Clinical Safety & Efficacy of a New Infant Formula With Specific Medical Purpose Containing Human Milk Oligosaccharides

Study Overview
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Brief Summary
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The primary objective of the study is to show that infants with cow milk protein allergy (CMPA) fed with a new FSMP infant formula with reduced level of protein & with 2 Human Milk Oligosaccharides (HMOs) (test formula) have a growth in line with infants fed with a comparable FSMP formula but without HMOs (control formula). The secondary objectives are to assess whether consumption of Test formula by CMPA infants (i) reduces medication use and risk for infections in particular lower respiratory tract infections/morbidity, (ii) is well tolerated and allows for age appropriate growth and (iii) reduces health care costs.

Detailed Description
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Infants with physician diagnosed CMPA, aged between birth and 6 months of age will take either the control or new test infant formula for 4 months and if judged suitable by physician, up to maximum of 12 months of age. Growth, adverse events, medication use and tolerance to formula will be assessed. As part of exploratory objectives, the study will also explore possible mode of action of the Test formula in CMPA infants, by assessing whether consumption of Test formula by CMPA infants affects stool microbiota and metabolic signatures as well as urine metabolic signatures and whether such changes can be associated to the intestinal inflammatory/health status, and the clinical measures.

Official Title
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Clinical Safety & Efficacy of a New Infant Formula With Specific Medical Purpose (FSMP) Containing 2 Human Milk Oligosaccharides (HMOs)

Conditions
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Cow's Milk Protein Allergy

Intervention / Treatment
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* Other: Test infant formula with HMOs
* Other: Control infant formula without HMOs


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Full term infant (37 weeks ≤ gestation ≤ 42 weeks) 2500g ≤ birth weight ≤ 4500g Written informed consent. Infant aged between birth and 6 months. Not being breastfed at time of enrollment or mothers of CMPA infant doing breastfeeding and independently elected before enrollment to exclusively formula feed. Infants with physician diagnosed (and untreated with extensively hydrolysed or amino acid infant formula) Cow Milk Protein Allergy as per standard clinical practice and with at least 2 protocol specified symptoms present. Exclusion Criteria: Prior treatment with extensively hydrolysed infant formula for more than 72 hours or with amino acid infant formula. Congenital illness or malformation that may affect growth. Demonstrated chronic malabsorption not due to CMPA. Significant pre-natal and/or serious post-natal disease other than CMPA before enrollment (per investigator's medical decision). Minor parent(s). Infants whose parents or caregivers cannot be expected to comply with study procedures. Currently participating or having participated in another clinical trial since birth.

Ages Eligible for Study
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Maximum Age: 6 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Test infant formula with HMOs<br>Extensively hydrolysed infant formula with HMOs taken by infant according to age, weight and appetite. | Other: Test infant formula with HMOs<br>* Extensively hydrolysed (whey protein) specialty infant formulas (FSMP) with HMOs intended for management of CMPA.<br>|
| Active Comparator: Control infant formula without HMOs<br>Extensively hydrolysed infant formula without HMOs taken by infant according to age, weight and appetite | Other: Control infant formula without HMOs<br>* Extensively hydrolysed (whey protein) specialty infant formulas (FSMP) intended for management of CMPA.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Growth expressed as weight gain in grams per day | Body weight measured from enrollment to 4 months of study formula intake | 4 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and medication use | Assessment of occurrence of adverse events (Number of adverse events per infant and number of infants with adverse events) and use of concomitant medication (specifically amount of antipyretics and antibiotics used per infant and number of infants using these) from enrollment until infants are 12 months of age. | 12 months | 
| Growth in terms of body weight. | Body weight (in kilograms) measured from enrollment until infants are 12 months of age. | 12 months | 
| Growth in terms of body length | Body length (in centimetres) measured from enrollment until infants are 12 months of age. | 12 months | 
| Growth in terms of head circumference | Head circumference (in centimetres) measured from enrollment until infants are 12 months of age. | 12 months | 
| Digestive tolerance and alleviation of CMPA | Recording of stool characteristics and frequency in diary, assessment of infant behavior pattern and medical assessment at each visit from enrollment until last study formula intake. Outcome will be reported as score on CoMiSS tool. | 12 months | 
| Compliance to study formula intake | Recording daily quantity consumed (in milliliters) in feeding diary for 3 days immediately before each visit from enrollment until last study formula intake. | 12 months | 
| Healthcare resource use assessed with Questionnaire | Questionnaire completed from enrollment until infants are 12 months of age. | 12 months |