E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Spinal Cord Injury and Growth Hormone Deficiency |
Lesión medular espinal y deficiencia de la hormona de crecimiento |
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E.1.1.1 | Medical condition in easily understood language |
Spinal Cord Injury and Growth Hormone Deficiency |
Lesión medular espinal y deficiencia de la hormona de crecimiento |
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E.1.1.2 | Therapeutic area | Body processes [G] - Bones and nerves physological processes [G11] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10041558 |
E.1.2 | Term | Spinal cord injury thoracic |
E.1.2 | System Organ Class | 10022117 - Injury, poisoning and procedural complications |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10041554 |
E.1.2 | Term | Spinal cord injury cervical |
E.1.2 | System Organ Class | 10022117 - Injury, poisoning and procedural complications |
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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 |
Evaluate the efficacy and safety of Nutropin® in combination with intensive rehabilitation compared to placebo with intensive rehabilitation in patients with Spinal Cord Injury |
Evaluar la eficacia y seguridad de Nutropin® asociado a rehabilitación intensiva comparado con placebo más rehabilitación intensiva en pacientes con lesión medular |
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E.2.2 | Secondary objectives of the trial |
- to assess QoL and spasticity changes - to assess neurological motor and sensibility changes - to assess safety |
- evaluar los cambios en la QoL y en la espasticidad - evaluar los cambios neurológicos motores y los cambios en la sensibilidad - evaluar la seguridad |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Men or women aged 18 to 50 years (inclusive) -Cervical or thoracic (dorsal) spine injuries (C5-T12) -Complete lesions (ASIA A) with SSEP and EMG evaluation -Stability of the recovery (at least 12 months since injury) -Maintenance of muscular structure (no more than 24 months since injury) -Partial (<10 ng/ml) or total (<5 ng/ml) GH deficiency (assessed by GH stimulation tests) -Signed informed consent |
-Hombres o mujeres entre 18 y 50 años de edad (inclusive) -Lesiones (C5-T12) cervicales o torácicas (dorsales) -Lesiones completas (ASIA A) con evaluaciones de SSEP y EMG -Estabilidad de la recuperación (al menos 12 meses desde la lesión) -Mantenimiento de la estructura muscular (no mayor a 24 meses desde la lesión) -Deficiencia parcial (<10 ng/ml) o total (<5 ng/ml) de la GH (hormona de crecimiento) (evaluado a través de pruebas de estimulación de la GH) - Firma del consentimiento informado |
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E.4 | Principal exclusion criteria |
-Spinal cord injury of non-traumatic origin -Adverse effects or hypersensitivity to GH or its excipients -Lack of neurophysiological assessment/confirmation of SCI -Previous very intensive rehabilitation -Previous growth hormone therapy -Evidence of any active malignancy or cancer in the last 5 years -Acute illnesses at time of randomization - Pregnancy -Participation in another interventional study -Inability to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study |
-Lesión de la médula espinal de origen no traumático -Efectos adversos o hipersensibilidad conocida a la GH o sus excipientes - Falta de evaluación neurofisiológica/confirmación de SCI -Rehabilitación intensiva previa (la rehabilitación convencional no se considerará criterio de exclusión) -Terapia previa con hormona de crecimiento -Evidencia de neoplasia activa o cáncer en los últimos 5 años -Enfermedad grave al momento de la aleatorización -Embarazo -Participación en otro estudio no intervencional -Incapacidad para cumplir con los requerimientos del estudio o sujetos que, en la opinión del investigador, no deberían participar en el estudio |
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E.5 End points |
E.5.1 | Primary end point(s) |
Changes in Spinal Cord Independence Measure (SCIM) III score |
Cambios en el resultado de la escala Spinal Cord Independence Measure (SCIM) III |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
SCIM III will be assesed 4 times in 6 months: at visit 1 (day1), visit 2(month 1), visit 3(month 3) and visit 4 (month 6) |
SCIM III se evaluará 4 veces en 6 meses: en la visita 1 (día 1), visita 2 (mes 1), visita 3 (mes 3) y visita 4 (mes 6) |
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E.5.2 | Secondary end point(s) |
- Changes in quality of life: Nottingham Health Profile (NHP) score - Neurological motor changes: percentage of patients with selective muscle activity >1 spinal metamere below lesion. - Neurological profound and superficial sensory changes: percentage of patients with > 1 spinal metamere below lesion: Metamere neurological activity mapping (ASIA) at physical examination (Neurophysiology) confirmed by: a.Electromyogram with measurement of individualized motor units to measure efferent motoneurons activity (A? and C fibers). b.Somatosensory evoked potencials (SSEP) to measure direct posterior A? and A? fibers activity (Goll and Burdach posterior Fasciculae). -Changes in spasticity assessment: Modified Ashworth Score (MAS) (Rehabilitation) |
-Cambios en cuanto a la calidad de vida: resultados según el Perfil de Salud de Nottingham (NHP, Nottingham Health Profile) -Cambios motores neurológicos: porcentaje de pacientes con actividad muscular selectiva >1 metámera por debajo de la lesión medular -Cambios neurológicos en la sensibilidad superficial y profunda: porcentaje de pacientes con cambios en la sensibilidad superficial y profunda > 1 metámera por debajo de la lesión: mapeo de la actividad neurológica de la metámera (ASIA) durante el examen físico (neurofisiología) confirmado a través de: a.Electromiograma con medición de unidades motoras individuales para medir la actividad de la motoneurona eferente (fibras A? y C). b.Potenciales evocados somatosensitivos (SSEP) para medir la actividad de las fibras A? y A? posteriores directas (fascículos posteriores de Goll y Burdach). -Cambios en la evaluación de la espasticidad: escala de Ashworth Modificada (MAS) (Rehabilitación) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Secondary endpoint will be assesed during the study visits (Visit 0, 1, 2, 3 and 4) |
Los objetivos secundarios se evaluarán durante las visitas del estudio (visita 0, 1, 2, 3, y 4) |
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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 | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 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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The study ends with the last visit of the last patient participating in the trial. |
El estudio finaliza con la última visita del último paciente que participa en el ensayo. |
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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 | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 0 |