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E.5.2 | Secondary end point(s) |
- Aberrant Behavour Checklist - Community Edition (ABC-C): Total score
and subscales
- Clinical Global Impression - Improvement (CGI-I) scale
- Repetitive Behaviour Symptom-Research version (RBS-R) - total score and subscale scores |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- ABC-C: Week 4, week 12, week 24, week 36
- CGI - I: Week 4, week 12, week 24, 36 week
- RBS: Week 4, week 36
Time frame: 9 months for each |
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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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 10 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
Denmark |
France |
Germany |
Italy |
Switzerland |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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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last visit of last subject |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 14 |