E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Thyroid Eye Disease (TED) |
|
E.1.1.1 | Medical condition in easily understood language |
Thyroid Eye Disease (TED) |
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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 | 10015919 - Eye disorders |
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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 |
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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 |
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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 study drug
Additional inclusion criteria are defined in the protocol. |
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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. |
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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. |
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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. |
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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 | 4 |
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 |
New Zealand |
Australia |
Canada |
Georgia |
Israel |
Japan |
United Kingdom |
United States |
Belgium |
Germany |
Hungary |
Italy |
Latvia |
Poland |
Slovakia |
Spain |
Türkiye |
|
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. |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 23 |
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 |