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Phase 3 Study for Thyroid Eye Disease Begins

OCTOBER 25, 2017
James Radke
A confirmatory Phase 3 clinical trial testing teprotumumab for moderate-to-severe active thyroid eye disease (TED) is officially underway.

Today, Horizon Pharma announced that the first patient has been enrolled in its clinical trial, “Treatment of Graves’ Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study (OPTIC)”.

TED, also known as Graves’ eye disease or Graves’ orbitopathy, is an autoimmune disorder in which the tissue behind the eye becomes inflamed leading to the eyes being pushed forward (proptosis). These persons typically have an appearance of having bulging or popping eyes.

Teprotumumab is a monoclonal antibody targeting insulin growth factor 1 receptor (IGF-1R). The drug has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA). 

The Phase 3 study will enroll a total of 76 patients at 11 treatment centers in United States, Germany and Italy. The primary endpoint will be the percentage of patients who have a proptosis responder rate of ≥ 2 mm reduction of proptosis in the study eye at the end of the study. Patients will be randomized to receive 8 infusions of teprotumumab or placebo every three weeks for 21 weeks. 

Inclusion criteria for the study includes:
  • Clinical diagnosis of Graves' disease associated with moderate-to-severe active TED.
  • Onset of active TED symptoms within 9 months prior to Baseline.
  • Participants must be euthyroid with the baseline disease under control or have mild hypo- or hyperthyroidism.
  • Does not require immediate surgical ophthalmological intervention.
  • Between the ages of 18 and 75 years
  • Alanine aminotransferase (ALT) or AST ≤ 3 times the upper limit of normal (ULN) or serum creatine <1.5 times the ULN 
  • Diabetic participants must have well-controlled stable disease 
  • Women of childbearing potential must have a negative serum pregnancy test at Screening and negative urine pregnancy tests at all protocol-specified timepoint
  • Male participants must be surgically sterile or must agree to use barrier contraceptive method during the study

The current Phase 3 study follows the positive results of the Phase 2 study, published in the New England Journal of Medicine earlier this year. In that study, teprotumumab met its primary endpoint (a reduction of 2 points or more in the Clinical Activity Score and a reduction of 2 mm or more in proptosis at week 24) with 29 of 42 patients given the drug having a response to treatment compared to 9 of 45 patients in the placebo group.

Per Horizon Pharma: “People living with TED experience debilitating, life-changing symptoms, including swelling of the eyelids, sensitivity to light, double vision and eye bulging,” said Raymond Douglas, M.D., Ph.D., one of the study’s principal investigators and director of the orbital and thyroid eye disease program, Cedars-Sinai Medical Center.  “Based on positive results from the Phase 2 study evaluating teprotumumab, we are excited to participate in the OPTIC study and move closer to potentially bringing forward the first treatment option for moderate-to-severe active TED.”

Image courtesy of Wikimedia Commons.

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