A Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease

Официальное название

An Open-label Phase 2 Study to Investigate the Efficacy and Safety of Rilzabrutinib in Adult Participants With Graves' Disease

Обзор

This is a parallel group, Phase 2, 2-arm study to measure the treatment effect and safety of rilzabrutinib dose 1 or rilzabrutinib dose 2 in participants with Graves' disease, with and without Graves' orbitopathy, aged 18 years or older. Study details include: * Screening period (up to 4 weeks). * Treatment period (up to 16 weeks). * Follow-up period (4 weeks). The number of visits will be up to 13.

Вмешательства

• Препарат Rilzabrutinib dose 1
  Pharmaceutical form:Tablet-Route of administration:Oral
• Препарат Rilzabrutinib dose 2
  Pharmaceutical form:Tablet-Route of administration:Oral

Первичные конечные точки

• Percent change from baseline in FT4 levels [Срок оценки: At Week16]

Вторичные конечные точки (4)

• Percent change from baseline in FT3 levels [Срок оценки: At Week16]
• Proportion of participants with FT4 levels within normal limits [Срок оценки: At Week16]
• Proportion of participants with FT3 levels within normal limits [Срок оценки: At Week16]
• Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs) and withdrawals due to TEAEs during the study period [Срок оценки: Up to week 20]

Критерии участия

Критерии включения

• Participants who have a confirmed diagnosis of Graves' disease with active hyperthyroidism, with or without active Graves' orbitopathy at the time of screening.
• A subset of participants will have a diagnosis of active Graves' orbitopathy, as confirmed by ophthalmic exam at screening and a clinical activity score (CAS) ≥3 for the most severely affected eye, and associated with one or more of the following: lid retraction ≥2 mm, moderate or severe soft tissue involvement, proptosis ≥2 mm, and/or intermittent or constant diplopia.
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Критерии исключения

• History of hyperthyroidism not caused by Graves' disease (eg, hyperthyroidism due to toxic multinodular goiter, autonomous thyroid nodule, acute inflammatory thyroiditis).
• History of thyroid storm or at high risk of developing thyroid storm as determined by evaluating clinician.
• Enlarged thyroid goiter causing upper airway obstruction and/or requiring surgical intervention during the study period.
• For participant with Graves' orbitopathy, requires immediate surgical ophthalmological intervention or is planning corrective surgery/irradiation during the course of the study.
• Sight threatening Graves' orbitopathy or decreased visual acuity due to optic neuropathy within the last 6 months.
• Corneal decompensation unresponsive to medical management.
• Onset of Graves' orbitopathy symptoms >9 months prior to baseline.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Италия · 5 центров

• Investigational Site Number : 3800005 — Merano
• Investigational Site Number : 3800001 — Milan
• Investigational Site Number : 3800004 — Milan
• Investigational Site Number : 3800003 — Palermo
• Investigational Site Number : 3800002 — Pisa

Канада · 3 центра

• Investigational Site Number : 1240003 — Calgary
• Investigational Site Number : 1240002 — Surrey
• Investigational Site Number : 1240001 — Sherbrooke

Испания · 3 центра

• Investigational Site Number : 7240002 — Barcelona
• Investigational Site Number : 7240001 — Pamplona
• Investigational Site Number : 7240004 — Seville

Германия · 2 центра

• Investigational Site Number : 2760002 — Essen
• Investigational Site Number : 2760001 — Frankfurt

Великобритания · 2 центра

• Investigational Site Number : 8260003 — Milton Keynes
• Investigational Site Number : 8260001 — Newcastle upon Tyne

Идентификаторы

NCT: NCT06984627 · PDY18663 · 2025-521023-75 · U1111-1316-0208

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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