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


Study First Submitted Date 2019-07-30
Study First Posted Date 2019-08-01
Last Update Posted Date 2020-03-16
Verification Month Year March 2020
Verification Date 2020-03-31
Last Update Posted Date 2020-03-16

Detailed Descriptions

Sequence: 20696487
Description Requests for access to the Sponsor must be made by a licensed physician for a specific patient, based on a determination with the patient that the benefits of treatment with the investigational drug outweigh the risks. Licensed physician’s with eligible patients must apply and meet requirements for participation in the expanded access program.


Sequence: 199781514 Sequence: 199781515 Sequence: 199781516 Sequence: 199781517 Sequence: 199781518 Sequence: 199781519 Sequence: 199781520 Sequence: 199781521
Name MACRO Trials Name Bascom Palmer Eye Institute Name NorthShore University HealthSystem Name Institute of Ophthalmology and Visual Science, Rutgers University New Jersey Medical School Name Eye and Facial Plastic Surgery Consultants Name Prisma Health Upstate/Endocrinology Specialists and Thyroid Center Name University of Tennessee Medical Center Name TN Oculoplastics
City Beverly Hills City Miami City Skokie City Newark City Plymouth Meeting City Greenville City Knoxville City Nashville
State California State Florida State Illinois State New Jersey State Pennsylvania State South Carolina State Tennessee State Tennessee
Zip 90211 Zip 33136 Zip 60076 Zip 07103 Zip 19462 Zip 29605 Zip 37920 Zip 37203
Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States


Sequence: 52103263 Sequence: 52103264
Name Thyroid Eye Disease Name Graves’ Orbitopathy
Downcase Name thyroid eye disease Downcase Name graves’ orbitopathy

Id Information

Sequence: 40105428
Id Source org_study_id
Id Value HZNP-TEP-401


Sequence: 42507907
Name United States
Removed False


Sequence: 52417309
Intervention Type Biological
Name Teprotumumab
Description 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.


Sequence: 79760096 Sequence: 79760097 Sequence: 79760098 Sequence: 79760099 Sequence: 79760100 Sequence: 79760101
Name Proptosis Name Human monoclonal antibody Name insulin-like growth factor-1 receptor Name Thyroid-Associated Ophthalmopathy Name Hyperthyroidism Name Autoimmune Thyroid Disease
Downcase Name proptosis Downcase Name human monoclonal antibody Downcase Name insulin-like growth factor-1 receptor Downcase Name thyroid-associated ophthalmopathy Downcase Name hyperthyroidism Downcase Name autoimmune thyroid disease

Browse Conditions

Sequence: 193220141 Sequence: 193220142 Sequence: 193220143 Sequence: 193220144 Sequence: 193220145 Sequence: 193220146 Sequence: 193220147 Sequence: 193220148 Sequence: 193220149 Sequence: 193220150 Sequence: 193220151 Sequence: 193220152 Sequence: 193220153
Mesh Term Eye Diseases Mesh Term Graves Ophthalmopathy Mesh Term Thyroid Diseases Mesh Term Endocrine System Diseases Mesh Term Eye Diseases, Hereditary Mesh Term Graves Disease Mesh Term Exophthalmos Mesh Term Orbital Diseases Mesh Term Genetic Diseases, Inborn Mesh Term Goiter Mesh Term Hyperthyroidism Mesh Term Autoimmune Diseases Mesh Term Immune System Diseases
Downcase Mesh Term eye diseases Downcase Mesh Term graves ophthalmopathy Downcase Mesh Term thyroid diseases Downcase Mesh Term endocrine system diseases Downcase Mesh Term eye diseases, hereditary Downcase Mesh Term graves disease Downcase Mesh Term exophthalmos Downcase Mesh Term orbital diseases Downcase Mesh Term genetic diseases, inborn Downcase Mesh Term goiter Downcase Mesh Term hyperthyroidism Downcase Mesh Term autoimmune diseases Downcase Mesh Term immune system diseases
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48257039
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Horizon Pharma USA, Inc.


Sequence: 30726536
Gender All
Minimum Age 18 Years
Maximum Age N/A
Criteria Inclusion Criteria:

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.

Exclusion Criteria:

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.
Any other condition that, in the opinion of the Sponsor, would preclude inclusion in the expanded access program.
Previous enrollment in this study or participation in a prior teprotumumab clinical study.
Human immunodeficiency virus, hepatitis C or hepatitis B infections.

Adult True
Child False
Older Adult True

Calculated Values

Sequence: 253977646
Number Of Facilities 8
Registered In Calendar Year 2019
Were Results Reported False
Has Us Facility True
Has Single Facility False
Minimum Age Num 18
Minimum Age Unit Years

Intervention Other Names

Sequence: 26635041
Intervention Id 52417309
Name HZN-001


Sequence: 4381909
Url http://tepezza.com
Description Related Info

Responsible Parties

Sequence: 28839391
Responsible Party Type Sponsor