Phase 2 N=87 Randomized Quadruple-blind Treatment

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

Thyroid Associated Ophthalmopathies · Thyroid-Associated Ophthalmopathy

Bottom Line

View on ClinicalTrials.gov: NCT01868997 ↗

Enrolled (actual)

Serious AEs

6.9%

Results posted

Aug 2017

Primary outcome: Primary: Responder Status at Week 24 — 9; 29; 36; 13 Participants — p=< 0.001

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: teprotumumab (Drug); normal saline (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Amgen
Primary completion: Mar 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Responder Status at Week 24	9; 29; 36; 13	< 0.001 sig
SECONDARY Overall Average Change From Baseline in Graves' Ophthalmopathy Quality of Life (GO-QOL) Scale - Overall to Week 24 (Mixed-Model Repeated Measures [MMRM])	6.77; 17.74	0.001 sig
SECONDARY Overall Average Change From Baseline in Proptosis of the Study Eye to Week 24 (MMRM)	-0.15; -2.46	< 0.001 sig
SECONDARY Overall Average Change From Baseline in CAS to Week 24 (MMRM)	-1.85; -3.43	< 0.001 sig
SECONDARY Overall Average Change From Baseline in GO-QOL Scale - Visual Functioning to Week 24 (MMRM)	7.51; 21.67	< 0.001 sig
SECONDARY Overall Average Change From Baseline in GO-QOL Scale - Appearance to Week 24 (MMRM)	6.60; 12.92	0.101

Summary

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"

Eligibility Criteria

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 2 g/dL below the lower limit of normal

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01868997). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.