Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome (NCT07476625) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome
106 participantsStarted 2026-04-01
Plain-language summary
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.
Who can participate
Age range
3 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Meet the 2019 Eurofever or 2020 CARRA diagnostic criteria for PFAPA syndrome.
. Aged 3 to 18 years (inclusive) at the time of screening.
. Have experienced at least 3 febrile episodes within the past six months.
. History of responsiveness to glucocorticoid treatment during at least 3 previous episodes, but with continued recurrence. (Responsiveness is defined as normalization of body temperature within 24 hours after a maximum dose of 2 mg/kg \[up to 60 mg\] administered as a single or two divided doses).
Exclusion criteria
.Diagnosis of monogenic or other polygenic periodic fever syndromes. 2.Presence of immunodeficiency or neoplastic diseases. 3.Active bacterial, fungal, or viral infection during the screening period. 4.Prior treatment with immunosuppressive agents. 5.Prior use of thalidomide or colchicine. 6.Laboratory parameters at screening that meet any of the following (based on the most recent test result at the study hospital prior to the first dose):
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Proportion of Participants Achieving Complete Remission at 6 Months