Health TrialsbyHealthPanel

Sirolimus vs Corticosteroids in Treatment of Thyroid Eye Disease

ConditionThyroid-Associated Ophthalmopathy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-80
SponsorHaukeland University Hospital

About this trial

The purpose of this study is to determine whether Sirolimus is more effective and burdened with less side effects than conventional treatment with corticosteroids in patients with active thyroid eye disease.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

The participant want treatment for active thyroid eye disease and is willing to be included in the study

Clinical diagnosis of Graves' disease associated with active TED with a Clinical Activity Score (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 participant records) within 9 months prior to inculsion

Disqualifiers

The participant dont want treatment for active thyroid eye disease or dont want to participate in the study

Decreased vision due to optic neuropathy as defined by a significant decrease in best corrected visual acuity, new visual field defect, or color defect secondary to optic nerve involvement within the last 6 months

Corneal decompensation unresponsive to medical management

Previous orbital irradiation or surgery for TED Any steroid use (intravenous [IV] or oral) with a cumulative dose equivalent to ≥ 1 g of methylprednisolone for the treatment of TED. Previous steroid use (IV or oral) with a cumulative dose of <1 g methylprednisolone or equivalent for the treatment of TED and previous use of steroid eye drops is allowed if the corticosteroid was discontinued at least 4 weeks prior to inclusion

Trial design

Design model

Parallel

Treatments tested in this trial

  • Sirolimus 1 mg Oral Tablet

    Drug

    2 mg Sirolimus (two 1 mg tablets) on the first day, followed by 0,5 mg Sirolimus (half 1 mg tablet) per day for 12 weeks.

  • Methylprednisolone

    Drug

    500 mg Methylprednisolone intravenously once a week for 6 weeks, followed by 250 mg once a week for 6 weeks. Total period of treatment 12 weeks.

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: SirolimusActive comparator 1 intervention
Group B: CorticosteroidsActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

Percentage of participants who were CAS categorical responders at week 12

CAS categorical responders were defined as participants with a reduction of ≥ 2 CAS points. The CAS is a 7-item description of clinical activity, including: 1. Spontaneous orbital pain; 2. Gaze evoked orbital pain; 3. Eyelid swelling that is considered to be due to active (inflammatory phase) thyroid eye disease/ Graves' Ophthalmopathy or Orbitopathy (TED/GO); 4. Eyelid erythema; 5. Conjunctival redness that is considered to be due to active (inflammatory phase) TED/GO (ignore "equivocal" redness); 6. Chemosis; 7. Inflammation of caruncle or plica. Each item is scored (1=present; 0=absent) and scores for each item are summed for total score of 0 (no inflammatory symptoms) to 7 (most inflammatory symptoms).

Time frame
Week 12

Secondary outcomes

1

Percentage of participants who were proptosis responders at week 12

Proptosis responders were defined as percentage of participants with ≥ 2 mm reduction from baseline in proptosis in one eye at week 12.

Time frame
Week 12
2

Percentage of participants who were eyelid retraction responders at week 12

Eyelid retraction responders were defined as percentage of participants with ≥ 3 mm reduction from baseline in vertical lid aperture in one eye at week 12

Time frame
Week 12
3

Percentage of participants who were diplopia responders at week 12

Diplopia responders were defined as percentage of participants with ≥ 1 class improvement of eye motility from baseline assessed by Gorman score. Gorman score: 1 = no diplopia, 2 =intermittent diplopia, 3 = inconstant (gaze-evoked) diplopia, 4 = constant diplopia in primary or reading position.

Time frame
Baseline, up to Week 12

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Haukeland University Hospital

Lead sponsor

Source ClinicalTrials.gov
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