A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease.

Официальное название

A Phase 3, Randomized, Double-Masked, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease Inadequately Controlled With Antithyroid Drugs

Обзор

The main purpose of this study is to look at how efgartigimod affects thyroid function in adults with Graves' Disease (GD). The study will also check whether efgartigimod is safe and well tolerated. It will look at how efgartigimod is distributed and eliminated in the body, how it changes antibody levels, and how the immune system responds to it. The study consists of a part A double-blinded treatment period, a part B treatment/observation period and a part C open-label treatment/observation period. During the part A and part B treatment periods, participants will receive efgartigimod PH20 SC via Prefilled Syringe (PFS) or placebo. During the part C open-label treatment period, participants will receive efgartigimod PH20 SC PFS. Participation in the different parts of the study will depend on the participant's response to treatment. The total study duration for participants ranges from 63 to 135 weeks, depending on the response to treatment. More information can be found here: https://clinicaltrials.argenx.com/vitalithy

Вмешательства

• Биопрепарат efgartigimod PH20 SC
  Subcutaneous injection of Efgartigimod PH20 Via Prefilled Syringe (PFS)
• Другое Placebo PH20 SC
  Subcutaneous injection of placebo PH20 via Prefilled Syringe (PFS)

Первичные конечные точки

• Percentage of participants who are euthyroid (fT3, fT4, and TSH within normal ranges) off ATDs at week 24 in part A [Срок оценки: up to 24 weeks (part A)]

Вторичные конечные точки (12)

• Time to becoming euthyroid off ATDs in part A [Срок оценки: Up to 24 weeks (part A)]
• Percentage of participants who are euthyroid and receiving MMI ≤5 mg/day or CBZ ≤7.5 mg/day at week 24 in part A [Срок оценки: up to 24 weeks (part A)]
• Percentage of participants with fT3 and fT4 levels below the ULN off ATDs at week 24 in part A [Срок оценки: Up to 24 weeks (part A)]
• Percentage of participants who are euthyroid off ATDs and TRAb seronegative at week 24 in part A [Срок оценки: up to 24 weeks (part A)]
• Time to becoming euthyroid off ATDs and TRAb seronegative [Срок оценки: up to 24 weeks (part A) + up to 135 weeks]
• Time to becoming euthyroid and receiving an ATD dose of MMI ≤5 mg/day or CBZ ≤7.5 mg/day (part A) [Срок оценки: up to 24 weeks]
• Time to becoming euthyroid [Срок оценки: up to 24 weeks (part A) + up to 135 weeks]
• Time to having fT3 and fT4 levels within normal ranges among participants who had fT3 and/or fT4 more than the ULN at baseline (part A) [Срок оценки: up to 24 weeks (part A)]
• Time to having TSH within normal ranges among participants who have TSH lower than the LLN at baseline (part A) [Срок оценки: up to 24 weeks (part A)]
• Percentage of participants who remain euthyroid without ATDs [Срок оценки: up to 74 weeks (part B)]
• Percentage of participants who remain euthyroid without ATDs and without efgartigimod PH20 SC PFS for 6, 12, and 18 months [Срок оценки: up to 74 weeks (part B) + up to 135 weeks]
• Percentage of participants who remain euthyroid without ATDs and without efgartigimod PH20 SC PFS [Срок оценки: up to 72 weeks (part C)]

Критерии участия

Критерии включения

• Is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF.
• Has a documented diagnosis of GD with TRAb (anti-thyrotropin receptor antibody) levels >=ULN (upper limit of normal) at screening
• Has active hyperthyroidism due to GD with TSH (thyroid-stimulating hormone) <0.1 mIU/L at screening
• Has been treated with MMI (methimazole) or CBZ (carbimazole) for at least 3 months before screening

Критерии исключения

• History of hyperthyroidism not caused by GD (eg, toxic adenoma or toxic multinodular goiter)
• History of RAI (radioactive iodine) therapy or received a total thyroidectomy
• T3- or T4-containing medication or supplement (eg, levothyroxine, liothyronine, desiccated thyroid preparations, or thyroid-support supplements) received <6 weeks before screening
• Any complication of hyperthyroidism or underlying medical condition that would put the participant at undue risk. This includes arrhythmia or tachyarrhythmia related to GD, such as atrial fibrillation or atrial flutter not sufficiently controlled with medications.
• Graves' orbitopathy/Thyroid Eye Disease (GO/TED) requiring systemic therapy (eg, corticosteroids), orbital injections, orbital surgery, or orbital radiation, or expected immediate surgical intervention and/or planned corrective surgery/irradiation or medical therapy during the study

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

Четверное слепое

Основная цель

Лечение

Центры проведения

Япония · 4 центра

• Aso Iizuka Hospital — Iizuka
• National Hospital Organization Kyoto Medical Center — Kyoto
• SHOWA Medical University Northern Yokohama Hospital — Yokohama
• Tottori University Hospital — Yonago

Грузия · 3 центра

• High Technology Hospital Medcenter LLC — Batumi
• First Medical Clinic Ltd — Tbilisi
• LTD MediClub Georgia — Tbilisi

Идентификаторы

NCT: NCT07596849 · ARGX-113-25-GRD-3001 · 2025-523333-26-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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