Thyroid Eye DiseaseEAPNCT04040894
Expanded Access Protocol of Teprotumumab (HZN-001) for Patients With Active Thyroid Eye Disease
A study testing Teprotumumab in people with Thyroid Eye Disease and Graves' Orbitopathy, run by Amgen at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thyroid Eye Disease, Graves' Orbitopathy
- What is being tested?
- Teprotumumab
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 8 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
MACRO Trials
Beverly Hills, California, 90211
Prisma Health Upstate/Endocrinology Specialists and Thyroid Center
Greenville, South Carolina, 29605
University of Tennessee Medical Center
Knoxville, Tennessee, 37920
Bascom Palmer Eye Institute
Miami, Florida, 33136
TN Oculoplastics
Nashville, Tennessee, 37203
Institute of Ophthalmology and Visual Science, Rutgers University New Jersey Medical School
Newark, New Jersey, 07103
Eye and Facial Plastic Surgery Consultants
Plymouth Meeting, Pennsylvania, 19462
NorthShore University HealthSystem
Skokie, Illinois, 60076
About this study
Official title: Phase 3b, Multicenter, Open-label, Single-Arm Expanded Access Protocol of TEPROTUMUMAB (HZN-001)
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.
Read the detailed 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.
Study acquired from Horizon in 2024.
- Conditions
- Thyroid Eye Disease, Graves' Orbitopathy
- Being tested
- Teprotumumab
- Keywords
- Proptosis, Human monoclonal antibody, insulin-like growth factor-1 receptor, Thyroid-Associated Ophthalmopathy, Hyperthyroidism, Autoimmune Thyroid Disease
- Medical subject headings
- Graves Ophthalmopathy, Exophthalmos, Hyperthyroidism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TeprotumumabBiological
Also called HZN-001
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thyroid Eye DiseaseStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04040894
- Sponsor's protocol ID
- HZNP-TEP-401
- Acronym
- EAP
- Status last verified
- Jun 2024
- First submitted
- 30 Jul 2019
- Last updated
- 20 Jun 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3b, Multicenter, Open-label, Single-Arm Expanded Access Protocol of TEPROTUMUMAB (HZN-001)
- ClinicalTrials.gov ID
- NCT04040894
- Acronym / sponsor ID
- EAP / HZNP-TEP-401
- Conditions
- Thyroid Eye Disease, Graves' Orbitopathy
- Keywords
- Proptosis, Human monoclonal antibody, insulin-like growth factor-1 receptor, Thyroid-Associated Ophthalmopathy, Hyperthyroidism, Autoimmune Thyroid Disease
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- First posted
- 1 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study acquired from Horizon in 2024.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TeprotumumabAlso: HZN-001Treatment 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. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jun 2024.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD study director
Amgen
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Teprotumumab in people with Thyroid Eye Disease and Graves' Orbitopathy, run by Amgen at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jun 2024.