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NCT Number: NCT07476625

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.

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Key information

Conditions

Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Autoinflammatory Diseases Infections Mouth Diseases Otorhinolaryngologic Diseases Pharyngeal Diseases Pharyngitis Respiratory Tract Diseases Respiratory Tract Infections Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases Stomatitis Stomatitis, Aphthous Stomatognathic Diseases

Age range

3 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Thalidomide (50mg)

Drug

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).

Colchicine

Drug

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 outcomes

  1. Proportion of Participants Achieving Complete Remission at 6 Months

    Time frame: 6 months

    Complete remission is defined as the total absence of febrile episodes (zero attacks) during the treatment period.

Secondary outcomes

  1. Complete Remission Rate at Multiple Time Points

    Time frame: 3 months

    Proportion of participants with zero febrile episodes.

  2. Complete Remission Rate at Multiple Time Points

    Time frame: 9 months

    Proportion of participants with zero febrile episodes.

  3. Complete Remission Rate at Multiple Time Points

    Time frame: 12 months

    Proportion of participants with zero febrile episodes.

  4. Partial Remission Rate

    Time frame: 3 months

    Proportion of participants achieving a reduction in the frequency of febrile episodes compared to baseline.

  5. Recurrence Rate Post-discontinuation

    Time frame: 6 months post-treatment

    Proportion of participants experiencing a relapse of symptoms after stopping the medication.

  6. Recurrence Rate Post-discontinuation

    Time frame: 12 months post-treatment

    Proportion of participants experiencing a relapse of symptoms after stopping the medication.

  7. Change in Growth Parameters (Z-scores)

    Time frame: 6 months

    Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).

  8. Change in Growth Parameters (Z-scores)

    Time frame: 12 months

    Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).

  9. Change in Inflammatory Markers

    Time frame: 6 months

    Changes in CRP, ESR, SAA, and cytokine levels.

Other outcomes

  1. Incidence of Adverse Events

    Time frame: 1 month

    Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).

  2. Incidence of Adverse Events

    Time frame: 6 months

    Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).

  3. Incidence of Adverse Events

    Time frame: 12 months

    Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).

  4. Quality of Life (PedsQL-SF15)

    Time frame: 6 months

    Change in Pediatric Quality of Life Inventory Short Form 15 scores.

  5. Quality of Life (PedsQL-SF15)

    Time frame: 12 months

    Change in Pediatric Quality of Life Inventory Short Form 15 scores.

Study contacts

Contact information is provided by the study sponsor or research team.

Chang Liu

CONTACT

[email protected]

86+13736320153

Sponsors and collaborators

Lead sponsor

Wenjie Zheng

Other

Collaborators

  • Children's Hospital of Soochow University
  • Jiangxi Province Children's Hospital
  • Jinan children's hospital
  • Nanjing Children's Hospital
  • Ningbo Women & Children's Hospital
  • The First Affiliated Hospital of Xiamen University
  • Tianjin Children's Hospital

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
Mar 17, 2026
Registry last updated
Mar 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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