E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Thyroid Eye Disease (TED) |
Enfermedad Ocular Tiroidea (EOT) |
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E.1.1.1 | Medical condition in easily understood language |
Thyroid Eye Disease (TED) |
Enfermedad Ocular Tiroidea (EOT) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Eye Diseases [C11] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 23.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10084358 |
E.1.2 | Term | Thyroid eye disease |
E.1.2 | System Organ Class | 100000004853 |
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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 |
To describe batoclimab maintenance of efficacy as assessed by proptosis |
Describir la continuidad de la eficacia de batoclimab, evaluada mediante la proptosis. |
|
E.2.2 | Secondary objectives of the trial |
• To describe the efficacy of batoclimab in participants previously treated with placebo and did not achieve proptosis response in the feeder studies
• To describe the treatment effect of batoclimab in participants previously treated with batoclimab but did not achieve proptosis response in the feeder studies |
• Describir la eficacia de batoclimab en participantes tratados previamente con placebo y que no lograron una respuesta de la proptosis en los estudios preliminares.
• Describir el efecto del tratamiento con batoclimab en participantes tratados previamente con batoclimab pero que no lograron respuesta de la proptosis en los estudios preliminares. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
For all participants: 1. Have completed the Week 24 visit of the feeder study.
For participants assigned to the Open-label Treatment Cohort: 1. Do not require immediate surgical intervention and is not planning corrective surgery/irradiation or medical therapy for TED during the course of the study. 2. Did not permanently discontinue batoclimab
Additional inclusion criteria are defined in the protocol. |
Para todos los participantes: 1. Haber completado la visita de la semana 24 de los estudios preliminares.
Para los participantes asignados a la cohorte de tratamiento abierto: 1. No requerir intervención quirúrgica inmediata y no tener planificada una cirugía o irradiación correctiva ni tratamiento médico para la EOT durante el transcurso del estudio. 2. No haber suspendido permanentemente el tratamiento con batoclimab.
Los demás criterios de inclusión están definidos en el protocolo |
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E.4 | Principal exclusion criteria |
For all participants: 1. In the Investigator's judgement, the benefits of entry in the study do not outweigh the risk. |
Para todos los participantes: 1. A juicio del investigador, los beneficios de la participación en el estudio no superan el riesgo. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Duration of proptosis response off treatment in study eye in participants who were batoclimab responders in the feeder studies. |
Duración de la respuesta de la proptosis sin tratamiento en el ojo del estudio en los participantes que respondieron a batoclimab en los estudios preliminares. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• Proportion of proptosis responders at Week 24 in study eye in participants who were placebo non-responders in the feeder studies.
• Proportion of proptosis responders at Week 24 in study eye in participants who were batoclimab non-responders in the feeder studies. |
• Porcentaje de pacientes con respuesta de la proptosis en la semana 24 en el ojo del estudio en los participantes que no respondieron al placebo en los estudios preliminares.
• Porcentaje de pacientes con respuesta de la proptosis en la semana 24 en el ojo del estudio en los participantes que no respondieron a batoclimab en los estudios preliminares. |
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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 | Yes |
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 | 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 | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 39 |
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 |
Canada |
Israel |
Japan |
United States |
Austria |
France |
Latvia |
Poland |
Sweden |
Netherlands |
Spain |
Switzerland |
Czechia |
Germany |
Italy |
Belgium |
Denmark |
Hungary |
Slovakia |
Turkey |
United Kingdom |
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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
|
The end of study is defined as the date of the last visit of the last participant in the study. |
El final del estudio se define como la fecha de la última visita del último participante en el estudio. |
|
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 | 5 |
E.8.9.1 | In the Member State concerned days | |
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 | 5 |