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Probiotics and Corticosteroids for Treating Periodic Fever, Aphthous Stomatitis, Pharyngitis, Cervical Adenitis (PFAPA) (PFAPA)

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ClinicalTrials.gov Identifier: NCT02535962
Recruitment Status : Withdrawn (sponsor withdrawal)
First Posted : August 31, 2015
Last Update Posted : January 24, 2018
Sponsor:
Collaborator:
DuPont Nutrition and Health
Information provided by (Responsible Party):
Nicholas Bennett, Connecticut Children's Medical Center

Brief Summary:
The purpose of study is to see if adding probiotics to corticosteroid treatment for children with PFAPA could improve the health and daily of patients through reduction in febrile period frequency and length, along with concomitant reduction of associated symptoms. Current standard of care incorporates the administration of corticosteroids; however, while limiting the symptoms associated with PFAPA, corticosteroid use has been shown to increase the frequency at which these symptoms occur. Investigators hypothesize that administration of probiotics along with corticosteroids will work to decrease the frequency at which the febrile episodes occur. Additionally, probiotics may decrease the maximal fever experienced during these episodes, amount of corticosteroid needed to control the symptoms, average length of the episodes, and the number of patients who ultimately undergo tonsillectomy due to unsuccessful treatment with medication.

Condition or disease Intervention/treatment Phase
Periodic Fever Aphthous Stomatitis Pharyngitis Cervical Adenitis Biological: Lactobacillus acidophilus and Bifidobacterium lactis Other: Placebo Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Effects of Probiotic Addition to Corticosteroid Treatment for PFAPA
Study Start Date : October 2016
Actual Primary Completion Date : September 2017
Actual Study Completion Date : September 2017


Arm Intervention/treatment
Experimental: Corticosteriod + Probiotic Treatment

Corticosteroid dosing of 1 mg/kg of body weight given once at the onset of the febrile period, repeated once within 24 hours if necessary, but no more than two doses per cycle. The second dose of corticosteroid treatment is only to be given within one day following the initial dosage if the fever persists.

Investigational drug (Intervention is Lactobacillus acidophilus and Bifidobacterium lactis): Patients will be instructed to take one sachet of the study product mixed into a 60 ml of water that is not hot. Each sachet will contain Lactobacillus acidophilus NCFM and Bifidobacterium lactis Bi-07 at a dose of 5*109 CFU of each strain. The investigational product will be taken daily for the duration of the study, which is a year.

Biological: Lactobacillus acidophilus and Bifidobacterium lactis
study product will be freeze dried and put into foil sachets
Other Name: HOWARU® Protect Kids

Placebo Comparator: Corticosteriod + PlaceboTreatment

Corticosteroid dosing of 1 mg/kg of body weight given once at the onset of the febrile period, repeated once within 24 hours if necessary, but no more than two doses per cycle. The second dose of corticosteroid treatment is only to be given within one day following the initial dosage if the fever persists.

Placebo: will be taken daily and patients will be instructed to take one sachet of placebo mix into 60ml of water that is not to hot. The placebo will be taken daily for the duration of the study, which is one year. Placebo will be 1g of sucrose that has identical appearance, smell, and taste with the study product.

Other: Placebo
Placebo will look and taste like the investigational product. This also will be provided in a foil sachets




Primary Outcome Measures :
  1. Effects of the investigational treatment [ Time Frame: Time to event: number of fever-free days will be calculated, as well as the average number of days between fever cycles from baseline to end of study which is 12 months. ]
    Increased healthy intervals between cyclic febrile episodes in patients with PFAPA compared to patients being solely treated with corticosteroids.

  2. Safety of the investigational treatment - reported descriptively as percentages of patients experiencing adverse and serious adverse events. [ Time Frame: Time to event: number of fever-free days will be calculated, as well as the average number of days between fever cycles from baseline to end of study which is 12 months. ]
    Safety will be reported descriptively as percentages of patients experiencing adverse and serious adverse events.


Secondary Outcome Measures :
  1. Maximal fever experienced during a PFAPA febrile episode will be recorded as an average of the peak temperature recorded during each episode throughout the year and compared between the study groups [ Time Frame: Maximal fever experienced during a PFAPA febrile episode will be recorded as an average of the peak temperature recorded during each episode throughout the year ]
    Decreased maximal fever experienced during a PFAPA febrile episode.

  2. Number of administrations of corticosteroid necessary for treatment of PFAPA syndrome, recorded as the average number of administrations per fever episode throughout the year [ Time Frame: Number of administrations of corticosteroid necessary for treatment of PFAPA syndrome, recorded as the average number of administrations per fever episode throughout the year ]
    Decreased administrations of corticosteroid necessary for treatment of PFAPA syndrome.

  3. The average duration of the individual cyclic febrile episodes [ Time Frame: average duration of the individual cyclic febrile episodes in patients with PFAPA will be assessed from baseline to the end of study which is 1 year ]
    Decrease duration of the individual cyclic febrile episodes in patients with PFAPA.

  4. The number of patients undergoing tonsillectomy will be compared between the study groups [ Time Frame: At any time during the study from baseline to end of study, which is 12 months this will recorded ]
    Fewer patients undergoing tonsillectomy as a treatment for PFAPA syndrome



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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:

  • diagnosis or confirmation of diagnosis of PFAPA from Dr. Bennett based on clinical or laboratory data
  • is or will be undergoing treatment for PFAPA at CCMC
  • agrees to the consent and, if necessary, assent forms
  • is between 1 and 12 years of age

Exclusion Criteria:

  • is currently taking another probiotic regularly (>=2 times/ week)
  • is allergic to ingredients in the probiotic or placebo
  • may react adversely to the probiotic due to any form of immune deficiency or chronic disease including pulmonary, renal, cardiac disorders including underlying structural heart disease, gastrointestinal disease, or diabetes
  • is not a proficient English speaker
  • does not agree to the consent and/or assent forms
  • patients who use antibiotics or have used them within a month of the study start

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): NCT02535962


Sponsors and Collaborators
Connecticut Children's Medical Center
DuPont Nutrition and Health
Investigators
Principal Investigator: Nicholas Bennett, MBBChir PhD Connecticut Children's Medical Center

Responsible Party: Nicholas Bennett, Medical Director of Infectious Disease Department, Connecticut Children's Medical Center
ClinicalTrials.gov Identifier: NCT02535962     History of Changes
Other Study ID Numbers: 15-017
First Posted: August 31, 2015    Key Record Dates
Last Update Posted: January 24, 2018
Last Verified: January 2018

Keywords provided by Nicholas Bennett, Connecticut Children's Medical Center:
PFAPA

Additional relevant MeSH terms:
Fever
Pharyngitis
Stomatitis
Stomatitis, Aphthous
Familial Mediterranean Fever
Amyloidosis
Lymphadenitis
Body Temperature Changes
Signs and Symptoms
Pharyngeal Diseases
Stomatognathic Diseases
Respiratory Tract Infections
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Mouth Diseases
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Proteostasis Deficiencies
Metabolic Diseases
Lymphatic Diseases