E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Progressive Supranuclear Palsy |
Parálisis supranuclear progresiva |
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E.1.1.1 | Medical condition in easily understood language |
PSP is a disease that attacks the brain, causing problems with body movement, memory, thinking, and behavior. Symptoms get worse over time and eventually make doing normal, daily tasks impossible. |
La PSP es una enf q ataca el cerebro y causa problemas relac con el movcorporal, la mem, el pmto y el comptto. Los sínts empeoran con el tiempo y hace q sea imposible realizar las tareas diarias norm. |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036813 |
E.1.2 | Term | Progressive supranuclear palsy |
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 |
- To assess the efficacy of ABBV-8E12 in slowing disease progression in subjects with progressive supranuclear palsy as measured by the PSP Rating Scale (PSP-RS). - To assess the long term safety and tolerability of ABBV-8E12 for up to 52 weeks in subjects with progressive supranuclear palsy. |
- Evaluar la eficacia de ABBV-8E12 en la ralentización de la progresión de la enfermedad en sujetos con parálisis supranuclear progresiva, medida por la Escala de Clasificación de PSP (PSP-RS). - Evaluar la seguridad y tolerabilidad a largo plazo de ABBV-8E12 durante 52 semanas en sujetos con parálisis supranuclear progresiva. |
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E.2.2 | Secondary objectives of the trial |
- To assess the pharmacokinetics of ABBV-8E12 in subjects with PSP. - To assess the efficacy of ABBV-8E12 in slowing disease progression and functional impairment in subjects with PSP as measured by secondary endpoints. - To assess the efficacy of ABBV-8E12 in slowing regional and/or whole brain atrophy in subjects with PSP as measured by volumetric MRI. |
- Evaluar la farmacocinética de ABBV-8E12 en sujetos con PSP. - Evaluar la eficacia de ABBV-8E12 en la ralentización de la progresión de la enfermedad y el deterioro funcional en sujetos con PSP, medida por objetivos secundarios. - Evaluar la eficacia de ABBV-8E12 en la disminución de la atrofia cerebral regional y/o total en sujetos con PSP, medida por resonancia magnética volumétrica. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Male or female subject with age 40 years or greater at the time of signed consent. • Meets the following criteria for possible or probable progressive supranuclear palsy (Steele-Richardson-Olszewski Syndrome) - gradually progressive disorder, with age at disease onset greater than or equal to 40 years - either or both of the following two items are met: 1. vertical supranuclear gaze palsy 2. slowing of vertical saccades AND postural instability with falls within the first 3 years of PSP symptoms • Presence of PSP symptoms for less than 5 years. • Subject is able to walk 5 steps with minimal assistance (stabilization of one arm or use of cane/walker). • Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend). |
• Sujeto masculino o femenino con 40 años de edad o más en el momento de la firma del consentimiento informado • Cumple con los siguientes criterios para la posible o probable parálisis supranuclear progresiva (Síndrome de Steele-Richardson-Olszewski): - trastorno gradualmente progresivo, con edad mayor o igual a 40 años al inicio de la enfermedad - Que se cumplan uno o ambos de los siguientes elementos: 1. parálisis supranuclear vertical de la mirada 2. ralentización de los movimientos sacádicos verticales e inestabilidad postural con caídas dentro de los primeros 3 años de síntomas de PSP • Presencia de síntomas de PSP de menos de 5 años. • Sujeto que pueda caminar 5 pasos con asistencia mínima (estabilización de un brazo o uso de bastón/andador). • Sujeto que tenga un compañero de estudio identificado y de confianza (por ejemplo, un cuidador, un miembro de la familia, un trabajador social o un amigo). |
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E.4 | Principal exclusion criteria |
• Subjects who weigh less than 44 kg (97 lbs) at screening. • MMSE score less than 15 at screening • Any contraindication or inability to tolerate brain magnetic resonance imaging (MRI) • Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period. • Evidence of any clinically significant neurological disorder other than PSP • The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria. • Subject has had a significant illness or infection requiring medical intervention in the past 30 days. |
• Sujetos que pesan menos de 44 kg (97 libras) en el periodo de selección • Puntuación de MMSE menor a 15 en el periodo de selección • Cualquier contraindicación o incapacidad para tolerar la resonancia magnética cerebral (RM) • Sujeto que reside en una institución de cuidados especializados de enfermería o demencia, o cuya admisión a dicha instalación esté planificada durante el período de estudio • Evidencia de cualquier trastorno neurológico clínicamente significativo que no sea PSP • Sujeto con un historial de esquizofrenia, trastorno esquizoafectivo o trastorno bipolar, o que lo padece actualmente, de acuerdo con los criterios DSM-V o ICD-10. • Sujeto ha tenido una enfermedad o infección importante que requirió intervención médica en los últimos 30 días. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The change from baseline to Week 52 on the PSP-RS total score. |
El cambio desde el inicio hasta la Semana 52 en la puntuación total de PSP-RS. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Weeks 12, 24, 36 and 52 |
Semanas 12, 24, 36 y 52 |
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E.5.2 | Secondary end point(s) |
1. To assess the pharmacokinetics of ABBV-8E12 in subjects with progressive supranuclear palsy. 2. To assess the efficacy of ABBV-8E12 in slowing disease progression and functional impairment in subjects with progressive supranuclear palsy as measured by the SEADL, UPDRS Part II, CGI-S, CGI-C, and PSP-QoL. 3. To assess the efficacy of ABBV-8E12 in slowing regional and/or whole brain atrophy in subjects with progressive supranuclear palsy as measured by volumetric MRI. |
1. Evaluar la farmacocinética de ABBV-8E12 en sujetos con parálisis supranuclear progresiva. 2. Evaluar la eficacia de ABBV-8E12 en la ralentización de la progresión de la enfermedad y el deterioro funcional en sujetos con parálisis supranuclear progresiva medida por SEADL, UPDRS Parte II, CGI-S, CGI-C y PSP-QoL. 3. Evaluar la eficacia de ABBV-8E12 en la ralentización de la atrofia cerebral regional y/o total en sujetos con parálisis supranuclear progresiva, medida por resonancia magnética volumétrica. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Day 1, Day 5 (C1), Day 15, Day 29, Day 57 (C1), Day 85, Day 89 (C1), Day 99 (C1), Week 16 (C1), Week 24, Week 36, Week 52, Week 60, Week 68 2. SEADL, UPDRS, – Screen, Day 1, Week 12, Week 24, Week 36, Week 52 CGI-S-SV1, SV2, Day 1, Week 8, Week 12, Week 20, Week 24, Week 32, Week 36, Week 48 and Week 52 CGI-C-Weeks 8, Week 12, Week 20, Week 24, Week 32, Week 36, Week 48 and Week 52 PSP-QoL-SV1, Day 1, Week 12, Week 24, Week 36, Week 52 3. Screen, Day 15 (C1), Week 12, Week 24, Week 52 |
1. Día 1, Día 5 (C1), Día 15, Día 29, Día 57 (C1), Día 85, Día 89 (C1), Día 99 (C1), Semana 16 (C1), Semana 24, Semana 36, Semana 52, Semana 60, Semana 68 2. SEADL, UPDRS, - Periodo de selección, Día 1, Semana 12, Semana 24, Semana 36, Semana 52 CGI-S-SV1, SV2, Día 1, Semana 8, Semana 12, Semana 20, Semana 24, Semana 32, Semana 36, Semana 48 y Semana 52 CGI-C-Semanas 8, Semana 12, Semana 20, Semana 24, Semana 32, Semana 36, Semana 48 y Semana 52 PSP-QoL-SV1, Día 1, Semana 12, Semana 24, Semana 36, Semana 52 3. Periodo de selección, Día 15 (C1), Semana 12, Semana 24, Semana 52 |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | Yes |
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 | Yes |
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 | 3 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 16 |
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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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 | 6 |
E.8.9.1 | In the Member State concerned days | 20 |
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 | 6 |
E.8.9.2 | In all countries concerned by the trial days | 20 |