Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome

The goal of this clinical trial is to evaluate the efficacy and safety of thalidomide in the treatment of children with Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis (PFAPA) syndrome. The study focuses on children diagnosed with PFAPA syndrome. The main questions it aims to answer are: Can thalidomide significantly reduce the frequency of febrile episodes in children with PFAPA syndrome? What is the safety profile and tolerability of thalidomide in this pediatric population? Researchers will compare the thalidomide group to a colchicine group to see if thalidomide is more effective in controlling recurrent fever and associated symptoms. Participants will: Take the assigned medication (thalidomide or colchicine) daily for a duration of 6 months. Attend follow-up visits every 4 weeks at the clinic. Maintain a diary to record the frequency of fever episodes and any other clinical symptoms. Undergo safety assessments and physical examinations during each scheduled visit.

NCT Number	NCT07476625
Lead Sponsor	Wenjie Zheng Collaborators: Nanjing Children's Hospital, Children's Hospital of Soochow University, The First Affiliated Hospital of Xiamen University, Tianjin Children's Hospital, Ningbo Women & Children's Hospital, Children's Hospital Affiliated to Shandong University, Jiangxi Province Children's Hospital
Conditions	Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome
Enrollment	106 participants
Start Date	2026-04-01
Primary Completion	2027-03-31 (estimated)
Study Completion	2028-03-30 (estimated)
Updated on ClinicalTrials.gov	2026-03-17