E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Alzheimer?s Disease and behavioral variant Frontotemporal Dementia |
Enfermedad de Alzheimer o demencia frontotemporal, variante conductual |
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E.1.1.1 | Medical condition in easily understood language |
Alzheimer's Disease and Frontotemporal Dementia |
Enfermedad de Alzheimer o demencia frontotemporal, variante conductual |
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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 | 17.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10068968 |
E.1.2 | Term | Frontotemporal dementia |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10001896 |
E.1.2 | Term | Alzheimer's disease |
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 objectives of this study are to provide subjects who have completed participation in a Phase 2 or Phase 3 trial continued access to therapy and to evaluate the long-term safety and tolerability of LMTM given in flexible doses of up to 300 mg/day. |
El objetivo del estudio es proporcionar a los pacientes que ha completado su participación en estudios de fase 2 o fase 3 un acceso continuado a la terapia y evaluar la seguridad y tolerancia a largo término del LMTM dado en dosis flexibles por encima de 300 mg/día. |
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E.2.2 | Secondary objectives of the trial |
Not applicable |
No aplicable |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects with a diagnosis according to NIA/AA criteria of all cause dementia and probable Alzheimer?s disease at enrollment and who completed participation in one of the following three TauRx studies (inclusive of the 4-week post-treatment follow-up visit): TRx-237-005, TRx-237-008, or TRx-237-015.
OR
Subjects with a diagnosis of probable bvFTD according to the International Consensus Criteria for bvFTD at enrollment and who completed participation in TauRx study TRx-237-007 through Visit 9 (Week 52).
Treatment will not be made available to subjects who have withdrawn from the double-blind study of prior participation prior to completion.
2. Females of childbearing potential must continue to use adequate contraception defined as follows (or, if in Italy, agree to avoid pregnancy): ? Barrier method (such as condom, diaphragm or cervical/vault cap) with spermicidal foam, gel, film, cream, or suppository ? Intrauterine device [IUD] or system ? Oral or long-acting injected or implanted contraceptives for at least 3 months prior to Baseline ? Vasectomized partner (with the appropriate post-vasectomy documentation of the absence of spermatozoa in the ejaculate) ? True abstinence (when this is in line with the preferred and usual lifestyle of the subject) Subjects must agree to continue to maintain adequate contraception throughout participation in the study.
3. Subject and/or, in the case of reduced decision-making capacity, legally acceptable representative(s) consistent with national law and IRB/EC approval, is/are able to read, understand, and provide written informed consent in the designated language of the study site ? In Germany, subjects must be able to provide their own written informed consent.
4. Has an identified adult caregiver who meets the following criteria: ? Either lives with the subject or sees the subject on average for ? 1 hour/day ? 3 days/week, and in the investigator?s opinion, the extent of contact is sufficient to provide meaningful assessment of changes in subject behavior and function over time and provide information on safety and tolerability ? Is willing to provide written informed consent for his/her own participation ? Is able to read, understand, and speak the designated language at the study site ? Agrees to accompany the subject to each study visit ? Is able to verify daily compliance with study drug
5. Able to comply with the study procedures in the view of the investigator |
1. Sujetos con un diagnóstico conforme a los criterios NIA/AA de demencia por todas las causas y probable enfermedad de Alzheimer (EA) en la inclusión y que completaron la participación en uno de los tres estudios de TauRx siguientes (incluyendo la visita de seguimiento postratamiento a las 4 semanas): TRx-237-008, TRx-237-015 y TRx-237-005.
O
Sujetos con un diagnóstico de probable demencia frontotemporal, variante conductual (DFTvc) conforme a los Criterios del Consenso Internacional de DFTvc en la inclusión y que completaron la participación en el estudio de TauRx, TRx-237-007, hasta la visita 9 (semana 52).
El tratamiento no estará disponible para los sujetos que abandonaron el estudio doble ciego en el que participaron previamente antes de completarlo. 2. Las mujeres con capacidad de gestación deben continuar utilizando anticonceptivos eficaces (o, si están en Italia, aceptar evitar el embarazo) como se define a continuación: ? método de barrera (como preservativo, diafragma o capuchón cervical) con espermicida en espuma, gel, película, crema u óvulo; sistema o dispositivo intrauterino (DIU); anticonceptivos hormonales orales o inyectados o implantados de acción prolongada durante al menos 3 meses antes de la visita basal; o pareja vasectomizada (con ausencia documentada de espermatozoides en el eyaculado tras la vasectomía); o abstinencia real (cuando concuerda con el estilo de vida preferida y habitual del sujeto) ? los sujetos deben aceptar continuar utilizando anticonceptivos eficaces durante toda la participación en el estudio 3. El sujeto y/o, en caso de una capacidad reducida para la toma decisiones, el(los) representante(s) legalmente autorizado(s) de conformidad con la legislación nacional y la aprobación del CEIC, son capaces de leer, entender y otorgar el consentimiento informado por escrito en el idioma designado del centro del estudio ? En Alemania, los sujetos deben ser capaces de otorgar su propio consentimiento informado por escrito 4. El sujeto tiene un cuidador adulto identificado que cumple los siguientes criterios: ? o vive con el sujeto o ve al sujeto una media de ?1 hora/día ?3 días/semana y, en opinión del investigador, el contacto es suficiente para proporcionar una evaluación significativa de los cambios en la conducta y la función del sujeto con el tiempo y aportar información sobre la seguridad y la tolerabilidad ? quiere otorgar el consentimiento informado por escrito para su propia participación ? es capaz de leer, entender y hablar el idioma designado del centro del estudio ? acepta acompañar al sujeto a cada visita del estudio ? es capaz de verificar el cumplimiento diario con el medicamento del estudio 5. El sujeto es capaz de cumplir con los procedimientos del estudio en la opinión del investigador |
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E.4 | Principal exclusion criteria |
1. History of swallowing difficulties (note: study drug should be swallowed whole and MUST NOT be broken, crushed or chewed or dissolved in fluids prior to ingestion)
2. Pregnant or breastfeeding
3. Clinically significant laboratory, pulse co-oximetry, electrocardiogram, or imaging abnormality (in originating study) or emergent intercurrent illness that, in the judgment of the principal investigator, could result in the risk of participation outweighing the potential benefit
4. Current participation in, or intent to enroll in, a clinical trial of a drug, biologic, device, or medical food |
1. Historia de dificultad para tragar (nota: el medicamento del estudio se debe tragar entero y NO DEBE romperse, triturarse, masticarse o disolverse en líquido antes de la ingesta) 2. Estar embarazada o en periodo de lactancia materna 3. Anomalía clínicamente significativa de laboratorio, pulsioximetría con niveles de CO, electrocardiograma o imagen (en el estudio original) o enfermedad intercurrente emergente que, en opinión del investigador principal, podrían hacer que el riesgo de participación fuera mayor que el posible beneficio 4. Participación actual, o intención de inscribirse, en un ensayo clínico de un medicamento, producto biológico, dispositivo o alimento médico |
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E.5 End points |
E.5.1 | Primary end point(s) |
Not applicable to this open-label extension study. |
No aplicable. Es un estudio abierto |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Not applicable to this open-label extension study. |
No aplicable. Es un estudio abierto |
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E.5.2 | Secondary end point(s) |
Not applicable to this open-label extension study. |
No aplicable. Es un estudio abierto |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Not applicable to this open-label extension study. |
No aplicable. Es un estudio abierto |
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability and resource utilisation |
Tolerabilidad y la utilización de recursos |
|
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 | Yes |
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 | 72 |
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 |
Argentina |
Australia |
Belgium |
Bulgaria |
Canada |
Croatia |
Finland |
France |
Germany |
Italy |
Korea, Republic of |
Malaysia |
Netherlands |
Poland |
Romania |
Russian Federation |
Singapore |
Spain |
Taiwan |
United Kingdom |
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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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 |