E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic Lateral Sclerosis (ALS) |
esclerosis lateral amiotrófica |
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E.1.1.1 | Medical condition in easily understood language |
Lou Gehrig's disease (ALS) |
esclerosis lateral amiotrófica (ELA) |
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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 | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002026 |
E.1.2 | Term | Amyotrophic lateral sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 of the study is to evaluate the long-term safety profile of dexpramipexole in subjects with ALS. |
El objetivo principal del estudio es evaluar el perfil de seguridad a largo plazo del dexpramipexol en sujetos con ELA. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to evaluate the long-term efficacy of dexpramipexole in this study population using clinical endpoints measuring function and survival. |
El objetivo secundario de este estudio es evaluar la eficacia a largo plazo del dexpramipexol en esta población de estudio mediante criterios de valoración clínicos que miden la función y la supervivencia. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subject has the ability to understand the purpose and risks of the study and provide signed and dated informed consent (or have the consent confirmed by a witness if unable to write) and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations. 2. Subject was enrolled in either Study CL211 or Study 223AS302. 3. Subject has completed their last visit in Study CL211 or Study 223AS302. 4. Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment. For further details of contraceptive requirements for this study |
1. El sujeto es capaz de comprender el objetivo y los riesgos del estudio y de dar el consentimiento informado firmado y fechado (o hacer que un testigo confirme el consentimiento, si no es capaz de escribir) y la autorización para usar información de salud protegida (protected health information, PHI), de acuerdo con las normativas nacionales y locales sobre la privacidad de los sujetos. 2. El sujeto estuvo inscrito en el estudio CL211 o en el estudio 223AS302. 3. El sujeto ha completado su última visita en el estudio CL211 o en el estudio 223AS302. 4. Los sujetos con capacidad reproductiva deben utilizar medidas anticonceptivas eficaces durante el estudio, así como estar dispuestos y ser capaces de continuar con ellas durante 1 mes (mujeres) o 3 meses (varones) después de su última dosis del tratamiento del estudio. Para obtener más detalles sobre los requisitos relativos al uso de métodos anticonceptivos para este estudio |
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E.4 | Principal exclusion criteria |
1. Subject withdrew prematurely from Study CL211 or Study 223AS302. 2. Subject permanently discontinued study treatment in Study CL211 or Study 223AS302 for any reason other than enrollment into this study. 3. Subject from Study CL211 or Study 223AS302 has a significant change in medical history (including laboratory tests or a clinically significant condition) that in the opinion of the Investigator would impair the subject?s medical fitness for participation and preclude treatment. 4. Female subject who is pregnant or breastfeeding. 5. Subject is currently enrolled in any investigational drug study other than Study CL211 or Study 223AS302. 6. Subject is taking pramipexole, other dopamine agonists, any other agent with dopaminergic activity, or any other disallowed concomitant medication 7. Subject is unwilling or unable to comply with the requirements of the protocol including the presence of any condition (physical, mental, or social) that is likely to affect the subject?s ability to comply with the protocol. At a minimum, subjects who are not able to travel to the study site must be willing to agree to remote blood draws for clinical laboratory evaluations and telephone visits to report AEs, concomitant medications, and ALSFRS-R scores. |
1. El sujeto se retiró anticipadamente del estudio CL211 o del estudio 223AS302. 2. El sujeto interrumpió permanentemente el tratamiento del estudio en el estudio CL211 o 223AS302 por cualquier motivo distinto a la inscripción en el presente estudio. 3. El sujeto del estudio CL211 o 223AS302 tiene un cambio significativo en la historia clínica (incluidas pruebas de laboratorio o una enfermedad clínicamente significativa) que según la opinión del investigador impediría que el sujeto tuviera el estado médico adecuado para participar e impediría el tratamiento. 4. Sujeto del sexo femenino que esté embarazada o en periodo de lactancia. 5. El sujeto está inscrito en la actualidad en un estudio de algún fármaco en investigación distinto al estudio CL211 o al 223AS302. 6. El sujeto está tomando pramipexol, otros agonistas de la dopamina, algún otro medicamento con actividad dopaminérgica o cualquier otro medicamento concomitante prohibido. 7. El sujeto no quiere o no puede cumplir con los requisitos del protocolo, lo que incluye presencia de cualquier enfermedad (física, mental o social) que probablemente afecte la capacidad del sujeto de cumplir con el protocolo. Como mínimo, los sujetos que no puedan desplazarse hasta el centro del estudio deben estar dispuestos a someterse a extracciones de sangre a distancia para la realización de las evaluaciones de laboratorio clínico y a llevar a cabo entrevistas telefónicas para informar de los AA, de los medicamentos concomitantes y de las puntuaciones de la ALSFRS-R. |
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E.5 End points |
E.5.1 | Primary end point(s) |
- incidence of AEs and SAEs - discontinuation of study treatment due to an AE - changes in vital signs, clinical laboratory assessments (hematology, blood chemistry, and urinalysis), 12-lead ECGs, and body weight - incidence of laboratory abnormalities |
- La incidencia de AA y AAG - La interrupción del tratamiento del estudio debido a un AA - Los cambios en las constantes vitales, las evaluaciones de laboratorio clínico (hematología, bioquímica sanguínea y análisis de orina), los electrocardiogramas (ECG) de 12 derivaciones y el peso corporal - La incidencia de anomalías de laboratorio |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
As necessary |
Lo necesario |
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E.5.2 | Secondary end point(s) |
- change in ALSFRS-R score - decline in SNIP - time to death - time to death or death equivalent (tracheostomy or permanent assisted ventilation [PAV], defined as use of noninvasive ventilation [NIV] for ?22 hours per day for ?10 days) |
- El cambio en la puntuación de la ALSFRS-R - La reducción de la SNIP - El tiempo hasta la muerte - El tiempo hasta la muerte o equivalente a la muerte (traqueotomía o ventilación asistida permanente [VAP], definida como el uso de ventilación no invasiva [VNI] durante ? 22 horas al día durante ? 10 días) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
As necessary |
Lo necesario |
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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) | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 28 |
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 |
Australia |
Canada |
United States |
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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 is last subject, last visit. |
El fin de estudio es último paciente, última visita. |
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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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |