PFAPA SyndromeNCT03331497
Tonsillotomy or Follow-up in PFAPA Syndrome
A study testing Tonsillotomy in people with PFAPA Syndrome, run by University of Oulu at 3 sites in Finland.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 12 years
- Conditions the study is for
- PFAPA Syndrome
- What is being tested?
- Tonsillotomy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 3 sitesFinland
- Study dates
- 24 Oct 2017to 2 Feb 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 1 year to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosed PFAPA syndrome: regularly occurring fever episodes for 3-5 days for at least five times or for 6 months
Who cannot join
- prior tonsil surgery
Trial locations and contacts
3 sites in 1 country.
Finland3 sites
Oulu Unversity Hospital
Oulu
Tampere University Hospital
Tampere
Turku University Hospital
Turku
About this study
Official title: Tonsillotomy or Follow-up in PFAPA -Syndrome - Randomised, Controlled Study Using Sequential Design
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.
Read the 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.
- Conditions
- PFAPA Syndrome
- Being tested
- Tonsillotomy
- Medical subject headings
- Marshall syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TonsillotomyProcedure or surgery
About 2/3 of the palatine tonsil tissue is removed with coplator or bipolar knife.
| Study group | Role | What they receive |
|---|---|---|
| Tonsillotomy | Experimental | The patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation. Receives: Procedure: Tonsillotomy |
| Follow up | No intervention | The patients diagnosed with PFAPA will be monitored for 3 months time. If the symptoms still persist, tonsillectomy will be performed |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cure
Proportion of patients who do not have any PFAPA symptoms
Measured: 3 months from randomization
Secondary outcomes (3)
Rescue surgery
Proportion of patients needing rescue surgery (tonsillectomy)
Measured: 12 months from randomization
Days with fever
Number of days with fever >38C in symptom diaries
Measured: 3 months from randomization
Cure
Proportion of patients who do not have any PFAPA symptoms
Measured: 6 months from randomization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2017
- Study start24 Oct 2017
- Primary completion30 Dec 2021
- Study completion2 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PFAPA 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Oulu
- Sponsor type
- University, hospital or other
- Collaborators
- Oulu University Hospital, Tampere University, Tampere University Hospital, Turku University Hospital, University of Turku
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03331497
- Sponsor's protocol ID
- TT 50_2017
- Status last verified
- Feb 2022
- First submitted
- 31 Oct 2017
- Last updated
- 21 Feb 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tonsillotomy or Follow-up in PFAPA -Syndrome - Randomised, Controlled Study Using Sequential Design
- ClinicalTrials.gov ID
- NCT03331497
- Acronym / sponsor ID
- TT 50_2017
- Conditions
- PFAPA Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- University of OuluUniversity, hospital or other
- Collaborators
- Oulu University Hospital, Tampere University, Tampere University Hospital, Turku University Hospital, University of Turku
- Study start
- 24 Oct 2017 (actual)
- Primary completion
- 30 Dec 2021 (actual)
- Study completion
- 2 Feb 2022 (actual)
- First posted
- 6 Nov 2017 (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.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tonsillotomy | Experimental | Procedure: TonsillotomyThe patients diagnosed with PFAPA will have tonsillotomy performed in one month from randomisation. |
| Follow up | No intervention | –The patients diagnosed with PFAPA will be monitored for 3 months time. If the symptoms still persist, tonsillectomy will be performed |
Who may qualify
- Ages
- 1 year to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Diagnosed PFAPA syndrome: regularly occurring fever episodes for 3-5 days for at least five times or for 6 months
Key exclusion criteria (1 listed)
- prior tonsil surgery
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 |
|---|---|---|
| TonsillotomyAbout 2/3 of the palatine tonsil tissue is removed with coplator or bipolar knife. | Procedure or surgery | Tonsillotomy |
Primary outcome
Cure
Time frame: 3 months from randomization
Proportion of patients who do not have any PFAPA symptoms
3 secondary outcomes
Rescue surgery
Time frame: 12 months from randomization
Days with fever
Time frame: 3 months from randomization
Cure
Time frame: 6 months from randomization
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2022.
Related trials for PFAPA Syndrome
RecruitingObservational study
Evaluating the Genetics and Immunology of Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome and Other Tonsil Disorders
Periodic Fever, Aphthous Stomatitis, Pharyngitis, And Cervical Adenitis (Pfapa), Obstructive Sleep Apnea
Not yet recruiting
Efficacy and Safety of Thalidomide for Pediatric PFAPA Syndrome
Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis Syndrome
RecruitingObservational study
AutoInflammatory Disease Alliance Registry (AIDA)
Hereditary Autoinflammatory Diseases, Schnitzler Syndrome
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Tonsillotomy in people with PFAPA Syndrome, run by University of Oulu at 3 sites in Finland.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 21 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Finland. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cure (time frame: 3 months from randomization). 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 21 Feb 2022.