E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hereditary Spastic Paraplegia |
Hereditaire Spastische Paraplegie |
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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] - Nervous System Diseases [C10] |
MedDRA Classification |
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 primary objectives of this study is to investigate whether BTX injections, injected bilaterally in the spastic adductors of patients with HSP can improve lateral stepping and gait width. |
De primaire doelstelling van deze studie is te onderzoeken of BTX injecties, bilateraal geïnjecteerd in de spastische adductoren van HSP patiënten, lateraal stappen en de loopbreedte kan verbeteren. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are balance confidence, Berg Balance Scale, comfortable and maximum overground gait velocity, 6-Min Walk Test, Timed Up and Go test and gait adaptability. |
The secundaire doelstellingen zijn de balans tevredenheid, Berg Balans Schaal, comfortabele en maximale loopsnelheid, 6-Min Loop Test, Time Up and Go test en aanpassingsvermogen tijdens lopen. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-diagnosis of pure HSP based on molecular diagnosis (e.g. SPG-4 mutations) or based on inheritance
-age at least 18 years
-suffer from (bilateral) hip adductor spasticity without contracture (modified Ashworth scale score 1-4)
-being a minimally independent household ambulator without walking aid (Functional Ambulation Categories 4-5)
-gait velocity > 0.4 m/s
-complain of balance problems due to a narrow base of support or problems with lateral stepping
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-diagnose van pure HSP gebaseerd op moleculaire diagnose (bijv. SPG-4) of op basis van erfelijkheid
-minimaal 18 jaar
-Lijden aan (bilaterale) heup adductor spasticiteit zonder contractuur (modified Ashworth scale score 1-4)
-minimaal onafhankelijke lopers in bekende omgeving zonder hulpmiddelen (Functional Ambulation Categories 4-5)
-Loopsnelheid > 0.4 m/s
-Klachten over balansproblemen door te kleine standbreedte of problemen met zijwaarts stappen |
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E.4 | Principal exclusion criteria |
-Last BTX treatment of the hip adductors is shorter than 6 months ago or when a next BTX treatment of these muscles cannot be postponed for 4 months without increasing fall risk or jeopardizing patients’ safety in any other way.
-Any previous BTX injections in the calf muscles are accepted, but it must be possible to safely postpone subsequent injections of these muscles for 4 months after inclusion
-Insufficient vision
-Insufficient cardiopulmonary endurance
-Insufficient sensorimotor abilities
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-Laatste BTX behandeling van de heup adductoren korter dan 6 maanden geleden of wanneer een BTX behandeling van deze spieren niet uitgesteld kan worden naar 4 maanden na inclusie zonder het risico van vallen te vergroten of de patiënt op enige andere wijze in gevaar te brengen
-Voorgaande BTX injecties in kuitspieren worden geaccepteerd, maar het moet mogelijk zijn deze veilig uit te stellen naar 4 maanden na inclusie.
-Onvoldoende visie
-Onvoldoende cardiopulmonaal uithoudingsvermogen
-Onvoldoende sensorimotorische capaciteiten |
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E.5 End points |
E.5.1 | Primary end point(s) |
Increased succesratio of recovering balance by a lateral step. Increased frontal leg angle. Increased gait width |
Verhoogde succes-ratio in balanshandhaving door een zijwaartse stap. Verhoogde frontale beenhoek. Verbeterde loopbreedte |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
before, 6 weeks and 16 weeks after the injection |
voor, 6 en 16 weken na de injectie |
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E.5.2 | Secondary end point(s) |
Increased balance confidence, gait velocity and gait adaptability |
Verhoogde balans tevredenheid, loopsnelheid en aanpassingsvermogen tijdens het lopen. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
before, 6 weeks and 16 weeks after the injection |
voor, 6 en 16 weken na de injectie |
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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 | 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) | 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 |
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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |