Expanded Access Protocol of Teprotumumab (HZN-001) for Patients With Active Thyroid Eye Disease

NCT04040894 | Unknown

• 1 other identifier: HZNP-TEP-401
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 8

Brief Summary

This is an expanded access protocol intended to provide access to teprotumumab for the treatment of up to 60 patients in the United States with active moderate to severe thyroid eye disease where there is no comparable or satisfactory alternative therapy for treatment.

Conditions

Thyroid Eye Disease; Graves' Orbitopathy

Keywords

Proptosis; Human monoclonal antibody; insulin-like growth factor-1 receptor; Thyroid-Associated Ophthalmopathy; Hyperthyroidism; Autoimmune Thyroid Disease

Interventions

Teprotumumab BIOLOGICAL

Treatment with 8 infusions of Teprotumumab q3W for a total of 21 weeks. Teprotumumab 10 mg/kg will be administered on Day 1 and Teprotumumab 20 mg/kg will be administered q3W for the remaining 7 infusions.

Also known as: HZN-001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent.
• Male or female patient at least 18 years old.
• Clinical diagnosis active TED with a CAS ≥ 4 (on the 7-item scale).
• Moderate-to-severe active TED (not sight-threatening but has an appreciable impact on daily life), usually associated with one or more of the following: lid retraction ≥ 2 mm, moderate or severe soft tissue involvement, exophthalmos ≥ 3 mm above normal for race and gender, and/or inconstant or constant diplopia.
• Onset of Active TED symptoms (as determined by patient records) within approximately 12 months prior to eligibility review.
• Patients must be euthyroid with the baseline disease under control or have mild hypo- or hyperthyroidism (defined as free thyroxine \[FT4\] and free triiodothyronine \[FT3\] levels \< 50% above or below the normal limits) at Eligibility review. Every effort should be made to correct the mild hypo- or hyperthyroidism promptly and to maintain the euthyroid state for the full duration of participation.
• Does not require immediate surgical ophthalmological intervention.
• Diabetic patients must have well-controlled stable disease (defined as HbA1c \< 9.0%).
• Women of childbearing potential (including those with an onset of menopause \<2 years, non-therapy-induced amenorrhea for \<12 months, or not surgically sterile \[absence of ovaries and/or uterus\]) must have a negative serum pregnancy test within 3 weeks prior to the first infusion and negative urine pregnancy tests at all protocol-specified timepoints (i.e., prior to each infusion); patients who are sexually active with a non-vasectomized male partner must agree to use 2 reliable forms of contraception during the expanded access program, one of which is recommended to be hormonal, such as an oral contraceptive. Hormonal contraception must be started at least one full cycle prior to the first infusion and continue for 180 days after the last dose of investigational drug. Highly effective contraceptive methods (with a failure rate less than 1% per year), when used consistently and correctly, includes implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner.
• Male patients must be surgically sterile or, if sexually active with a female partner of childbearing potential, must agree to use a barrier contraceptive method from the first infusion through 180 days after the last dose of investigational drug.
• Patient is willing and able to comply with the prescribed treatment protocol and evaluations for the duration of their participation.

You may not qualify if:

• Any treatment with rituximab (Rituxan® or MabThera®) within 12 months prior to the first infusion of teprotumumab or tocilizumab (Actemra® or Roactemra®) within 6 months prior to the first infusion of teprotumumab. Use of any other non-steroid immunosuppressive agent within 3 months prior to the first infusion of teprotumumab.
• Use of an investigational agent for any condition within 60 days prior to the first infusion or anticipated use during the course of the protocol.
• Pregnant or lactating women.
• Biopsy-proven or clinically suspected IBD (e.g., diarrhea with or without blood or rectal bleeding associated with abdominal pain or cramping/colic, urgency, tenesmus, or incontinence for more than 4 weeks without a confirmed alternative diagnosis OR endoscopic or radiologic evidence of enteritis/colitis without a confirmed alternative diagnosis).
• Known hypersensitivity to any of the components of teprotumumab or prior hypersensitivity reactions to mAbs.
• Previous enrollment in this study or participation in a prior teprotumumab clinical study.
• Human immunodeficiency virus, hepatitis C or hepatitis B infections.

Sponsors & Collaborators

• Amgen: lead

Study Sites (8)

MACRO Trials

Beverly Hills, California, 90211, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

NorthShore University HealthSystem

Skokie, Illinois, 60076, United States

Location

Institute of Ophthalmology and Visual Science, Rutgers University New Jersey Medical School

Newark, New Jersey, 07103, United States

Location

Eye and Facial Plastic Surgery Consultants

Plymouth Meeting, Pennsylvania, 19462, United States

Location

Prisma Health Upstate/Endocrinology Specialists and Thyroid Center

Greenville, South Carolina, 29605, United States

Location

University of Tennessee Medical Center

Knoxville, Tennessee, 37920, United States

Location

TN Oculoplastics

Nashville, Tennessee, 37203, United States

Location

Related Links

• Related Info

MeSH Terms

Conditions

Graves Ophthalmopathy; Exophthalmos; Hyperthyroidism

Interventions

teprotumumab

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Graves Disease; Orbital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Goiter; Thyroid Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2019
• First Posted: August 1, 2019
• Last Updated: June 20, 2024

Record last verified: 2024-06

Locations

US (8)