Efgartigimod for Thyroid Eye Disease (UplighTED Trial)
Palo Alto (17 mi)Age: 18+
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: argenx
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 4 jurisdictions
Trial Summary
What is the purpose of this trial?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.
An alternative list of clinical sites open for recruitment could be found in the other UplighTED study record (https://www.clinicaltrials.gov/study/NCT06307613)
Is the drug Efgartigimod PH20 SC a promising treatment for Thyroid Eye Disease?The drug Efgartigimod PH20 SC is considered promising for Thyroid Eye Disease because it targets the immune system, which plays a role in the disease. This approach could help reduce symptoms and improve the condition of patients with this eye disease.12359
What safety data is available for Efgartigimod in treating Thyroid Eye Disease?The provided research does not contain specific safety data for Efgartigimod or its variants in the treatment of Thyroid Eye Disease. The studies mentioned focus on other treatments like Batoclimab and Teprotumumab. Therefore, no safety data for Efgartigimod is available in the given research.168910
What data supports the idea that the drug Efgartigimod for Thyroid Eye Disease is an effective treatment?The available research does not provide specific data on the effectiveness of Efgartigimod for Thyroid Eye Disease. Instead, it highlights teprotumumab as a promising treatment for this condition, showing remarkable effectiveness in clinical trials and being approved by the U.S. FDA. Without specific data on Efgartigimod, we cannot conclude its effectiveness for Thyroid Eye Disease based on the provided information.34679
Do I have to stop taking my current medications for the trial?The trial protocol does not specify if you must stop taking your current medications. However, it mentions that the use of some medications before screening is an exclusion criterion, so you may need to discuss your current medications with the study team.
Eligibility Criteria
Adults with active, moderate-to-severe thyroid eye disease (TED) who've had symptoms start within the last year can join. They must be over 18, able to consent and follow the study plan, have their thyroid condition under control or only mildly off balance. Participants need to use birth control and women must test negative for pregnancy.Treatment Details
The trial is testing Efgartigimod PH20 SC against a placebo in people with TED. It's a randomized study where two-thirds get the real drug and one-third gets a fake drug (placebo), given by injection for 24 weeks. Responders are observed for another year without treatment; nonresponders get more of the real drug.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Efgartigimod armExperimental Treatment1 Intervention
Participants with active, moderate-to-severe TED receiving Efgartigimod PH20 SC (subcutaneously) via pre-filled syringe (PFS)
Group II: Placebo armPlacebo Group1 Intervention
Participants with active, moderate-to-severe TED receiving Placebo PH20 SC (subcutaneously) via pre-filled syringe (PFS)
Efgartigimod PH20 SC is already approved in European Union, United States, Japan, China for the following indications:
🇪🇺 Approved in European Union as VYVGART for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
🇺🇸 Approved in United States as VYVGART Hytrulo for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
- Chronic inflammatory demyelinating polyneuropathy (CIDP)
🇯🇵 Approved in Japan as VYVDURA for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
🇨🇳 Approved in China as Efgartigimod alfa injection (subcutaneous injection) for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
Find a clinic near you
Research locations nearbySelect from list below to view details:
Advanced ResearchBoynton Beach, FL
American Institute of ResearchLos Angeles, CA
Martel Eye Medical GroupRancho Cordova, CA
Butchertown Clinical TrialsLouisville, KY
More Trial Locations
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Who is running the clinical trial?
argenxLead Sponsor
References
Immunologically mediated cytotoxicity against human eye muscle and thyroid cells in euthyroid and thyrotoxic Graves' ophthalmopathy. [2019]We have studied the role of eye muscle and thyroid autoimmunity in patients with Graves' hyperthyroidism with or without ophthalmopathy in an area of relatively low iodine intake. Antibody dependent cell mediated cytotoxicity (ADCC) and complement mediated antibody dependent cytotoxicity (CMAC) against thyroid and eye muscle cells, and levels of antibodies against TSH receptor antigen and the thyroid microsomal antigen (thyroid peroxidase) were determined in three groups of patients: (1) thyrotoxic with exophthalmos (TX, n = 28), (2) thyrotoxic without ophthalmopathy (GR, n = 10), and (3) euthyroid ophthalmopathy (EU, n = 12). The thyroid glandular mass of the EU group was significantly less (P less than 0.01) compared with TX or GR. Mean (+/- SD) TSH receptor antibody (TRAb) level was 27 +/- 14% in EU which was significantly lower compared with TX (52.4 +/- 20%) and GR (59 +/- 18%). The prevalence of microsomal antibodies were similar and not significantly different in the three groups. On the other hand the prevalence of positive ADCC and CMAC tests was significantly greater, and at higher levels, in EU (ADCC THY CELLS 10.9 +/- 8.9% SL, ADCC Eye muscle = 25.9 +/- 20% SL, CMAC = 70.2 +/- 43% SL) and TX (ADCC THY CELLS = 9.3 +/- 9.2% SL, ADCC Eye muscle = 20.1 +/- 19% SL, CMAC = 62.4 +/- 30% SL) compared to GR (ADCC THY CELLS = 4.4 +/- 9.5% SL, ADCC Eye muscle = 7.7 +/- 6.7% SL, CMAC = 24.7 +/- 23% SL).(ABSTRACT TRUNCATED AT 250 WORDS)
Increased frequency of euthyroid ophthalmopathy in patients with Graves' disease associated with myasthenia gravis. [2019]We previously showed that myasthenia gravis (MG) has a mild clinical expression when associated with autoimmune thyroid diseases (AITD). In the present study we have investigated the frequency of thyroid-associated ophthalmopathy (TAO) in patients with Graves' disease (GD) associated with MG as compared with GD patients without MG. A total of 418 patients with GD were studied, 31 with MG and 387 without MG. TAO was evaluated by physical examination, exophthalmometry, computerized tomography, and computerized visual fields assessment. The overall prevalence of TAO was similar in GD patients with MG (61.2%) and in those without MG (56.4%). When the analysis was restricted to GD patients with ocular MG, a greater frequency of TAO was found (84.6%), compared with GD patients without MG or with GD patients with generalized MG, although the differences did not reach the statistical significance. GD patients with MG had a significantly greater prevalence (12.9%) of euthyroid ophthalmopathy (clinically overt ophthalmopathy without previous and/or current hyperthyroidism) than those without MG (3.1%; p = 0.003). The results suggest a preferential association between the ocular manifestations of GD and MG, which may be due to immunological cross-reactivity against common autoimmune targets in the eye muscle as well as to a common genetic background.
Pentoxifylline (PTX)--an alternative treatment in Graves' ophthalmopathy (inactive phase): assessment by a disease specific quality of life questionnaire and by exophthalmometry in a prospective randomized trial. [2017]To investigate the effect of pentoxifylline (PTX) in subjects with inactive Graves' ophthalmopathy (GO) through a specific quality of life (QOL) questionnaire and exophthalmometry readings.
Euthyroid and primarily hypothyroid patients develop milder and significantly more asymmetrical Graves ophthalmopathy. [2022]Retrospective, observational study to compare clinical symptoms and TSH-receptor antibodies (TRAb) in Graves ophthalmopathy (GO) in euthyroid and primarily hypothyroid patients to those in hyperthyroid patients.
Presence of thyroid-associated ophthalmopathy in Hashimoto's thyroiditis. [2021]To determine the prevalence of ophthalmopathy in Hashimoto's patients and to make a comparison in subgroups of patients.
Thyroid-associated ophthalmopathy: Emergence of teprotumumab as a promising medical therapy. [2021]Thyroid-associated ophthalmopathy (TAO) remains a vexing autoimmune component of Graves' disease that can diminish the quality of life as a consequence of its impact on visual function, physical appearance and emotional well-being. Because of its relative rarity and variable presentation, the development of highly effective and well-tolerated medical therapies for TAO has been slow relative to other autoimmune diseases. Contributing to the barriers of greater insight into TAO has been the historical absence of high-fidelity preclinical animal models. Despite these challenges, several agents, most developed for treatment of other diseases, have found their way into consideration for use in active TAO through repurposing. Among these, teprotumumab is a fully human inhibitory monoclonal antibody against the insulin-like growth factor I receptor. It has shown remarkable effectiveness in moderate to severe, active TAO in two completed multicenter, double masked, and placebo controlled clinical trials. The drug exhibits a favorable safety profile. Teprotumumab has recently been approved by the U.S. F.D.A, and may rapidly become the first line therapy for this disfiguring and potentially blinding condition.
Teprotumumab as a Novel Therapy for Thyroid-Associated Ophthalmopathy. [2021]Thyroid-associated ophthalmopathy (TAO) has remained a vexing and poorly managed autoimmune component of Graves' disease where the tissues surrounding the eye and in the upper face become inflamed and undergo remodeling. This leads to substantial facial disfigurement while in its most severe forms, TAO can threaten eye sight. In this brief paper, I review some of the background investigation that has led to development of teprotumumab as the first and only US FDA approved medical therapy for TAO. This novel treatment was predicated on recognition that the insulin-like growth factor I receptor plays an important role in the pathogenesis of TAO. It is possible that a similar involvement of that receptor in other autoimmune disease may lead to additional indications for this and alternative insulin-like growth factor I receptor-inhibiting strategies.
Proof-of-concept and Randomized, Placebo-controlled Trials of an FcRn Inhibitor, Batoclimab, for Thyroid Eye Disease. [2023]Inhibition of the neonatal fragment crystallizable receptor (FcRn) reduces pathogenic thyrotropin receptor antibodies (TSH-R-Ab) that drive pathology in thyroid eye disease (TED).
Efficacy of teprotumumab therapy in patients with long-duration thyroid eye disease. [2023]Teprotumumab, an inhibitor of the insulin-like growth factor 1 receptor (IGF-1R), was approved by the US Food and Drug Administration in January 2020 for the treatment of thyroid eye disease (TED). The clinical trials leading to its approval enrolled patients with recent disease onset and significant inflammatory symptoms and signs. Subsequent real-world teprotumumab use in patients with longer duration of disease also may be effective, and there have been several publications reporting on experience in these patient groups.
Teprotumumab-Related Adverse Events in Thyroid Eye Disease: A Multi-Center Study. [2023]To assess the duration, incidence, reversibility, and severity of adverse events (AEs) in patients with thyroid eye disease (TED) treated with teprotumumab.