Functional ConstipationNCT05340712
Evaluation of the Efficacy of a New Specific Infant Formula in Case of Functional Constipation
A study testing new infant formula and Standard formula in people with Functional Constipation, run by United Pharmaceuticals at 2 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 month to 12 months
- Conditions the study is for
- Functional Constipation
- What is being tested?
- new infant formula and Standard formula
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 2 sitesItaly
- Study dates
- 20 Jul 2022to Sep 2024
- 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 month to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
Who cannot join
None listed.
Trial locations and contacts
2 sites in 1 country.
Italy2 sites, 2 recruiting
- Recruiting
University of Naples "Federico II"
Naples, 80131
Annamaria Staiano, Prof., 0039 0817462679, staiano@unina.it
Annamaria Staiano, Prof.
- Not yet recruiting
University of Campania Luigi Vanvitelli
Naples, 80138
Caterina Strisciuglio, Prof., caterinastrisciuglio@hotmail.it
Caterina Strisciuglio, Prof.
About this study
Official title: Evaluation of the Efficacy of a New Specific Infant Formula With a Prebiotic and Probiotics in Case of Functional Constipation
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.
Read the 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.
- Conditions
- Functional Constipation
- Being tested
- new infant formula, Standard formula
- Keywords
- Constipation, Infants, Infant formula, Nutrition, Defecation, Prebiotic, Probiotics
- Medical subject headings
- Constipation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
new infant formulaDietary supplement
New formula containing a prebiotic and a mix of probiotics, randomly assigned to infants during a 2-month period and allocated to all infants during the optional open-label follow-up period of 2 months.
Standard formulaDietary supplement
Also called Novalac 1
Standard infant formula containing no prebiotic nor probiotic, randomly assigned to infants during a 2-month period followed by an optional open-label follow-up period of 2 months with the new infant formula
| Study group | Role | What they receive |
|---|---|---|
| IT formula | Experimental | The test product (IT formula) is an infant formula based on cow's milk proteins containing a high level of lactose, a prebiotic, a high concentration of magnesium and a mix of probiotics. Receives: Dietary Supplement: new infant formula |
| Standard formula | Placebo comparator | The control product is a standard infant formula based on cow's milk proteins containing usual lactose and magnesium content, no prebiotic nor probiotic. Receives: Dietary Supplement: Standard formula |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hard stool consistency or painful defecation
Proportion of hard or painful stools per total number of spontaneous bowel movements by participant. A spontaneous bowel movement is defined as described in outcome 2.
Stool consistency will be evaluated based on the Brussels Infant and Toddler Stool Scale (BITSS). BITSS is a reliable instrument to assess stools of non-toilet trained children. It consists of color photographs of nappies containing stools from infants and toddlers categorized as hard, formed, loose, or watery (Huysentruyt et al., 2019).
The consistency and painful defecation of each stool will be assessed by parents at Day 30.
Measured: 30 days
Secondary outcomes (21)
Total and spontaneous defecation frequency
Spontaneous bowel movement is defined as a bowel movement not produced by the use of rescue medication. Stools occurring within 24h after the use of rescue medication will not be considered spontaneous. If 2 stools occur within less than 1 hour, only 1 stool will be considered (Koppen et al., 2018).
Defecation frequency will be assessed by parents by recording the time of each stool during the week preceding each visit.
Measured: 30, 60, 90 and 120 days
Stool consistency
Parents will record the consistency of every stool the week preceding each visit as described in the outcome 1 using the BITSS scale (Brussels Infant and Toddler Stool Scale) which consists of 4 stools consistency categories (hard, formed, loose, or watery ; Huysentruyt et al., 2019)
Measured: 30, 60, 90 and 120 days
Painful defecation
Painful defecation will be evaluated by parents for each stool during the week preceding each visit (Yes/No).
Measured: 30, 60, 90 and 120 days
Excessive stool retention
"Excessive stool retention" is defined as "the infant withholding stools". It will be evaluated by parents at each visit (presence/absence).
Measured: 30, 60, 90 and 120 days
Large diameter stools
Large diameter stools will be assessed by parents at each visit using the picture of the largest stool in the BITSS as an example of the large diameter stool (presence/absence).
Measured: 30, 60, 90 and 120 days
Treatment success
Treatment success is defined as no longer meeting the modified Rome IV criteria for functional constipation, i.e. the treatment will be considered as a success if the infant does not meet more than one of the following criteria per week during the week preceding each visit:
- At least one painful or hard spontaneous bowel movement (Yes/No)
- Two or fewer spontaneous defecations per week (Yes/No)
- Excessive stool retention (Yes/No)
- Large-diameter stools (assessed using the BITSS as described in the outcome 6) (Yes/No)
Measured: 30, 60, 90 and 120 days
Rescue medication use
The frequency of use, type and dosage of rescue medication will be assessed by parents during the whole study.
Measured: 30, 60, 90 and 120 days
Weight
Weight will be expressed in kg and in z scores according to the World Health Organization (WHO) Child Growth Standards.
Measured: 30, 60, 90 and 120 days
Height
Height will be expressed in cm and in z scores according to the WHO Child Growth Standards.
Measured: 30, 60, 90 and 120 days
Head circumference
Head circumference will be expressed in cm and in z scores according to the WHO Child Growth Standards.
Measured: 30, 60, 90 and 120 days
BMI
BMI will be expressed in value and z scores according to the WHO Child Growth Standards.
Measured: 30, 60, 90 and 120 days
Adverse events
Any untoward medical condition occurring during the study in an infant receiving study products regardless of a causal relationship with the product or not will be considered an adverse event and reported as such. This definition includes events occurring from the moment the parents have signed the Informed Consent form (day -7) up until the study end (or early termination).
Measured: Through study completion, i.e. 67 days or 127 days if participation to the optional follow-up period
Nutritional status
Nutritional status will be assessed based on the evaluation of specific blood biological markers (albumin, creatinine, urea, hemoglobin, sodium, potassium, chloride).
Measured: 0 and 60 days
Crying time
Daily crying duration will be assessed by parents on 3 days in the week preceding each visit.
Measured: 30, 60, 90 and 120 days
Regurgitations
The number of regurgitation episodes, the volume of regurgitation and regurgitation severity will be assessed by parents on 3 days in the week preceding each visit. Regurgitation severity will be assessed through the Vandenplas score ranging from 0 (=less than 2 regurgitation episodes / day) to 6 (=regurgitation of the complete volume after each feeding).
Measured: 30, 60, 90 and 120 days
Sleep quality
Sleep quality will be evaluated by parents at each visit by asking them if their child's sleep was quiet or agitated.
Measured: 30, 60, 90 and 120 days
Sleep time satisfaction
Sleep time satisfaction will be evaluated by parents at each visit (Yes/No).
Measured: 30, 60, 90 and 120 days
Parents' quality of life (QoL)
Parents' QoL will be assessed based on a questionnaire adapted from the Parental Opinions of Pediatric Constipation (POOPC) scale (Silverman et al., 2015). Questions from this scale were chosen based on their relevance to constipated infants. This questionnaire is composed of 4 items, i.e. Burden/Worry, Family, Treatment and Social. Each question is scored on a scale of 1 (=Strongly Disagree) to 5 (=Strongly Agree) with higher scores indicating that infant's constipation greatly affects parents' QoL. The total score is calculated by summing the scores of all individual question, ranging from 15 to 75 where a score of 15 indicates that infant's constipation does not affect parents' QoL whereas a score of 75 indicates that parents' QoL is extremely affected by infant's constipation.
Measured: 30, 60, 90 and 120 days
Parents' satisfaction
Parents' adequate relief will be assessed at each visit (Yes/No).
Measured: 30, 60, 90 and 120 days
Gut microbiota composition
Gut microbiota composition will be analyzed by 16S rRNA gene sequencing from stool samples.
Measured: 0 and 60 days
Short-chain fatty acids (SCFAs)
SCFAs will be measured in stool samples by gas chromatography/mass spectrometry (GC/MS).
Measured: 0 and 60 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2022
- Study start20 Jul 2022
- Primary completionDec 2023 (estimated)
- Study completionSep 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Functional ConstipationStudies 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
- Parallel groups
- 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- United Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Delta Consultants
- Responsible party
- Sponsor
- Investigators
- Annamaria Staiano, Prof., Study director, Federico II UniversityMarc Benninga, Prof., Study chair, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)Caterina Strisciuglio, Prof., Principal investigator, University of Campania Luigi Vanvitelli
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05340712
- Sponsor's protocol ID
- UP2021-02-FALCON
- Status last verified
- Nov 2022
- First submitted
- 8 Apr 2022
- Last updated
- 7 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 30672767Koppen 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.
PubMed 29380480Silverman 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.
PubMed 25840448
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy of a New Specific Infant Formula With a Prebiotic and Probiotics in Case of Functional Constipation
- ClinicalTrials.gov ID
- NCT05340712
- Acronym / sponsor ID
- UP2021-02-FALCON
- Conditions
- Functional Constipation
- Keywords
- Constipation, Infants, Infant formula, Nutrition, Defecation, Prebiotic, Probiotics
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- United PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Delta Consultants
- Study start
- 20 Jul 2022 (actual)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Sep 2024 (estimated)
- First posted
- 22 Apr 2022 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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
| Arm | Type | Receives |
|---|---|---|
| IT formula | Experimental | Dietary Supplement: new infant formulaThe test product (IT formula) is an infant formula based on cow's milk proteins containing a high level of lactose, a prebiotic, a high concentration of magnesium and a mix of probiotics. |
| Standard formula | Placebo comparator | Dietary Supplement: Standard formulaThe control product is a standard infant formula based on cow's milk proteins containing usual lactose and magnesium content, no prebiotic nor probiotic. |
Who may qualify
- Ages
- 1 month to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- 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)
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| new infant formulaNew formula containing a prebiotic and a mix of probiotics, randomly assigned to infants during a 2-month period and allocated to all infants during the optional open-label follow-up period of 2 months. | Dietary supplement | IT formula |
| Standard formulaAlso: Novalac 1Standard infant formula containing no prebiotic nor probiotic, randomly assigned to infants during a 2-month period followed by an optional open-label follow-up period of 2 months with the new infant formula | Dietary supplement | Standard formula |
Primary outcome
Hard stool consistency or painful defecation
Time frame: 30 days
Proportion of hard or painful stools per total number of spontaneous bowel movements by participant. A spontaneous bowel movement is defined as described in outcome 2. Stool consistency will be evaluated based on the Brussels Infant and Toddler Stool Scale (BITSS). BITSS is a reliable instrument to assess stools of non-toilet trained children. It consists of color photographs of nappies containing stools from infants and toddlers categorized as hard, formed, loose, or watery (Huysentruyt et al., 2019). The consistency and painful defecation of each stool will be assessed by parents at Day 30.
21 secondary outcomes
Total and spontaneous defecation frequency
Time frame: 30, 60, 90 and 120 days
Stool consistency
Time frame: 30, 60, 90 and 120 days
Painful defecation
Time frame: 30, 60, 90 and 120 days
Excessive stool retention
Time frame: 30, 60, 90 and 120 days
Large diameter stools
Time frame: 30, 60, 90 and 120 days
Treatment success
Time frame: 30, 60, 90 and 120 days
Rescue medication use
Time frame: 30, 60, 90 and 120 days
Weight
Time frame: 30, 60, 90 and 120 days
Height
Time frame: 30, 60, 90 and 120 days
Head circumference
Time frame: 30, 60, 90 and 120 days
BMI
Time frame: 30, 60, 90 and 120 days
Adverse events
Time frame: Through study completion, i.e. 67 days or 127 days if participation to the optional follow-up period
Nutritional status
Time frame: 0 and 60 days
Crying time
Time frame: 30, 60, 90 and 120 days
Regurgitations
Time frame: 30, 60, 90 and 120 days
Sleep quality
Time frame: 30, 60, 90 and 120 days
Sleep time satisfaction
Time frame: 30, 60, 90 and 120 days
Parents' quality of life (QoL)
Time frame: 30, 60, 90 and 120 days
Parents' satisfaction
Time frame: 30, 60, 90 and 120 days
Gut microbiota composition
Time frame: 0 and 60 days
Short-chain fatty acids (SCFAs)
Time frame: 0 and 60 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2022.
- Background: PubMed 30672767Huysentruyt 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.
- Background: PubMed 29380480Koppen 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.
- Background: PubMed 25840448Silverman 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.
- Individual participant dataSharing planned: No
Related trials for Functional Constipation
RecruitingPhase 2
A Study of Brenipatide (LY3537031) in Participants With Irritable Bowel Syndrome-Constipation (IBS-C)
Irritable Bowel Syndrome, Constipation
Active, not recruitingPhase 2
This is a Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel-group Study of the Efficacy and Safety of 2 Weight-based Treatment Groups of Plecanatide Versus Placebo in Children and Adolescent Participants 6 to Less Than 18 Years of Age With FC
Functional Constipation (FC)
Not yet recruitingPhase 3
Rifaximin Treatment in Bloating Predominant Functional Bowel Disorders
Bloating, Rifaximin
Recruiting
Effects of Connective Tissue Massage Plus Biofeedback for Dyssynergic Defecation
Dyssynergic Defecation
RecruitingObservational study
Relationship Between Interoception and Postural Control in Constipation-Predominant Irritable Bowel Syndrome
Constipation, Irritable Bowel Disease
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.
Annamaria Staiano, Prof. study director
Federico II University
Marc Benninga, Prof. study chair
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Caterina Strisciuglio, Prof. principal investigator
University of Campania Luigi Vanvitelli
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing new infant formula and Standard formula in people with Functional Constipation, run by United Pharmaceuticals at 2 sites in Italy.
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 2 inclusion and 0 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: status not confirmed, as of the sponsor's last update on 7 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. 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. Some participants in this study may receive a placebo. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anne-Sophie Garreau (0033-155372222, as.garreau@novalac.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hard stool consistency or painful defecation (time frame: 30 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05340712, which also lists 3 linked publications.
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 7 Nov 2022.