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

NCT ID: NCT01868997

Last Updated: 2024-12-17

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

88 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-07-31

Study Completion Date

2017-02-22

Brief Summary

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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"

Detailed Description

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Study with completed results acquired from Horizon in 2024.

Conditions

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Thyroid Associated Ophthalmopathies Thyroid-Associated Ophthalmopathy

Keywords

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Thyroid Eye Disease, Graves Orbitopathy, Thyroid Associated Ophthalmopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

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

Group Type PLACEBO_COMPARATOR

normal saline

Intervention Type DRUG

Teprotumumab

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.

Group Type EXPERIMENTAL

teprotumumab

Intervention Type DRUG

Interventions

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teprotumumab

Intervention Type DRUG

normal saline

Intervention Type DRUG

Other Intervention Names

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RV 001 HZN-001

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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Jules Stein Eye Institute at UCLA

Los Angeles, California, United States

Site Status

University of Denver

Aurora, Colorado, United States

Site Status

Emory University Department of Ophthalmology

Atlanta, Georgia, United States

Site Status

University of Iowa Hospitals and Clinics, Department of Ophthalmology

Iowa City, Iowa, United States

Site Status

Kellogg Eye Center at University of Michigan

Ann Arbor, Michigan, United States

Site Status

Washington University Department of Ophthalmology

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center Department of Ophthalmology

Omaha, Nebraska, United States

Site Status

Casey Eye Institute at Oregon Health and Science University

Portland, Oregon, United States

Site Status

Hamilton Eye Institute at University of Tennessee

Memphis, Tennessee, United States

Site Status

Eye Wellness Center

Houston, Texas, United States

Site Status

Medical College of Wisconsin, The Eye Institute

Milwaukee, Wisconsin, United States

Site Status

Johannes Gutenberg University Medical Center

Mainz, , Germany

Site Status

Fondazione Ca' Granda Ospedale Policlinico Graves GO Center

Milan, , Italy

Site Status

University of Pisa, Azienda Ospedaliera

Pisa, , Italy

Site Status

Moorfields Eye Hospital

London, , United Kingdom

Site Status

Countries

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United States Germany Italy United Kingdom

References

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Smith TJ, Kahaly GJ, Ezra DG, Fleming JC, Dailey RA, Tang RA, Harris GJ, Antonelli A, Salvi M, Goldberg RA, Gigantelli JW, Couch SM, Shriver EM, Hayek BR, Hink EM, Woodward RM, Gabriel K, Magni G, Douglas RS. Teprotumumab for Thyroid-Associated Ophthalmopathy. N Engl J Med. 2017 May 4;376(18):1748-1761. doi: 10.1056/NEJMoa1614949.

Reference Type RESULT
PMID: 28467880 (View on PubMed)

Xin Y, Xu F, Gao Y, Bhatt N, Chamberlain J, Sile S, Hammel S, Holt RJ, Ramanathan S. Pharmacokinetics and Exposure-Response Relationship of Teprotumumab, an Insulin-Like Growth Factor-1 Receptor-Blocking Antibody, in Thyroid Eye Disease. Clin Pharmacokinet. 2021 Aug;60(8):1029-1040. doi: 10.1007/s40262-021-01003-3. Epub 2021 Mar 26.

Reference Type DERIVED
PMID: 33768488 (View on PubMed)

Wang Y, Smith TJ. Current concepts in the molecular pathogenesis of thyroid-associated ophthalmopathy. Invest Ophthalmol Vis Sci. 2014 Mar 20;55(3):1735-48. doi: 10.1167/iovs.14-14002.

Reference Type DERIVED
PMID: 24651704 (View on PubMed)

Other Identifiers

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FD004792-01A1

Identifier Type: OTHER

Identifier Source: secondary_id

2014-000113-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TED01RV

Identifier Type: -

Identifier Source: org_study_id