E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
An immune system disorder that results in the overproduction of thyroid hormones (hyperthyroidism). |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 26.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018706 |
E.1.2 | Term | Graves' disease |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
|
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 investigate the effects of batoclimab on thyroid hormone levels in participants with GD as assessed by reduction of FT3 and FT4 at Week 24 |
|
E.2.2 | Secondary objectives of the trial |
To investigate the effects of batoclimab on thyroid hormone levels in participants with GD as assessed by reduction of FT3 and FT4 and changes in ATD at Week 24.
To investigate the effects of batoclimab on thyroid hormone levels in participants with GD as assessed by reduction of FT3 and FT4 at Week 24 and ATD treatment.
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female >= 18 years of age. 2. Have a serologically confirmed GD as documented by presence of elevated stimulatory TSH-R-Ab level (i.e., > SRR 140 %) at the Screening Visit. 3. Have active hyperthyroidism due to GD with the following laboratory values at the Screening Visit: • TSH < lower limit of normal (LLN) • FT3 > upper limit of normal (ULN) and ≤5× ULN • FT4 > ULN and ≤5 × ULN Note: Participants who have T3 thyrotoxicosis (i.e., TSH <ULN, FT3 > ULN and ≤5× ULN, but FT4 within normal range) at the Screening Visit may be enrolled, if they have serologically confirmed GD as per Inclusion Criterion 1. 4. Meet all of the following ATD requirements at the Screening Visit: • Have been on an ATD for ≥12 weeks before the Screening Visit. • Had starting total daily ATD dose of ≥20 mg of methimazole or carbimazole, or ≥200 mg of propylthiouracil. • Are currently treated with a total daily dose of ≥10 mg of methimazole or carbimazole, or ≥100 mg of propylthiouracil. 5. Other, more specific inclusion criteria are defined in the protocol. |
|
E.4 | Principal exclusion criteria |
1. History of hyperthyroidism not caused by GD (e.g., toxic adenoma or toxic multinodular goiter), and/or history or presence of thyroid storm. 2. History of treatment with radioactive iodine or thyroid surgery. Note: Participants may be enrolled if they meet Inclusion Criteria 3 and 4 listed in this summary (numbered as Inclusion Criteria 2 and 3 in the Protocol). 3. Total IgG level <6 grams per liter (g/L) at the Screening Visit. 4. Albumin level <3.5 grams per deciliter (g/dL) (<35 g/L) at the Screening Visit. 5. Absolute neutrophil count <1000 cells per cubic millimeter (cells/mm^3) at the Screening Visit. 6. Have been enrolled in a previous RVT-1401, IMVT-1401 clinical trial. Note: Participants previously enrolled in an IMVT-1401 clinical trial, with documented assignment to placebo treatment only, may be enrolled in this study. 7. Other, more specific exclusion criteria are defined in the protocol.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of participants who, at Week 24, without increase in ATD dose compared to baseline, have achieved normalization of FT3 and FT4, or have FT3 and/or FT4 below the LLN. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
1. Proportion of participants who achieve normalization of FT3 and FT4 at Week 24 with ATD dose ≤50% of the baseline ATD dose by Week 24. 2. Proportion of participants who are off ATD treatment and achieve normalization of FT3 and FT4, or have FT3 and/or FT4 below the LLN at Week 24.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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) | 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 | 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 | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
|
|
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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
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 | 0 |