Real World Study to Describe the Effectiveness and Safety of Teprotumumab Among Thyroid Eye Disease Patients
- Registration Number
- NCT07085117
- Lead Sponsor
- Amgen
- Brief Summary
The main objective of this study is to assess the number of participants with proptosis response as of last intravenous infusion of teprotumumab.
- Detailed Description
A retrospective cohort will be followed retrospectively for endpoints at the only facility providing treatment of teprotumumab in mainland China. Eligible participants' data were collected retrospectively over a period of approximately 3 years. The target population is patients with Thyroid Eye Disease (TED) that have received teprotumumab treatment in BOAO Pilot Zone. While the primary objective is to assess the proportion of patients with proptosis response as of the last intravenous infusion of teprotumumab, the secondary objectives are to describe baseline characteristics of patients at teprotumumab initiation, and the teprotumumab use at follow-up, to describe real-world effectiveness of clinical outcomes of interest, to describe patient-reported outcomes and to describe real-world safety of teprotumumab.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 5
- Receipt of at least 1 infusion of teprotumumab for treatment of the TED.
- Obtained study specific informed consent form (ICF), if required.
- Documentation of non-Chinese ethnicity.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Teprotumumab Teprotumumab Participants with Thyroid Eye Disease (TED) that have received teprotumumab treatment in BOAO Pilot Zone.
- Primary Outcome Measures
Name Time Method Number of Participants with Proptosis Response as of the Last IV Infusion Teprotumumab From baseline up to approximately 3 years Proptosis response is defined as having a ≥ 2 mm reduction in proptosis measurement from baseline in the more severely affected eye without deterioration (≥ 2 mm increase) of proptosis in the fellow eye following the last infusion.
- Secondary Outcome Measures
Name Time Method Demographics of Participants at Teprotumumab Initiation From baseline up to approximately 3 years Demographics include age in years and sex (male, female, unknown).
Clinical Characteristics of Participants at Teprotumumab Initiation: Body Mass Index at Index Date From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: Smoking Status From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: Disease Duration From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: TED Activity From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: TED Severity From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: Eye symptoms From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: Clinical Activity Score From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: Computed Tomography (CT)/Magnetic Resonance Imaging (MRI) Measurements From baseline up to approximately 3 years Clinical Characteristics of Participants at Teprotumumab Initiation: Quality of Life From baseline up to approximately 3 years Number of Participants with Relevant Medical History at Teprotumumab Initiation From baseline up to approximately 3 years Medical history includes history of compressive optic neuropathy, hyperglycemia, inflammatory bowel disease (IBD), infusion reactions, and hearing impairment; therapeutic history for thyroid disease (Graves' disease or others) before teprotumumab administration; therapeutic history for TED before teprotumumab administration and complications if any; comorbidities; concomitant treatments; lab tests.
Teprotumumab Use From baseline up to approximately 3 years Teprotumumab use includes dose and dosing schedule, total number of infusions and duration, dose reduction and modification (including causes), treatment interruption and causes, time interval from interruption to re-initiation, and subsequent re-initiation treatment (if any), treatment discontinuation (including causes).
Proptosis Measurements of Participants From baseline up to approximately 3 years Proptosis measurements include all available proptosis measurements by eye for each participant and the change in proptosis measurement between baseline and the last infusion by eye.
Clinical Activity Scores (CAS) of Participants From baseline up to approximately 3 years CAS will include all available measures of CAS items by eye for each participant and the number of participants achieving CAS value of 0 or 1 as of the last infusion among patients with active TED at baseline.
Overall Response as of the Last Infusion Among Participants with Active TED From baseline up to approximately 3 years Overall response is defined as having ≥ 2-point reduction in CAS AND ≥ 2 mm reduction in proptosis from baseline in the most severely affected eye, provided there is no corresponding deterioration (≥ 2-point increase in CAS or ≥ 2 mm increase in proptosis) in the fellow eye following the last infusion.
Active TED is defined as CAS≥3 on a 7-point scale at baseline.Diplopia Measurements for Participants From baseline up to approximately 3 years Diplopia measurements will include all available measures of diplopia for each participant, the binocular diplopia response as of the last infusion defined as having baseline binocular diplopia grade \> 0 and a reduction of ≥ 1 grade between baseline and the last infusion, and complete diplopia response defined as having baseline binocular diplopia grade \> 0 and achieving grade of 0 as of the last infusion.
Quality of Life (QoL) as Reported by Participants From baseline up to approximately 3 years All available participant-reported QoL for each participant, overall and at subscale level, and the change in QoL between baseline and the last infusion, overall and at subscale level.
Incidence of Adverse Events (AEs) From baseline up to approximately 3 years The incidence of AEs between the administration of the first dose and 30 days after the last dose of teprotumumab, including:
* Any AE
* Specific AEs, inclusive of infusion-related reactions, hyperglycemia, hearing impairment, and new onset or exacerbation of IBD
* Serious adverse event (SAE)
* AE leading to treatment discontinuation
* AE leading to death.Status of Specific AEs up to 6 Months After Last Infusion of Teprotumumab From baseline up to approximately 3 years Specific AEs include infusion-related reactions, hyperglycemia, hearing impairment, and new onset or exacerbation of IBD.
Trial Locations
- Locations (1)
Boao Vanguard Hospital
🇨🇳Qionghai, Hainan, China
Boao Vanguard Hospital🇨🇳Qionghai, Hainan, China