Rapamycin Plus Methylprednisolone Versus Methylprednisolone Alone in Active, Moderate-to-severe Graves' Orbitopathy.

• Status: Not yet recruiting
• Trial end date: 2023-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is a randomized, controlled, multi-center clinical study, the purpose is to evaluate the efficacy and safety of rapamycin combined with methylprednisolone in the treatment of moderate to severe active GO.

GO patients with moderate to severe activity were selected as the research objects, and the screening period was 1 week. Eligible and well-informed subjects were randomly assigned 1:1 to the experimental group (rapamycin + methylprednisolone) or the control group (methylprednisolone group). The control group was given methylprednisolone pulse 500 mg/time once a week for 6 weeks + 250 mg/time once a week for 6 weeks, and the experimental group was given rapamycin 2 mg/day orally for 24 weeks on the basis of methylprednisolone pulse therapy. The follow-up period was from 25th to 36th week.

Before treatment, 1 week after treatment, 6 weeks, 12 weeks, 24 weeks, and 36 weeks, visits were conducted to evaluate the improvement and safety evaluation of patients' GO eye activity, severity, and quality of life. At the end of the 1st week and the 12th week, the blood concentration of rapamycin was measured.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: First Affiliated Hospital Xi'an Jiaotong University
• Collaborators: Beijing Tongren Hospital; Cheloo Hospital (Qingdao), Shandong University; Jiangsu Province Hospital with Integration of Chinese and Western Medicine; Nanjing First Hospital, Nanjing Medical University; People's Hospital of Xinjiang Uygur Autonomous Region; Shunde Hospital, Southern Medical University; The Second Affiliated Hospital of Air Force Military Medical University; Zhejiang Provincial People's Hospital
• Treatments: Methylprednisolone; Methylprednisolone Acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Sirolimus

Criteria

Inclusion Criteria:

1)18-70 years old 2)Clinically diagnosed GO (a. Moderate-severe, b. Active stage)

1. Moderate-severe( CSS Grading Standards for EUGOGO 2021)

2. Active stage (CAS) ≥ 3

Exclusion Criteria:

1. Sight-threatening GO

2. There are obvious abnormalities in laboratory tests: liver damage: ALT and AST ≥ 3 times the upper limit of normal values; kidney damage: serum creatinine ≥ 1.5*ULN; blood routine: hemoglobin < 9g/dl, white blood cell count < 3000/µl or Platelets < 100,000/µl; blood lipids: total cholesterol > 300 mg/dl or triglycerides > 400 mg/dl after lipid-lowering therapy;

3. The patient developed an infection during treatment, requiring intravenous antibiotics, and did not show clinical improvement within 5 days;

4. The subject has a systemic allergic reaction to rapamycin or methylprednisolone;

5. The subject is pregnant during the test;

6. The patient requests to withdraw from the researcher;

7. Investigators believe that they cannot continue to participate in the experiment.