Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis SyndromeNCT07476625
Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome
A study testing Thalidomide (50mg) and Colchicine in people with Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome, run by Wenjie Zheng.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome
- What is being tested?
- Thalidomide (50mg) and Colchicine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 106 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2026to 30 Mar 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Thalidomide in Treating Pediatric PFAPA Syndrome: A Multicenter Randomized Controlled Trial
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.
Read more
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.
- Being tested
- Thalidomide (50mg), Colchicine
- Medical subject headings
- Hereditary Autoinflammatory Diseases, Stomatitis, Aphthous, Pharyngitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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).
ColchicineDrug
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).
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide Group | Experimental | Patients in this group will receive oral thalidomide treatment for 12 months. Receives: Drug: Thalidomide (50mg) |
| Colchicine Group | Active comparator | Patients in this group will receive oral colchicine treatment for 12 months. Receives: Drug: Colchicine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Secondary outcomes (9)
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Measured: 3 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Measured: 9 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Measured: 12 months
Partial Remission Rate
Proportion of participants achieving a reduction in the frequency of febrile episodes compared to baseline.
Measured: 3 months
Recurrence Rate Post-discontinuation
Proportion of participants experiencing a relapse of symptoms after stopping the medication.
Measured: 6 months post-treatment
Recurrence Rate Post-discontinuation
Proportion of participants experiencing a relapse of symptoms after stopping the medication.
Measured: 12 months post-treatment
Change in Growth Parameters (Z-scores)
Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).
Measured: 6 months
Change in Growth Parameters (Z-scores)
Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).
Measured: 12 months
Change in Inflammatory Markers
Changes in CRP, ESR, SAA, and cytokine levels.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2026
- Study start1 Apr 2026 (estimated)
- Primary completion31 Mar 2027 (estimated)
- Study completion30 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 106 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wenjie Zheng
- Sponsor type
- University, hospital or other
- 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, Children's Hospital Affiliated to Shandong University, Jiangxi Province Children's Hospital
- Responsible party
- Sponsor-investigator, Wenjie Zheng, Second Affiliated Hospital of Wenzhou Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07476625
- Sponsor's protocol ID
- SAHoWMU-CR2026-06-102
- Status last verified
- Mar 2026
- First submitted
- 9 Mar 2026
- Last updated
- 17 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Data contains sensitive patient information and is restricted by institutional policy.
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Thalidomide in Treating Pediatric PFAPA Syndrome: A Multicenter Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT07476625
- Acronym / sponsor ID
- SAHoWMU-CR2026-06-102
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 106 participants (estimated)
- Lead sponsor
- Wenjie ZhengUniversity, hospital or other
- 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, Children's Hospital Affiliated to Shandong University, Jiangxi Province Children's Hospital
- Study start
- 1 Apr 2026 (estimated)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- 30 Mar 2028 (estimated)
- First posted
- 17 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Thalidomide Group | Experimental | Drug: Thalidomide (50mg)Patients in this group will receive oral thalidomide treatment for 12 months. |
| Colchicine Group | Active comparator | Drug: ColchicinePatients in this group will receive oral colchicine treatment for 12 months. |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Thalidomide Group |
| ColchicineThe 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). | Drug | Colchicine Group |
Primary outcome
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.
9 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
- Individual participant dataSharing planned: No. Data contains sensitive patient information and is restricted by institutional policy.
Related trials for Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome
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RecruitingObservational study
AutoInflammatory Disease Alliance Registry (AIDA)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A study testing Thalidomide (50mg) and Colchicine in people with Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome, run by Wenjie Zheng.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chang Liu (86+13736320153, 1712400881@qq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Participants Achieving Complete Remission at 6 Months (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2026.