An interventional study of Thalidomide (50mg) and Colchicine in Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome, sponsored by Wenjie Zheng. Not yet recruiting. Open to participants aged 3 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-03-17.
Sponsored by Wenjie Zheng · Not applicable, Interventional, and Treatment
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.
12 studies on the registry are indexed under Hereditary Autoinflammatory Diseases; 5 are open to participants now.
Browse Hereditary Autoinflammatory Diseases studies →Wenjie Zheng is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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 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):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients in this group will receive oral thalidomide treatment for 12 months.
Drug: Thalidomide (50mg)
Patients in this group will receive oral colchicine treatment for 12 months.
Drug: Colchicine
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).
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.
Time frame: 6 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Time frame: 3 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Time frame: 9 months
Complete Remission Rate at Multiple Time Points
Proportion of participants with zero febrile episodes.
Time frame: 12 months
Partial Remission Rate
Proportion of participants achieving a reduction in the frequency of febrile episodes compared to baseline.
Time frame: 3 months
Recurrence Rate Post-discontinuation
Proportion of participants experiencing a relapse of symptoms after stopping the medication.
Time frame: 6 months post-treatment
Recurrence Rate Post-discontinuation
Proportion of participants experiencing a relapse of symptoms after stopping the medication.
Time frame: 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).
Time frame: 6 months
Change in Growth Parameters (Z-scores)
Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ).
Time frame: 12 months
Change in Inflammatory Markers
Changes in CRP, ESR, SAA, and cytokine levels.
Time frame: 6 months
Incidence of Adverse Events
Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).
Time frame: 1 month
Incidence of Adverse Events
Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).
Time frame: 6 months
Incidence of Adverse Events
Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).
Time frame: 12 months
Quality of Life (PedsQL-SF15)
Change in Pediatric Quality of Life Inventory Short Form 15 scores.
Time frame: 6 months
Quality of Life (PedsQL-SF15)
Change in Pediatric Quality of Life Inventory Short Form 15 scores.
Time frame: 12 months
No study locations are listed for this record.
Plan to share: No — Data contains sensitive patient information and is restricted by institutional policy.
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Thalidomide
Hereditary Autoinflammatory Diseases →