E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Spasticity of the upper and lower limb of the same body side due to cerebral causes |
Espasticidad de las extremidades superior e inferior del mismo lado del cuerpo debida a causas cerebrales |
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E.1.1.1 | Medical condition in easily understood language |
Increased muscle tension/uncontrollable muscle stiffness in the leg and arm of the same body side due to cerebral causes. |
Incremento en la tensión muscular/rigidez muscular incontrolable en la pierna y el brazo del mismo lado del cuerpo debido a causas cerebrales |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10058977 |
E.1.2 | Term | Spastic paresis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 objective of this clinical study is to assess the safety of doses of NT 201 of up to 800 U in subjects suffering from spasticity in both upper and lower limbs of the same side of the body due to cerebral causes over a period of up to 48 weeks with flexible injection intervals of 12 to 16 weeks. |
El objetivo principal de este estudio abierto de un solo grupo consiste en investigar la seguridad de NT 201 en el tratamiento de la espasticidad debida a causas cerebrales durante tres ciclos de inyección en pacientes que precisan dosis corporales totales de hasta 800 U de NT 201 por ciclo de inyección en las extremidades inferior y superior del mismo lado del cuerpo siguiendo una estrategia de ajuste de la dosis con un intervalo flexible de entre 12 y 16 semanas entre sesiones de inyección. |
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E.2.2 | Secondary objectives of the trial |
As a secondary objective, this study will evaluate the impact of the administration of NT 201 on several efficacy variables. |
El objetivo secundario de este estudio consiste en investigar la eficacia de NT 201 en el tratamiento con NT 201 mediante diferentes parámetros de resultados. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Age from 18-80 yrs - Upper and lower limb spasticity of the same body side due to cerebral causes - Time since event leading to spasticity in the target body side greater than 12 weeks - Need for 800 units Botulinum toxin type A |
- Edad de 18-80 años - Espasticidad de extremidades superiores e inferiores del mismo lado del cuerpo debido a causas cerebrales - Período mayor a 12 semanas desde que el último episodio que dio lugar a la espasticidad en el lado del cuerpo objetivo - Precisa una dosis corporal total de 800 U de NT 201 |
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E.4 | Principal exclusion criteria |
- Body weight below 50kg - Fixed contractures of the target joint - Generalized disorders of muscle activity like Myasthenia gravis that preclude use of Botulinum toxin type A - Infection at the injection site |
- Peso mayor menor de 50Kg - Contractura fija en la articulación objetivo - Trastornos generalizados de la actividad muscular como Miastenia grave que impide el uso de toxina botulínica tipo A - Infección en el lugar de inyección |
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E.5 End points |
E.5.1 | Primary end point(s) |
Occurence of treatment emergent adverse events (AEs), AEs of special interest (AESIs), and Serious AEs (SAEs) by injection cycle
Occurence of treatment-emergent AEs, AESIs and SAEs related to the administration of study medication by injection cycle
Investigator's global assessment of tolerability using a 4-point Likert scale at end of injection cycle visits |
? Aparición de AA, AAEI y AAG de aparición durante el tratamiento según el ciclo de inyección. ? Aparición de AA, AAEI y AAG de aparición durante el tratamiento relacionados con la administración de la medicación del estudio según el ciclo de inyección. ? Evaluación global de la tolerabilidad por parte del investigador en las visitas de final de los ciclos de inyección |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
week 12-16, week 24-32, week 36-48 |
semana 12-16, semana 24-32, semana 36-48 |
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E.5.2 | Secondary end point(s) |
Ashworth Scale (AS) of the target joint selected at study baseline visit
AS of every joint affected by clinical patterns of spasticity and treated at least once, of the same body side as the selected target joint
Resistance to passive movement scale (REPAS)
Functional ambulation classification (FAC) scale
Global attainment scale (GAS) for upper and lower limb, respectively
Disability Assessment Scale (DAS) in a selected principal therapeutic target domain affecting the upper limb
EuroQoL 5-dimensional questionaire (EQ-5D)
Global assessment of efficacy |
? AS de la articulación objetivo seleccionada en la visita basal del estudio (V2). ? AS de cada articulación que se encuentre afectada por los patrones clínicos de espasticidad y sea tratada al menos una vez, del mismo lado del cuerpo que la articulación objetivo seleccionada. ? Escala de Resistencia al movimiento pasivo (REPAS). ? Escala de clasificación funcional de la deambulación (FAC). ? Escala de consecución de objetivos (GAS) en las extremidades superior e inferior, respectivamente. ? Escala de evaluación de la discapacidad (DAS) en un dominio objetivo terapéutico principal seleccionado que afecte a la extremidad superior. ? Evaluación global de la eficacia ? Cuestionario EuroQoL de 5 dimensiones (EQ-5D). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
from baseline up to week 48 for: AS, REPAS, FAC, DAS,EQ-5D. Up to week 48 for: GAS and Global assessment of efficacy |
desde visita basal hasta semana 48 para AS, REPAS, FAC, DAS,EQ-5D. Hasta semana 48 para: GAS y Evaluación global de eficacia |
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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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | No |
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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 26 |
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 |
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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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The end of study will be defined as the last visit of the 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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |