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| Name | Class |
|---|---|
| Nanjing Children's Hospital | OTHER |
| Children's Hospital of Soochow University | OTHER |
| The First Affiliated Hospital of Xiamen University | OTHER |
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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Thalidomide Group | Experimental | Patients in this group will receive oral thalidomide treatment for 12 months. |
|
| Colchicine Group | Active Comparator | Patients in this group will receive oral colchicine treatment for 12 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Remission Rate at Multiple Time Points | Proportion of participants with zero febrile episodes. | 3 months |
| Complete Remission Rate at Multiple Time Points | Proportion of participants with zero febrile episodes. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events | Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness). | 1 month |
| Incidence of Adverse Events |
Inclusion Criteria:
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):
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Chang Liu | Contact | 86+13736320153 | 1712400881@qq.com |
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Data contains sensitive patient information and is restricted by institutional policy.
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| ID | Term |
|---|---|
| D056660 | Hereditary Autoinflammatory Diseases |
| D013281 | Stomatitis, Aphthous |
| D010612 | Pharyngitis |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D012871 | Skin Diseases |
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| ID | Term |
|---|---|
| D013792 | Thalidomide |
| D003078 | Colchicine |
| ID | Term |
|---|---|
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
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| Tianjin Children's Hospital |
| OTHER |
| Ningbo Women & Children's Hospital | OTHER |
| Jinan children's hospital | UNKNOWN |
| Jiangxi Province Children's Hospital | OTHER |
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|
| 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). |
|
| 9 months |
| Complete Remission Rate at Multiple Time Points | Proportion of participants with zero febrile episodes. | 12 months |
| Partial Remission Rate | Proportion of participants achieving a reduction in the frequency of febrile episodes compared to baseline. | 3 months |
| Recurrence Rate Post-discontinuation | Proportion of participants experiencing a relapse of symptoms after stopping the medication. | 6 months post-treatment |
| Recurrence Rate Post-discontinuation | Proportion of participants experiencing a relapse of symptoms after stopping the medication. | 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). | 6 months |
| Change in Growth Parameters (Z-scores) | Changes in Height-for-age Z-score (HAZ) and Weight-for-age Z-score (WAZ). | 12 months |
| Change in Inflammatory Markers | Changes in CRP, ESR, SAA, and cytokine levels. | 6 months |
Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness).
| 6 months |
| Incidence of Adverse Events | Percentage of participants experiencing diarrhea, abdominal pain, nausea, liver function abnormality, somnolence, constipation, rash, or peripheral neuropathy (numbness). | 12 months |
| Quality of Life (PedsQL-SF15) | Change in Pediatric Quality of Life Inventory Short Form 15 scores. | 6 months |
| Quality of Life (PedsQL-SF15) | Change in Pediatric Quality of Life Inventory Short Form 15 scores. | 12 months |
| D017437 | Skin and Connective Tissue Diseases |
| D013280 | Stomatitis |
| D009059 | Mouth Diseases |
| D009057 | Stomatognathic Diseases |
| D012141 | Respiratory Tract Infections |
| D007239 | Infections |
| D010608 | Pharyngeal Diseases |
| D012140 | Respiratory Tract Diseases |
| D010038 | Otorhinolaryngologic Diseases |
| D009930 |
| Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D000470 | Alkaloids |