Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome
1 other identifier
interventional
106
0 countries
N/A
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for not_applicable
Started Apr 2026
Typical duration for not_applicable
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 9, 2026
CompletedFirst Posted
Study publicly available on registry
March 17, 2026
CompletedStudy Start
First participant enrolled
April 1, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 30, 2028
March 17, 2026
March 1, 2026
12 months
March 9, 2026
March 12, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Proportion of Participants Achieving Complete Remission at 6 Months
Complete remission is defined as the total absence of febrile episodes (zero attacks) during the treatment period.
6 months
Secondary Outcomes (9)
Complete Remission Rate at Multiple Time Points
3 months
Complete Remission Rate at Multiple Time Points
9 months
Complete Remission Rate at Multiple Time Points
12 months
Partial Remission Rate
3 months
Recurrence Rate Post-discontinuation
6 months post-treatment
- +4 more secondary outcomes
Other Outcomes (5)
Incidence of Adverse Events
1 month
Incidence of Adverse Events
6 months
Incidence of Adverse Events
12 months
- +2 more other outcomes
Study Arms (2)
Thalidomide Group
EXPERIMENTALPatients in this group will receive oral thalidomide treatment for 12 months.
Colchicine Group
ACTIVE COMPARATORPatients in this group will receive oral colchicine treatment for 12 months.
Interventions
The starting dose of thalidomide is 1 mg/kg/day, administered orally before bedtime. If febrile episodes persist during treatment, the dosage will be increased starting the day after the next fever (maximum dose not to exceed 2 mg/kg/day, with a maximum total dose of 100 mg/day).
The starting dose of colchicine is 0.5 mg/day administered orally. If febrile episodes persist during treatment, the dosage will be increased starting the day after the next fever (maximum dose not to exceed 1.25 mg/day).
Eligibility Criteria
You may qualify if:
- 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).
You may not qualify if:
- 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):
- White Blood Cell (WBC) count \< 4 × 10⁹/L, Hemoglobin (HGB) \< 100 g/L, or Platelet (PLT) count \< 100 × 10⁹/L.
- Serum Alanine Aminotransferase (ALT) \> 2 times the Upper Limit of Normal (ULN).
- Glomerular Filtration Rate (GFR/CCR) \< 60 mL/min/1.73m².
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Wenjie Zhenglead
- Nanjing Children's Hospitalcollaborator
- Children's Hospital of Soochow Universitycollaborator
- The First Affiliated Hospital of Xiamen Universitycollaborator
- Tianjin Children's Hospitalcollaborator
- Ningbo Women & Children's Hospitalcollaborator
- Jinan children's hospitalcollaborator
- Jiangxi Children's Hospitalcollaborator
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Chief Physician
Study Record Dates
First Submitted
March 9, 2026
First Posted
March 17, 2026
Study Start
April 1, 2026
Primary Completion (Estimated)
March 31, 2027
Study Completion (Estimated)
March 30, 2028
Last Updated
March 17, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share
Data contains sensitive patient information and is restricted by institutional policy.