Trial Outcomes & Findings for A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants (NCT NCT00655083)

Results Overview

Nepadutant was measured in the 24-h urine collection post both doses (0.1 and 0.5 mg/kg dose), in the age strata 6-\<12 and 12-\<18 weeks, using urinary collection/extraction from pre-weighed special fiber based diapers.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

21 participants

Primary outcome timeframe

24 hours

Results posted on

2011-10-17

Participant Flow

The recruitment period extended from March 2008 to May 2009 at three Hospital Clinics

Twenty out of the 21 enrolled infants were treated with study medication (1 infant did not attend the clinic after screening). NOTE: although the goal was to enroll 24 subjects, slow subject enrollment and expired drug restricted enrollment to 20 subjects

Participant milestones

Participant milestones
Measure	Nepadutant 0.1 mg/kg Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg Nepadutant 0.5 mg/kg
Overall Study STARTED	12	8
Overall Study COMPLETED	12	8
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nepadutant 0.1 mg/kg n=12 Participants Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg n=8 Participants Nepadutant 0.5 mg/kg	Total n=20 Participants Total of all reporting groups
Age, Customized Age Group 6 to <12 weeks	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants
Age, Customized Age Group 12 to <18 weeks	4 participants n=5 Participants	5 participants n=7 Participants	9 participants n=5 Participants
Age, Customized Age Group 18 to 24 weeks	4 participants n=5 Participants	0 participants n=7 Participants	4 participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants	8 participants n=7 Participants	20 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 hours

Population: Urinary drug concentration was analysed by dose and age stratum of the infants (6-\<12 and 12- \<18 weeks of age). Imputation technique was adopted for urine samples highly contaminated by faeces.

Nepadutant was measured in the 24-h urine collection post both doses (0.1 and 0.5 mg/kg dose), in the age strata 6-\<12 and 12-\<18 weeks, using urinary collection/extraction from pre-weighed special fiber based diapers.

Outcome measures

Outcome measures
Measure	Nepadutant 0.1 mg/kg n=8 Participants Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg n=8 Participants Nepadutant 0.5 mg/kg
Drug Concentration Measurement in the Urine Collected by Diapers Along 24 Hours Post Dose and One Week After Dose in All Treated Infants and by Age and Dose Subgroups. 6- <12 week old	5041 ng Standard Deviation 7081	6474 ng Standard Deviation 2463
Drug Concentration Measurement in the Urine Collected by Diapers Along 24 Hours Post Dose and One Week After Dose in All Treated Infants and by Age and Dose Subgroups. 12- < 18 week old	2095 ng Standard Deviation 1151	3727 ng Standard Deviation 2845

PRIMARY outcome

Timeframe: 24 hours

Population: Urinary drug concentration was analysed by dose and age stratum of the infants (18-24 weeks of age). Imputation technique was adopted for urine samples highly contaminated by faeces.

Nepadutant was measured in the 24-h urine collection post both doses (0.1 and 0.5 mg/kg dose), in the age stratum 18-24 weeks, using urinary collection/extraction from pre-weighed special fiber based diapers.

Outcome measures

Outcome measures
Measure	Nepadutant 0.1 mg/kg n=4 Participants Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg Nepadutant 0.5 mg/kg
Drug Concentration Measurement in the Urine Collected by Diapers Along 24 Hours Post Dose and One Week After Dose in All Treated Infants and by Age and Dose Subgroups.	1127 ng Standard Deviation 1032	—

SECONDARY outcome

Timeframe: one week

Population: Number of adverse events by dose and age strata (6-\<12 and 12- \<18 weeks of age) are reported by system organ class and preferred term.

Number of adverse events (AE) reported by dose and age stratum 6-\<12 and 12-\<18 weeks.

Outcome measures

Outcome measures
Measure	Nepadutant 0.1 mg/kg n=8 Participants Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg n=8 Participants Nepadutant 0.5 mg/kg
Number of Adverse Events After Administration of Single Oral Doses up to 0.5 mg/kg of Nepadutant in Infants. 6- <12 week old	0 Adverse Events	3 Adverse Events
Number of Adverse Events After Administration of Single Oral Doses up to 0.5 mg/kg of Nepadutant in Infants. 12- < 18 week old	1 Adverse Events	1 Adverse Events

SECONDARY outcome

Timeframe: one week

Population: Number of adverse events by dose and age stratum (18-24 weeks of age) are reported by system organ class and preferred term.

Number of adverse events (AE) reported by dose and age stratum 18-24 weeks.

Outcome measures

Outcome measures
Measure	Nepadutant 0.1 mg/kg n=4 Participants Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg Nepadutant 0.5 mg/kg
Number of Adverse Events After Administration of Single Oral Doses up to 0.5 mg/kg of Nepadutant in Infants.	3 Adverse Events	—

Adverse Events

Nepadutant 0.1 mg/kg

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Nepadutant 0.5 mg/kg

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Nepadutant 0.1 mg/kg n=12 participants at risk Nepadutant 0.1 mg/kg	Nepadutant 0.5 mg/kg n=8 participants at risk Nepadutant 0.5 mg/kg
Gastrointestinal disorders diarrhoea	0.00% 0/12 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	12.5% 1/8 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.
Gastrointestinal disorders vomiting	0.00% 0/12 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	12.5% 1/8 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.
Gastrointestinal disorders hard stools	8.3% 1/12 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	0.00% 0/8 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.
General disorders pyrexia	8.3% 1/12 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	12.5% 1/8 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.
Skin and subcutaneous tissue disorders dermatitis by diaper	0.00% 0/12 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	12.5% 1/8 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.
Ear and labyrinth disorders middle ear infection	8.3% 1/12 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	0.00% 0/8 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.
Respiratory, thoracic and mediastinal disorders cough	8.3% 1/12 • Number of events 1 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.	0.00% 0/8 • Adverse events were recorded after 24 hours after dosing and at 1 week final follow up.

Additional Information

Angela Capriati, Clinical research Director

Menarini Group- Clinical Research

Phone: + 39-055 56809990

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60