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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04040894
Expanded Access Status : Available
First Posted : August 1, 2019
Last Update Posted : October 9, 2019
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

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.

Condition or disease Intervention/treatment
Thyroid Eye Disease Graves' Orbitopathy Biological: Teprotumumab

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.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: Phase 3b, Multicenter, Open-label, Single-Arm Expanded Access Protocol of TEPROTUMUMAB (HZN-001)

Resource links provided by the National Library of Medicine

Intervention Details:
  • Biological: Teprotumumab
    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.
    Other Name: HZN-001

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. Written informed consent.
  2. Male or female patient at least 18 years old.
  3. Clinical diagnosis active TED with a CAS ≥ 4 (on the 7-item scale).
  4. 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.
  5. Onset of Active TED symptoms (as determined by patient records) within approximately 12 months prior to eligibility review.
  6. 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.
  7. Does not require immediate surgical ophthalmological intervention.
  8. Diabetic patients must have well-controlled stable disease (defined as HbA1c < 9.0%).
  9. 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.
  10. 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.
  11. Patient is willing and able to comply with the prescribed treatment protocol and evaluations for the duration of their participation.

Exclusion Criteria:

  1. 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.
  2. 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.
  3. Pregnant or lactating women.
  4. 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).
  5. Known hypersensitivity to any of the components of teprotumumab or prior hypersensitivity reactions to mAbs.
  6. Any other condition that, in the opinion of the Sponsor, would preclude inclusion in the expanded access program.
  7. Previous enrollment in this study or participation in a prior teprotumumab clinical study.
  8. Human immunodeficiency virus, hepatitis C or hepatitis B infections.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04040894

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Contact: Horizon Medical Information 1-866-479-6742

Sponsors and Collaborators
Horizon Pharma USA, Inc.

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Responsible Party: Horizon Pharma USA, Inc. Identifier: NCT04040894     History of Changes
Other Study ID Numbers: HZNP-TEP-401
First Posted: August 1, 2019    Key Record Dates
Last Update Posted: October 9, 2019
Last Verified: October 2019
Keywords provided by Horizon Pharma USA, Inc.:
Human monoclonal antibody
insulin-like growth factor-1 receptor
Thyroid-Associated Ophthalmopathy
Autoimmune Thyroid Disease
Additional relevant MeSH terms:
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Graves Ophthalmopathy
Thyroid Diseases
Eye Diseases
Endocrine System Diseases
Eye Diseases, Hereditary
Graves Disease
Orbital Diseases
Genetic Diseases, Inborn
Autoimmune Diseases
Immune System Diseases
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs