E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Phenylketonuria, ADHD |
Fenylketonurie, ADHD |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
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 |
The primary objective is to determine the effectiveness of methylphenidate in reduction of ADHD symptomatology, operationalized by personalized goals that are important to the patient and its environment, in individuals with late-diagnosed PKU. |
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E.2.2 | Secondary objectives of the trial |
To determine the effect of methylphenidate on emotion dysregulation. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Minimum age of 18 years. - A definite diagnosis of classical PKU according to well-established guidelines. - Meet DSM-5 criteria for ADHD and diagnosed with ADHD by an expert multidisciplinary team consisting of an ID physician, a psychologist, and a psychiatrist. - Presence of a patient’s caregiver for proxy-reports. |
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E.4 | Principal exclusion criteria |
- Unable to take and/or send in dried blood spots. - Presence of ADHD in first- and second-degree relatives. - Presence of a contra-indication for treatment with methylphenidate (e.g. cardiovascular disease). - Planned surgery and/or general anaesthesia during the trial. - Pregnancy. - Breastfeeding (females). - During treatment with non-selective, irreversible monoamine oxidase (MAO) inhibitors, or within a minimum of one month of discontinuing those drugs. - Current substance or alcohol abuse. - Unable to swallow tablets / capsules. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome measure is GAS to identify and measure individualized goals with regard to the most obstructing symptoms experienced by caretakers. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
End of (interventional) periods |
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E.5.2 | Secondary end point(s) |
Secondary outcome measures are the hyperactivity / inattention subscale of the Strengths and Difficulties Questionnaire (SDQ), the Dutch version of the Emotion Dysregulation Inventory (EDI) reactivity index, and the PQ as a symptom list to compare assessments of personalized goals with those measured by GAS. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
End of (interventional) periods. |
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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 | No |
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 | 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 | Yes |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
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 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |