Tonsillotomy or Follow-up in PFAPA Syndrome
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03331497|
Recruitment Status : Recruiting
First Posted : November 6, 2017
Last Update Posted : November 24, 2020
|Condition or disease||Intervention/treatment||Phase|
|PFAPA Syndrome||Procedure: Tonsillotomy||Not Applicable|
Children with periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) -syndrome diagnosis will be randomised either to tonsillotomy (partial tonsillectomy) or 3 months follow up.
In both groups the symptoms are monitored with symptom diaries. At follow up visit 3 months after randomisation the children from either groups with ongoing symptoms will be sent to tonsillectomy (total remove of palatine tonsil).
The design is sequential trial. From the previous trials we know that the effect of tonsillectomy is about 90% and that during follow up for six months as much as 50% of the patients will recover. Because the syndrome is rare and because the rescue operations of the tonsils (tonsillectomy in a person who has gone through tonsillotomy), the sample size has to be as low as possible. That is the reason why we chose sequential design, with assessments after every 8 patient.
Randomization is made in blocks of four. In sequential design we use the following assumptions:
- We are looking for a 40% absolute difference in cure rate between tonsillotomy (90%) and follow up (50%) groups.
- Two sided Whitehead design
- Maximum likelihood estimation (MLE)
- Five assessments (sample size 4+4, 8+8. 12+12, 16+16 and 19+19)
- type 1 error 5 % and power 80%
- The sample size will be from 8 to 38 patients depending on when the trial can be stopped along assessments.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||38 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||Tonsillotomy or Follow-up in PFAPA -Syndrome - Randomised, Controlled Study Using Sequential Design|
|Actual Study Start Date :||October 24, 2017|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation.
About 2/3 of the palatine tonsil tissue is removed with coplator or bipolar knife.
No Intervention: Follow up
The patients diagnosed with PFAPA will be monitored for 3 months time. If the symptoms still persist, tonsillectomy will be performed
- Cure [ Time Frame: 3 months from randomization ]Proportion of patients who do not have any PFAPA symptoms
- Rescue surgery [ Time Frame: 12 months from randomization ]Proportion of patients needing rescue surgery (tonsillectomy)
- Days with fever [ Time Frame: 3 months from randomization ]Number of days with fever >38C in symptom diaries
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03331497
|Contact: Marjo Renko, email@example.com|
|Contact: Ulla Lantto, MDfirstname.lastname@example.org|
|Oulu Unversity Hospital||Recruiting|
|Contact: Marjo Renko, MD, PhD email@example.com|
|Contact: Terhi Tapiainen, MD, PhD firstname.lastname@example.org|
|Principal Investigator: Marjo Renko, MD, PhD|
|Sub-Investigator: Terhi Tapiainen, MD, PhD|
|Sub-Investigator: Ulla Lantto, MD|
|Tampere University Hospital||Recruiting|
|Contact: Elina Halme, MD, PhD|
|Contact: Jarno Kujansivu, MD|
|Turku University Hospital||Recruiting|
|Contact: Jaakko Piitulainen, LT, el|
|Contact: Jami Rekola, LT, el|