E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hereditary spastic paraplegias (HSP) are a rare neurodegenerative group of disorders characterised by slowly progressive symmetric spastic paraparesis, distal pallhypaesthesia of the lower limbs, and urinary dysfunction. Based on clinical hallmarks, noncomplicated HSP and complicated forms are known. Clinically, HSP forms are similar but genetically even more heterogeneous.
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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 |
To date, no causal therapy of the hereditary spastic paraplegias (HSP) is yet available. It is impossible to stop disease progression and to minimize the extent of clinical deficits. Therefore symptomatic therapy trails are necessary and important in the course of the patients´ treatment. Symptomatic approaches include physiotherapy and antispastic medication. However, side effects of antispastic drugs are often intolerable. In the planned clinical study, the effect of L-DOPA on the spasticity and mobility and, consecutively, on the patients´ daily activities and quality of life is to be investigated. In addition, own casuistic findings in HSP patients receiving L-DOPA for reasons such as RLS support these reports.
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E.2.2 | Secondary objectives of the trial |
- improvements in muscle strength (British Medical Research Council, BMRC) - occurrence of adverse effects using ”Fischer Somatische Symptome oder Unerwünschte Effekte Check List" (FSUCL). - blood pressure below 150 mmHg/90 mmHg - depression score (Beck´s Depression Inventory): occurence of depression - Hamilton Depression Scale (HAMD): occurence of depression - Quality of Life (SF-36)
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1.) patients (Females and males) aged between 18 to 80 years. 2.) definite noncomplicated spastic paraplegia 3.) ability to understanding the written patients information 4.) ability to cive a written consent 5.) normal electrocardiogramm 6.) females of childbearing age: use of pregnancy preventing arrangements
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E.4 | Principal exclusion criteria |
1.) participation in a clinical trial in the past 12 month using other medicinical pharmaceuticals 2.) contraindications for a therapy with Levodopa: pregnancy, lactation period 3.) Use of Levodopa or dopamine agonists in the past 3 months 4.) any diseases in which the release of functional testing could be impaired 5.) noncomplained patients 6) therapy with phenytoine, papaverine, inhibitors of monoaminooxidase A and B, drugs containing iron sulphit 7) coronary heart disease and cardiac arrhythmias 8) pregnancy, lactation period
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E.5 End points |
E.5.1 | Primary end point(s) |
objectives of the study:
a) primary objectives and end points: - improvements in HSP Rating Scale up to 10 points
b) secondary objectives: - muscle strength (British Medical Research Council, BMRC) - occurrence of adverse effects using ”Fischer Somatische Symptome oder Unerwünschte Effekte Check List" (FSUCL). - blood pressure below 150 mmHg/90 mmHg - depression score (Beck´s Depression Inventory) - Hamilton Depression Scale (HAMD) - Lebensqualität oder allgemeine Befindlichkeit (Fragebogen SF-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 | 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 | Information not present in EudraCT |
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 | Yes |
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.8.3 |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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The trail lasts in all patients 2 months. After then, all patients will be investigated in our outpatient clinic every two weeks for further 2 months. |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |