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Not yet recruiting NCT07476625

Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome

No phase Interventional Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Thalidomide (50mg), Colchicine.
Who it may be relevant to
Registry conditions: Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome. Basic parameters: 3 years — 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Efficacy and Safety of Thalidomide in Treating Pediatric PFAPA Syndrome: A Multicenter Randomized Controlled Trial

Overview

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.

Interventions

  • Drug Thalidomide (50mg)
    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).
  • Drug Colchicine
    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).

Primary outcome measures

  • Proportion of Participants Achieving Complete Remission at 6 Months [Time frame: 6 months]
Secondary outcome measures (9)
  • Complete Remission Rate at Multiple Time Points [Time frame: 3 months]
  • Complete Remission Rate at Multiple Time Points [Time frame: 9 months]
  • Complete Remission Rate at Multiple Time Points [Time frame: 12 months]
  • Partial Remission Rate [Time frame: 3 months]
  • Recurrence Rate Post-discontinuation [Time frame: 6 months post-treatment]
  • Recurrence Rate Post-discontinuation [Time frame: 12 months post-treatment]
  • Change in Growth Parameters (Z-scores) [Time frame: 6 months]
  • Change in Growth Parameters (Z-scores) [Time frame: 12 months]
  • Change in Inflammatory Markers [Time frame: 6 months]

Eligibility criteria

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

1.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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07476625 · SAHoWMU-CR2026-06-102

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗