- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06307626
A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease.
A Phase 3, Randomized, Double-Masked, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Thyroid Eye Disease
This study aims to evaluate the efficacy, safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of efgartigimod PH20 SC in participants with active, moderate-to-severe TED, compared with placebo PH20 SC.
After an up to 28-day screening period, eligible participants will be randomized in a 2:1 ratio to receive efgartigimod PH20 SC or placebo PH20 SC, respectively. Study drug will be administered subcutaneously during the 24-week double-blinded treatment period (DBTP). At the end of the DBTP (week 24), proptosis responders will enter a follow-up observational period (52 weeks) to assess the safety, tolerability, and durability of efgartigimod PH20 SC treatment while off therapy. Proptosis nonresponders and participants who have proptosis relapse during the 52-week follow-up observational period will receive open-label treatment with efgartigimod PH20 SC for up to 24 weeks.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Contacts and Locations
Study Contact
- Name: Sabine Coppieters, MD
- Phone Number: 857-350-4834
- Email: clinicaltrials@argenx.com
Study Locations
-
-
Florida
-
Boynton Beach, Florida, United States, 33437
- Recruiting
- Advanced Research
-
Contact:
- Sirtaz S. Sibia, MD
- Phone Number: 857-350-4834
- Email: clinicaltrials@argenx.com
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- The participant is at least 18 years of age
- The participant is capable of providing signed informed consent and following with protocol requirements
- The participant has a physician's diagnosis of active, moderate-to-severe thyroid eye disease (TED) associated with autoimmune thyroid conditions (Graves' disease or Hashimoto's thyroiditis) for the most severely affected eye
- The participant has onset of active TED symptoms within 12 months before screening
- The participant must have normal thyroid function with the baseline disease under control or have mild hypo or hyperthyroidism at screening. Every effort should be made to correct the mild hypo or hyperthyroidism promptly and to maintain the normal thyroid function for the full duration of the study
- The participant agrees to use birth control consistent with local regulations and the people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug
Exclusion Criteria:
- Optic neuropathy (damage to optic nerve), defined as new visual field defect (blind spot), relative afferent pupillary defect (pupils respond differently to light), or color defect secondary to optic nerve involvement within the 6 months before screening
- Corneal decompensation (swelling of the cornea) unresponsive to medical management
- Previous orbital irradiation or surgery for TED
- Use of some medications before screening (more information is found in the protocol)
- Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of TED or puts the participant at undue risk
- History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
- Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
- Current participation in another interventional clinical study or previous participation in an efgartigimod clinical study and at least 1 dose of study drug received or has received at least 1 dose of commercially available efgartigimod
- Known hypersensitivity to study drug or one of its excipients (inactive ingredients)
- History of or current alcohol, drug, or medication abuse within 12 months before screening as assessed by the investigator
- Pregnant or lactating state or intention to become pregnant during the study
- Live or live-attenuated vaccine received <4 weeks before screening
The complete list of exclusion criteria can be found in the protocol.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Efgartigimod arm
Participants with active, moderate-to-severe TED receiving Efgartigimod PH20 SC (subcutaneously) via pre-filled syringe (PFS)
|
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer
|
Placebo Comparator: Placebo arm
Participants with active, moderate-to-severe TED receiving Placebo PH20 SC (subcutaneously) via pre-filled syringe (PFS)
|
Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Percentage of participants who were proptosis responders
Time Frame: At week 24 of the Double-Blinded Treatment Period
|
At week 24 of the Double-Blinded Treatment Period
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in proptosis measurement in the study eye from baseline
Time Frame: Up to week 24 of the Double-Blinded Treatment Period
|
Up to week 24 of the Double-Blinded Treatment Period
|
|
Percentage of participants with a resolution of diplopia (responders)
Time Frame: At week 24 of the Double-Blinded Treatment Period
|
At week 24 of the Double-Blinded Treatment Period
|
|
Change in the total Graves' Orbitopathy Quality of Life (GO-QoL) score from baseline
Time Frame: Up to week 24 of the Double-Blinded Treatment Period
|
Minimum value: 0 (most negative impact on Quality of Life); Maximum value: 100 (No impact on Quality of Life)
|
Up to week 24 of the Double-Blinded Treatment Period
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ARGX-113-2309
- 2023-509198-22-00 (Ctis)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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