Phase 2
Completed N=7
Study of RVT-1401 for the Treatment of Patients With Moderate to Severe Active Graves' Ophthalmopathy (GO)
Source: ClinicalTrials.gov NCT03922321 ↗Enrolled (actual)
7
Serious AEs
0.0%
Results posted
Jan 2022
Primary outcomePrimary: Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death During the 6-week Treatment Period — 7; 0; 6; 0 Participants
Summary
The purpose of this study was to evaluate safety, tolerability, and pharmacodynamic parameters of RVT-1401 in graves' ophthalmopathy (GO) patients.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Serious AE (SAE), Treatment-related Adverse Event (AE), and Death During the 6-week Treatment Period |
7; 0; 6; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Findings Related to Vital Signs |
— | — |
| PRIMARY Number of Participants With a Change From Normal Physical Examination Findings at Baseline to Abnormal Physical Examination Findings at the End of the Study |
— | — |
| PRIMARY Number of Participants With Clinically Significant Findings Related to Electrocardiograms (ECGs) |
— | — |
| PRIMARY Percent Change From Baseline in Total Immunoglobulin G (IgG), IgG1, IgG2, IgG3, and IgG4 Levels |
-65.00; -64.83; -67.16; -56.91; -70.05; -61.28 | — |
| PRIMARY Mean Change From Baseline in Levels of Anti-thyroid-stimulating Hormone Receptor (Anti-TSHR) Antibodies at Week 7 |
19.72; -10.59 | — |
| SECONDARY Mean Change From Baseline in Proptosis in the Study Eye and Non-study Eye at Week 7 |
23.1; -1.25; 21.9; -1.25 | — |
| SECONDARY Number of Participants With an Overall Proptosis Response |
3 | — |
| SECONDARY Area Under the Concentration-time Curve From Time 0 to 168 Hours (AUC0-168h) of RVT-1401 |
— | — |
| SECONDARY Maximum Concentration (Cmax) of RVT-1401 |
— | — |
| SECONDARY Serum Concentration at the End of the Dosing Interval (Ctrough) of RVT-1401 |
4.018; 12.075; 1.284; 0.167; 0.660; 0.077 | — |
| SECONDARY Number of Participants With Anti-RVT-1401 Antibody and Confirmed Anti-RVT-1401 Antibody at Week 7 |
5; 2; 2 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female ≥ 18 years of age.
- Clinical diagnosis of Graves' disease with hyperthyroidism associated with active, moderate to severe GO with a Clinical Activity Score (CAS) ≥ 4 for the most severely affected eye at Screening (on the 7-item scale) and Baseline (on the 10-item scale).
- Onset of active GO within 9 months of screening.
- Moderate-to-severe active GO (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, proptosis ≥ 3 mm above normal for race and gender, and/or inconstant or constant diplopia.
- Other, more specific inclusion criteria are defined in the protocol
Exclusion Criteria
- Use of any steroid (intravenous [IV] or oral) with a cumulative dose equivalent to ≥ 1 g of methylprednisolone for the treatment of GO within 3 weeks prior to Screening.
- Use of rituximab, tocilizumab, or any monoclonal antibody for immunomodulation within the past 9 months prior to Baseline.
- Total IgG level < 6g/L at Screening.
- Absolute neutrophil count <1500 cells/mm3 at Screening.
- Participants with decreased best corrected visual acuity due to optic neuropathy as defined by a decrease in vision of 2 lines on the Snellen chart, new visual field defect, or color defect secondary to optic nerve involvement within the last 6 months at Screening.
- Previous orbital irradiation or surgery for GO.
- Other, more specific exclusion criteria are defined in the protocol
Data sourced from ClinicalTrials.gov (NCT03922321). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.