ClinicalTrials.gov record NCT07476625

Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Age3 years old to 18 years old
SexAll
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This randomized, open-label trial compares daily thalidomide with daily colchicine in children with PFAPA. Participants take the assigned drug, keep a diary of fever episodes, and attend clinic visits with safety checks every 4 weeks. The main outcome is complete remission (no fever attacks) at 6 months.

What it is studying. Whether thalidomide, compared with colchicine, affects the frequency of febrile episodes and what its safety and tolerability are in children with PFAPA.

What the researchers hope to learn. Measure the proportion of participants with complete remission (zero febrile episodes) at 6 months; compare remission rates at additional time points; assess relapse after stopping treatment; evaluate changes in growth measures and inflammatory markers; and document safety and tolerability.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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 being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Thalidomide (50mg)
  • Colchicine

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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):
  • 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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned to take either oral thalidomide or oral colchicine daily (treatment and dose adjustments are specified in the study) for up to 12 months. Families keep a diary of fever episodes and other symptoms and attend clinic visits about every 4 weeks for physical exams and safety assessments. Blood tests and growth measurements will be done to monitor safety and inflammation. Some participants are followed after stopping the study drug to check for recurrence of symptoms at specified times after treatment ends.

How the study is structured. Children with PFAPA are randomly assigned to one of two parallel groups and receive daily oral thalidomide or daily oral colchicine. The trial is not masked (open-label). The registry lists treatment duration inconsistently (a 6-month primary outcome is specified while the arm descriptions state 12 months of treatment); participants keep fever diaries and have clinic visits approximately every 4 weeks for safety assessments.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome. Does it apply to my diagnosis?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on familial Mediterranean fever

Clinical trials62 trials listed for familial Mediterranean feverEvery trial the registry lists for familial Mediterranean fever, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in familial Mediterranean fever researchLatest monthly update: September 2026. Includes: Colchicine form change. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 1, 2026 (estimated)
Primary completion
Mar 31, 2027 (estimated)
Study completion
Mar 30, 2028 (estimated)
Target enrollment
106 participants (estimated)
Last updated on registry
Mar 17, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

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
  • Jinan children's hospital
  • Jiangxi Province Children's Hospital

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
glucocorticoid
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Prior treatment
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
immunosuppressive
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Upper Limit of Normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Glomerular Filtration Rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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