Clinical trial NCT05340712: Evaluation of the Efficacy of a New Specific Infant Formula in Case of Functional Constipation
Official title: Evaluation of the Efficacy of a New Specific Infant Formula With a Prebiotic and Probiotics in Case of Functional Constipation
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Functional Constipation
Keywords: Constipation
Infants
Infant formula
Nutrition
Defecation
Prebiotic
Probiotics
Lead sponsor: United Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Delta Consultants
First posted: 2022-04-22
Enrollment: 80
Sex: ALL
Minimum age (years): 1
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Superiority, double-blind, randomized, placebo-controlled trial of 60 days followed by an optional 60 day, open-label follow-up period with the tested formula only
Primary purpose: TREATMENT

Brief summary: This study aims to assess the clinical efficacy, safety and tolerance of a new formula containing a prebiotic and a mix of probiotics in the specific population of infants suffering from functional constipation through a superiority, double-blind, randomized, placebo-controlled trial.

Detailed description: The study comprises a selection period of 1 week where the eligibility of the infant to participate in the study will be evaluated. Once his/her participation is confirmed, the tested formula will be compared to a standard formula during a 2-month study period followed by an optional follow-up period of 2 months where only the tested formula will be used.

Eligibility criteria: Selection Criteria:

Infants presenting with constipation according to modified Rome IV criteria, i.e. they must present with 1 month the following:

* Infants without spontaneous stools or with at least one painful or hard spontaneous bowel movement per week AND
* At least one of the following:

  * Two and fewer spontaneous defecations per week
  * History of excessive stool retention
  * History of large-diameter stools

Non-selection Criteria (non-exhaustive list):

* Preterm birth (\<34 weeks of gestational age)
* Exclusively or partially breastfed infants within 3 days prior to visit V-1
* Infants with organic causes of constipation (e.g. coeliac disease, paediatric intestinal pseudo-obstruction, hypothyroidism, spina bifida, anorectal malformation or Hirschsprung's disease) or constipation secondary to endocrine, metabolic, neurologic or autoimmune disorders, surgery or drugs.
* Infants on laxative treatment
* Infants taking drugs that are known to affect gastrointestinal motility as well as any probiotic supplements
* Infants treated with antibiotics
* Infants with a known allergy to one of the formula ingredients (e.g. cow's milk proteins...)
* Infants with anaemia who have been prescribed an oral iron supplement as treatment
* Infants participating in another trial
* Infants presenting with any other situation which, according to the investigator, may interfere with the study participation, or lead to a particular risk for the infant

Inclusion Criteria:

* Infants having completed the selection period and still compliant with non-selection criteria
* Infants being constipated according to modified Rome IV criteria based on information recorded in parents' diary during the week preceding visit V0 (inclusion visit)

Primary outcomes:
measure: Hard stool consistency or painful defecation
timeFrame: 30 days
Secondary outcomes:
measure: Total and spontaneous defecation frequency
timeFrame: 30, 60, 90 and 120 days
measure: Stool consistency
timeFrame: 30, 60, 90 and 120 days
measure: Painful defecation
timeFrame: 30, 60, 90 and 120 days
measure: Excessive stool retention
timeFrame: 30, 60, 90 and 120 days
measure: Large diameter stools
timeFrame: 30, 60, 90 and 120 days
measure: Treatment success
timeFrame: 30, 60, 90 and 120 days
measure: Rescue medication use
timeFrame: 30, 60, 90 and 120 days
measure: Weight
timeFrame: 30, 60, 90 and 120 days
measure: Height
timeFrame: 30, 60, 90 and 120 days
measure: Head circumference
timeFrame: 30, 60, 90 and 120 days
measure: BMI
timeFrame: 30, 60, 90 and 120 days
measure: Adverse events
timeFrame: Through study completion, i.e. 67 days or 127 days if participation to the optional follow-up period
measure: Nutritional status
timeFrame: 0 and 60 days
measure: Crying time
timeFrame: 30, 60, 90 and 120 days
measure: Regurgitations
timeFrame: 30, 60, 90 and 120 days
measure: Sleep quality
timeFrame: 30, 60, 90 and 120 days
measure: Sleep time satisfaction
timeFrame: 30, 60, 90 and 120 days
measure: Parents' quality of life (QoL)
timeFrame: 30, 60, 90 and 120 days
measure: Parents' satisfaction
timeFrame: 30, 60, 90 and 120 days
measure: Gut microbiota composition
timeFrame: 0 and 60 days
measure: Short-chain fatty acids (SCFAs)
timeFrame: 0 and 60 days
Other outcomes:
Locations:
city: Napoli
country: Italy
facility: University of Naples "Federico II"
city: Napoli
country: Italy
facility: University of Campania Luigi Vanvitelli
Study officials:
affiliation: Federico II University
name: Annamaria Staiano, Prof.
role: STUDY_DIRECTOR
affiliation: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
name: Marc Benninga, Prof.
role: STUDY_CHAIR
affiliation: University of Campania Luigi Vanvitelli
name: Caterina Strisciuglio, Prof.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Huysentruyt K, Koppen I, Benninga M, Cattaert T, Cheng J, De Geyter C, Faure C, Gottrand F, Hegar B, Hojsak I, Miqdady M, Osatakul S, Ribes-Koninckx C, Salvatore S, Saps M, Shamir R, Staiano A, Szajewska H, Vieira M, Vandenplas Y; BITSS working group. The Brussels Infant and Toddler Stool Scale: A Study on Interobserver Reliability. J Pediatr Gastroenterol Nutr. 2019 Feb;68(2):207-213. doi: 10.1097/MPG.0000000000002153.
citation: Koppen IJN, Saps M, Lavigne JV, Nurko S, Taminiau JAJM, Di Lorenzo C, Benninga MA. Recommendations for pharmacological clinical trials in children with functional constipation: The Rome foundation pediatric subcommittee on clinical trials. Neurogastroenterol Motil. 2018 Apr;30(4):e13294. doi: 10.1111/nmo.13294. Epub 2018 Jan 30.
citation: Silverman AH, Berlin KS, Di Lorenzo C, Nurko S, Kamody RC, Ponnambalam A, Mugie S, Gorges C, Sanghavi R, Sood MR. Measuring Health-Related Quality of Life With the Parental Opinions of Pediatric Constipation Questionnaire. J Pediatr Psychol. 2015 Sep;40(8):814-24. doi: 10.1093/jpepsy/jsv028. Epub 2015 Apr 2.