A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease. (UplighTED)
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ClinicalTrials.gov Identifier: NCT06307626 |
Recruitment Status :
Recruiting
First Posted : March 13, 2024
Last Update Posted : May 14, 2024
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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 the screening period, eligible participants will be randomized in a 2:1 ratio to receive efgartigimod PH20 SC or placebo PH20 SC, respectively during the double-blinded treatment period (DBTP). At the end of the DBTP, participants may enter a follow-up observational period while off study drug. Some participants may also enter the open-label treatment period with efgartigimod PH20 SC. The study duration varies from approximately 60 to 110 weeks.
Condition or disease | Intervention/treatment | Phase |
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Thyroid Eye Disease | Combination Product: Efgartigimod PH20 SC Other: Placebo PH20 SC | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 108 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | March 28, 2024 |
Estimated Primary Completion Date : | February 2026 |
Estimated Study Completion Date : | October 2027 |
Arm | Intervention/treatment |
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Experimental: Efgartigimod arm
Participants with active, moderate-to-severe TED receiving Efgartigimod PH20 SC (subcutaneously) via pre-filled syringe (PFS)
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Combination Product: Efgartigimod PH20 SC
Subcutaneous efgartigimod PH20 SC given by prefilled syringe |
Placebo Comparator: Placebo arm
Participants with active, moderate-to-severe TED receiving Placebo PH20 SC (subcutaneously) via pre-filled syringe (PFS)
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Other: Placebo PH20 SC
Subcutaneous placebo given by prefilled syringe |
- Percentage of participants who were proptosis responders [ Time Frame: At week 24 of the Double-Blinded Treatment Period ]
- Change in proptosis measurement in the study eye from baseline [ Time Frame: Up to 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)
- Percentage of participants with a resolution of diplopia (responders) [ Time Frame: At week 24 of the Double-Blinded Treatment Period ]
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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06307626
Contact: Sabine Coppieters, MD | 857-350-4834 | clinicaltrials@argenx.com |
United States, Florida | |
Advanced Research | Recruiting |
Boynton Beach, Florida, United States, 33437 | |
Contact: Sirtaz S. Sibia, MD 857-350-4834 clinicaltrials@argenx.com |
Responsible Party: | argenx |
ClinicalTrials.gov Identifier: | NCT06307626 |
Other Study ID Numbers: |
ARGX-113-2309 2023-509198-22-00 ( EU Trial (CTIS) Number ) |
First Posted: | March 13, 2024 Key Record Dates |
Last Update Posted: | May 14, 2024 |
Last Verified: | May 2024 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Eye Diseases Graves Ophthalmopathy Thyroid Diseases Endocrine System Diseases Eye Diseases, Hereditary Graves Disease Exophthalmos |
Orbital Diseases Genetic Diseases, Inborn Goiter Hyperthyroidism Autoimmune Diseases Immune System Diseases |