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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 19, 2019
Sponsor:
Collaborators:
Oulu University Hospital
Tampere University
Tampere University Hospital
Turku University Hospital
University of Turku
Information provided by (Responsible Party):
University of Oulu

Brief Summary:
Children with periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) -syndrome diagnosis will be randomised either to tonsillotomy (partial tonsillectomy) or 3 months follow up. At follow up visit 3 months after randomisation the children from either groups with ongoing symptoms will be sent to tonsillectomy.

Condition or disease Intervention/treatment Phase
PFAPA Syndrome Procedure: Tonsillotomy Not Applicable

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 38 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Tonsillotomy
The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation.
Procedure: Tonsillotomy
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



Primary Outcome Measures :
  1. Cure [ Time Frame: 3 months from randomization ]
    Proportion of patients who do not have any PFAPA symptoms


Secondary Outcome Measures :
  1. Rescue surgery [ Time Frame: 12 months from randomization ]
    Proportion of patients needing rescue surgery (tonsillectomy)

  2. Days with fever [ Time Frame: 3 months from randomization ]
    Number of days with fever >38C in symptom diaries



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosed PFAPA syndrome: regularly occurring fever episodes for 3-5 days for at least five times or for 6 months

Exclusion Criteria:

  • prior tonsil surgery

Information from the National Library of Medicine

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


Contacts
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Contact: Marjo Renko, prof +358503878887 marjo.renko@oulu.fi
Contact: Ulla Lantto, MD ulla.lantto@ppshp.fi

Locations
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Finland
Oulu Unversity Hospital Recruiting
Oulu, Finland
Contact: Marjo Renko, MD, PhD       marjo.renko@oulu.fi   
Contact: Terhi Tapiainen, MD, PhD       terhi.tapiainen@oulu.fi   
Principal Investigator: Marjo Renko, MD, PhD         
Sub-Investigator: Terhi Tapiainen, MD, PhD         
Sub-Investigator: Ulla Lantto, MD         
Tampere University Hospital Not yet recruiting
Tampere, Finland
Contact: Elina Halme, MD, PhD         
Contact: Jarno Kujansivu, MD         
Turku University Hospital Not yet recruiting
Turku, Finland
Contact: Jaakko Piitulainen, LT, el         
Contact: Jami Rekola, LT, el         
Sponsors and Collaborators
University of Oulu
Oulu University Hospital
Tampere University
Tampere University Hospital
Turku University Hospital
University of Turku
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Responsible Party: University of Oulu
ClinicalTrials.gov Identifier: NCT03331497    
Other Study ID Numbers: TT 50_2017
First Posted: November 6, 2017    Key Record Dates
Last Update Posted: November 19, 2019
Last Verified: November 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Syndrome
Disease
Pathologic Processes