E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of advanced levodopa-responsive Parkinson’s disease with severe motor fluctuations and hyper-/dyskinesia when available combinations of Parkinson medicinal products have not given satisfactory results. |
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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 collect health economic data depicting the initial levels and natural progression over time of resource usage, Parkinson’s disease (PD)-related costs, and health related quality of life (HRQoL), utilizing both the Unified Parkinson’s Disease Rating Scale (UPDRS) and the Euro QoL–5 Dimensions quality of life instrument (EQ-5D), for a cohort of advanced PD patients treated with Duodopa, of which about one-third will be Duodopa-naïve prior to the start of the study. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are: •to characterize the stage of PD according to the Hoehn and Yahr Scale •to rate the subject’s best “on” period according to the Schwab and England Scale •to recognize cognitive impairment and assess cognitive changes according to the Mini Mental Status Examination (MMSE) •to measure changes in depressive illness and response to therapy according to the Montgomery-Åsberg Depression Rating Scale (MADRS) •to study health related quality of life as measured by the PD specific quality of life instrument (PDQ-39)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients on permanent treatment with Duodopa for at least 12 weeks prior to the study. or Duodopa treatment naïve patients. In this case the following must be fulfilled: The Investigator considers changing conventional PD treatment. The criteria in the Summary of Product Characteristics for Duodopa must be fulfilled.
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E.4 | Principal exclusion criteria |
1. Patients suffering from other diseases that, in the opinion of the Investigator, might interfere with the study objectives.
2. Patients that, in the opinion of the Investigator, are unable to comply with study requirements. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall PD-related resource consumption will be collected at baseline (month -3), at month 0, and monthly thereafter until study completion for Duodopa-naïve patients. Overall PD-related resource consumption will be collected at month 0 and monthly thereafter until study completion for Duodopa-experienced patients.
The consumption of health care resources will be "costed" using appropriate national product- or service-specific unit costs taken from the relevant sources (including price lists and fee schedules).
The UPDRS will be used to measure disease progression, while the EQ-5D will be used to generate QoL utilities. For Duodopa-naïve patients, they will be assessed at baseline (month -3), at month 0, and thereafter quarterly for the first year and biannually from month 12 to month 36. For Duodopa-experienced patients, they will be assessed first at month 0 and thereafter quarterly for the first year and biannually from month 12 to month 36.
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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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
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.8.3 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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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If the patient is on treatment with Duodopa at inclusion the last visit for the last patient is 3 years after inclusion.
If the patient is Duodopa treatment naive at inclusion the last visit for the last patient is 3 years + at least 3 months after start of permanent treatment with Duodopa.
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |