E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Huntington's disease (HD) |
Enfermedad de Huntington (EH) |
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E.1.1.1 | Medical condition in easily understood language |
Huntington's disease is an inherited disease that causes the progressive breakdown (degeneration) of nerve cells in the brain |
La enfermedad de Huntington es una enfermedad hereditaria que causa el colapso progresivo (degeneración) de las células nerviosas en el cerebro. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
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 | 10070668 |
E.1.2 | Term | Huntington's disease |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 evaluate the safety and tolerability of RO7234292 |
Evaluar la seguridad y tolerabilidad de RO7234292 |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Prior enrollment in a Roche sponsored or Genentech-sponsored study in HD for the RO7234292 development program that made provision for entry into an OLE study - Ability and willingness to comply with the study protocol including the visit schedule and all assessments, in the investigator's judgment (apart from patients joining from Study BN40697 who are unable to undergo MRI) - For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, during the treatment period and for 5 months after the final dose of study drug - For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm during the treatment period and for 5 months after the final dose of study drug to avoid exposing the embryo |
-Participación previa en un estudio sobre la EH patrocinado por Roche o Genentech para el programa de desarrollo de RO7234292 en el que estuviera prevista la participación en una ERA. -Capacidad y voluntad de cumplir el protocolo del estudio, incluido el calendario de visitas y todas las evaluaciones, según el criterio del investigador (aparte de los pacientes procedentes del estudio BN40697 que no puedan someterse a una RM). -Mujeres en edad fértil: deberán comprometerse a practicar la abstinencia (no mantener relaciones sexuales) o a usar métodos anticonceptivos, durante el periodo de tratamiento y por 5 meses después de la última dosis del fármaco del estudio. -Varones: compromiso de practicar la abstinencia sexual (abstenerse de mantener relaciones heterosexuales) o de utilizar preservativo y compromiso de no donar semen, durante el periodo de tratamiento y durante al menos 5 meses después de la última dosis del fármaco del estudio para no exponer al embrión. |
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E.4 | Principal exclusion criteria |
- Withdrawal of consent from the preceding study - Permanent discontinuation of RO7234292 for any drug-related safety concern during the preceding study or meeting of any study treatment discontinuation criteria specified in the preceding study at the time of enrollment into this study - An ongoing, unresolved, clinically significant medical problem that in the judgment of the investigator would make it unsafe for the patient to participate in this study - Antiplatelet or anticoagulant therapy within 14 days prior to inclusion or anticipated use during the study, including, but not limited to, aspirin (unless <=81 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban - History of bleeding diathesis or coagulopathy - Platelet count less than the lower limit of normal - Concurrent participation in any therapeutic clinical trial (other than the preceding study) - Study treatment (RO7234292) is commercially marketed in the patient’s country for the patient-specific disease and is accessible to the patient - Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug |
-Retirada del consentimiento para el estudio anterior. -Suspensión permanente del tratamiento con RO7234292 por cualquier problema de seguridad relacionado con el fármaco durante el estudio precedente, o cumplimiento de alguno de los criterios de suspensión del tratamiento del estudio especificados en el estudio precedente en el momento de la inclusión en el presente estudio. -Un problema médico en curso, no resuelto y clínicamente importante que, en opinión del investigador, haría que no fuera segura la participación del paciente en el estudio. -Tratamiento con antiagregantes plaquetarios o anticoagulantes en los 14 días previos a la inclusión o uso previsto durante el estudio, entre ellos, ácido acetilsalicílico (a menos que sea <= 81 mg/día), clopidogrel, dipiridamol, warfarina, dabigatrán, rivaroxabán y apixabán. -Antecedentes de diátesis hemorrágica o coagulopatía. -Recuento de plaquetas por debajo del límite inferior de la normalidad -Participación simultánea en algún ensayo clínico terapéutico (diferente del estudio precedente). -El tratamiento del estudio (RO7234292) está comercializado en el país del paciente para su enfermedad específica y es accesible para el paciente. -Embarazo o lactancia, o intención de quedarse embarazada durante el estudio o en los 5 meses siguientes a la última dosis del fármaco del estudio. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale 2. Change from baseline in targeted vital signs 3. Change from baseline in physical findings 4. Change from baseline in neurological findings 5. Change from baseline in behavioral findings, as assessed by the Columbia-Suicide Severity Rating Scale 6. Change from baseline in cognition 7. Change from baseline in targeted ECG results 8. Change from baseline in targeted clinical laboratory test results |
1. Incidencia e intensidad de los acontecimientos adversos, con determinación de la intensidad según la Escala de Gradación de la Intensidad de los Acontecimientos Adversos 2. Variación con respecto al momento basal de constantes vitales seleccionadas. 3. Variación con respecto al momento basal de los signos físicos. 4. Variación con respecto al momento basal de los signos neurológicos. 5. Variación con respecto al momento basal de los signos conductuales, evaluados mediante la Escala de Valoración del Riesgo de Suicidio de Columbia (C-SSRS). 6. Variación con respecto al momento basal del estado cognitivo. 7. Variación con respecto al momento basal de los resultados de ECG seleccionados. 8. Variación con respecto al momento basal de los resultados de pruebas analíticas seleccionadas. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. Up to 5 years 2-8. Baseline to up to 5 years |
1. Hasta un máximo de 5 años 2-8. Desde basal hasta un máximo de 5 años |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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 |
PD Biomarker effects Immunogenicity |
Efectos FD de los biomarcadores Inmunogenicidad |
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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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 53 |
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 |
Austria |
Canada |
Chile |
Denmark |
France |
Germany |
Italy |
Netherlands |
New Zealand |
Poland |
Russian Federation |
Spain |
Switzerland |
United Kingdom |
United States |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
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 | 5 |
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 |