A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC PFS in Adult Participants With Graves' Disease.
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: efgartigimod PH20 SC, Placebo PH20 SC.
- Who it may be relevant to
- Registry conditions: Graves Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Georgia, Japan
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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
Interventions
- Biological efgartigimod PH20 SC
Subcutaneous injection of Efgartigimod PH20 Via Prefilled Syringe (PFS) - Other Placebo PH20 SC
Subcutaneous injection of placebo PH20 via Prefilled Syringe (PFS)
Primary outcome measures
- Percentage of participants who are euthyroid (fT3, fT4, and TSH within normal ranges) off ATDs at week 24 in part A [Time frame: up to 24 weeks (part A)]
Secondary outcome measures (12)
- Time to becoming euthyroid off ATDs in part A [Time frame: 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 [Time frame: 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 [Time frame: Up to 24 weeks (part A)]
- Percentage of participants who are euthyroid off ATDs and TRAb seronegative at week 24 in part A [Time frame: up to 24 weeks (part A)]
- Time to becoming euthyroid off ATDs and TRAb seronegative [Time frame: 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) [Time frame: up to 24 weeks]
- Time to becoming euthyroid [Time frame: 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) [Time frame: 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) [Time frame: up to 24 weeks (part A)]
- Percentage of participants who remain euthyroid without ATDs [Time frame: 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 [Time frame: up to 74 weeks (part B) + up to 135 weeks]
- Percentage of participants who remain euthyroid without ATDs and without efgartigimod PH20 SC PFS [Time frame: up to 72 weeks (part C)]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Japan · 4 centers
- Aso Iizuka Hospital — Iizuka
- National Hospital Organization Kyoto Medical Center — Kyoto
- SHOWA Medical University Northern Yokohama Hospital — Yokohama
- Tottori University Hospital — Yonago
Georgia · 3 centers
- High Technology Hospital Medcenter LLC — Batumi
- First Medical Clinic Ltd — Tbilisi
- LTD MediClub Georgia — Tbilisi
Identifiers
NCT: NCT07596849 · ARGX-113-25-GRD-3001 · 2025-523333-26-00