Teprotumumab (RV 001) Treatment in Patients With Active Thyroid Eye Disease

Amgen

Status and phase: Completed; Phase 2

Conditions: Thyroid-Associated Ophthalmopathy; Thyroid Associated Ophthalmopathies

Treatments: Drug: normal saline; Drug: teprotumumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT01868997

TED01RV

FD004792-01A1 (Other Identifier)

2014-000113-31 (EudraCT Number)

Details and patient eligibility

About

The primary objective of this study is to investigate the efficacy, safety, and tolerability of RV 001 (teprotumumab), a fully human anti-IGF1R antibody, administered q3W for 6 months, in comparison to placebo, in the treatment of participants suffering from active TED.

"Funding Source - FDA OOPD"

Full description

Study with completed results acquired from Horizon in 2024.

Enrollment: 88 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of Graves' disease associated with active TED and a clinical activity score of ≥ 4
• Fewer than 9 months from onset of TED
• No previous medical or surgical treatment, excluding local supportive measures and oral steroids if the maximum cumulative dose is less than 1000 mg methylprednisolone or equivalent with at least 6 weeks between last administration of oral steroids and randomization
• Euthyroid or with mild hypo or hyperthyroidism defined as free thyroxine (FT4) and free triiodothyronine (FT3) levels less than 50% above or below the normal limits (every effort should be made to correct the mild hypo- or hyperthyroidism promptly)

Exclusion criteria

• Optic neuropathy
• Corneal decompensation unresponsive to medical management
• Oral or IV steroid treatment for any non-TED reason in the preceding 3 months
• Poorly controlled diabetes
• Platelets < 100 x 10^9/L
• Hemoglobin concentration > 2 g/dL below the lower limit of normal

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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88 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

A placebo infusion (normal saline) administered q3W by IV infusion over a period of 24 weeks for a total of 8 infusions.

Treatment:

Drug: normal saline

Teprotumumab

Experimental group

Description:

Teprotumumab administered q3W by IV infusion over a period of 24 weeks for a total of 8 infusions. All participants start treatment at a dose of 10 mg/kg. At Week 3, the dose is escalated to 20 mg/kg and kept constant for the remainder of the study.

Treatment:

Drug: teprotumumab