Trial Outcomes & Findings for Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH) (NCT NCT05228184)
NCT ID: NCT05228184
Last Updated: 2026-05-29
Results Overview
The LT4 dose is calculated based on the daily LT4 dose (mcg) used in the time period preceding the visit (or the average daily dose on a weekly basis if more than one strength is used over the course of the week) and the body weight (kg) measured during the visit
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
34 participants
Primary outcome timeframe
1 year of age
Results posted on
2026-05-29
Participant Flow
34 subjects were screened in the study, 31 were randomized. Three subjects were excluded before randomization because of temporary pause of randomization for logistical reasons (2) or for withdrawal by parent/guardian (1)
Participant milestones
| Measure |
Treatment
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
11
|
|
Overall Study
COMPLETED
|
16
|
10
|
|
Overall Study
NOT COMPLETED
|
4
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)
Baseline characteristics by cohort
| Measure |
Treatment
n=20 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=11 Participants
Crushed levothyroxine sodium tablets
|
Total
n=31 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=51 Participants
|
7 Participants
n=14 Participants
|
15 Participants
n=65 Participants
|
|
Age, Customized
neonates (<28 days of age)
|
3 Participants
n=51 Participants
|
1 Participants
n=14 Participants
|
4 Participants
n=65 Participants
|
|
Age, Customized
infants (age 29 - 277 days of age)
|
17 Participants
n=51 Participants
|
10 Participants
n=14 Participants
|
27 Participants
n=65 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=51 Participants
|
6 Participants
n=14 Participants
|
19 Participants
n=65 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=51 Participants
|
5 Participants
n=14 Participants
|
12 Participants
n=65 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=51 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=65 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=51 Participants
|
0 Participants
n=14 Participants
|
1 Participants
n=65 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=51 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=65 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=51 Participants
|
1 Participants
n=14 Participants
|
2 Participants
n=65 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=51 Participants
|
6 Participants
n=14 Participants
|
19 Participants
n=65 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=51 Participants
|
3 Participants
n=14 Participants
|
5 Participants
n=65 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=51 Participants
|
1 Participants
n=14 Participants
|
4 Participants
n=65 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
9 Participants
n=51 Participants
|
3 Participants
n=14 Participants
|
12 Participants
n=65 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=51 Participants
|
1 Participants
n=14 Participants
|
4 Participants
n=65 Participants
|
|
age at randomization
|
112.75 days
STANDARD_DEVIATION 94.624 • n=51 Participants
|
128.55 days
STANDARD_DEVIATION 99.877 • n=14 Participants
|
118.35 days
STANDARD_DEVIATION 95.157 • n=65 Participants
|
|
TSH at congenital hypothyroidism diagnosis
|
260.17 mIU/L
STANDARD_DEVIATION 196.254 • n=51 Participants
|
149.17 mIU/L
STANDARD_DEVIATION 115.004 • n=14 Participants
|
220.78 mIU/L
STANDARD_DEVIATION 178.090 • n=65 Participants
|
|
free T4 at congenital hypothyroidism diagnosis
|
12.55 pmol/L
STANDARD_DEVIATION 9.724 • n=51 Participants
|
12.30 pmol/L
STANDARD_DEVIATION 7.074 • n=14 Participants
|
12.46 pmol/L
STANDARD_DEVIATION 8.715 • n=65 Participants
|
PRIMARY outcome
Timeframe: 1 year of agePopulation: intent-to-treat (total number of subjects with data at the visit of 1 year of age = 27)
The LT4 dose is calculated based on the daily LT4 dose (mcg) used in the time period preceding the visit (or the average daily dose on a weekly basis if more than one strength is used over the course of the week) and the body weight (kg) measured during the visit
Outcome measures
| Measure |
Treatment
n=17 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=10 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
LT4 Dose Required to Maintain TSH in Target Range (Unit: mcg/kg/Day)
|
3.73 mcg/kg/day
Standard Deviation 1.469
|
5.06 mcg/kg/day
Standard Deviation 1.127
|
SECONDARY outcome
Timeframe: 1 year of agePopulation: Intent-to-Treat (total number of subjects with data at the visit of 1 year of age: 26)
TSH (unit mIU/L)
Outcome measures
| Measure |
Treatment
n=16 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=10 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Hormonal Profile for TSH
|
4.36 mIU/L
Interval 0.13 to 34.01
|
1.16 mIU/L
Interval 0.02 to 3.91
|
SECONDARY outcome
Timeframe: 1 year of agePopulation: intent-to-treat (total number of subjects with data at the visit of 1 year of age: 26)
FT4 (unit pmol/L)
Outcome measures
| Measure |
Treatment
n=16 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=10 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Hormonal Profile for FT4
|
18.34 pmol/L
Standard Deviation 4.511
|
21.69 pmol/L
Standard Deviation 7.681
|
SECONDARY outcome
Timeframe: Up to 22 months based on age groupPercent number of subjects (%) who need a dose adjustment in the long-term follow up phase
Outcome measures
| Measure |
Treatment
n=20 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=11 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Frequency of Dose Adjustments
|
15 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Up to 22 months based on age groupTSH values above 4.5 mU/L
Outcome measures
| Measure |
Treatment
n=103 TSH values
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=60 TSH values
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Number of Events of TSH Values Above 4.5 mU/L
|
28 number of events
|
13 number of events
|
SECONDARY outcome
Timeframe: Up to 22 months based on age groupFT4 values below the middle of the laboratory normal range
Outcome measures
| Measure |
Treatment
n=103 FT4 values
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=61 FT4 values
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Number of Events of FT4 Values Below the Middle of the Laboratory Normal Range
|
26 number of events
|
17 number of events
|
SECONDARY outcome
Timeframe: 1 year of agePopulation: intent-to-treat (total number of subjects with data at the visit of 1 year of age: 26)
Subject growth measurements in Length (cm) of body
Outcome measures
| Measure |
Treatment
n=16 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=10 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Growth Pattern for Length of Body
|
74.93 cm
Standard Deviation 2.748
|
74.42 cm
Standard Deviation 1.946
|
SECONDARY outcome
Timeframe: 1 year of agePopulation: intent-to-treat (total number of subjects with data at the visit of 1 year of age: 26)
Subject growth measurements in Body weight (kg)
Outcome measures
| Measure |
Treatment
n=16 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=10 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Growth Pattern for Body Weight
|
10.47 kg
Standard Deviation 1.185
|
9.57 kg
Standard Deviation 1.044
|
SECONDARY outcome
Timeframe: 1 year of agePopulation: intent-to-treat (total number of subjects with data at the visit of 1 year of age: 25)
Subject growth measurements in Head circumference (cm)
Outcome measures
| Measure |
Treatment
n=15 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=10 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Growth Pattern for Head Circumference
|
46.55 cm
Standard Deviation 1.722
|
46.95 cm
Standard Deviation 1.707
|
SECONDARY outcome
Timeframe: 3 weeks after treatment startPopulation: intent-to-treat (total number of subjects with data at the first Parents Satisfaction Questionnaire evaluation after treatment start)
Parents Satisfaction Questionnaire is a study-specific tool measuring coping and mental well-being and satisfaction in 20 questions. 12 of these questions were scored from 1 to 5, with higher score meaning better outcome. Subject scores were calculated for each subject by visit as mean of the LN(single question score) \[min=0; max=1.61\].
Outcome measures
| Measure |
Treatment
n=14 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=6 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Parent/Caregiver Reports of Satisfaction and Ease of Administration
|
1.39 score (ln-transformed)
Standard Deviation 0.141
|
1.26 score (ln-transformed)
Standard Deviation 0.209
|
SECONDARY outcome
Timeframe: first treatment weekCaregiver Administered Children's Acceptance Tool (CareCAT) is a 5-point nominal scale used to assess the acceptance of oral medicines in infants and toddlers who are unable to verbally give their opinion about a medicine
Outcome measures
| Measure |
Treatment
n=102 Completed CareCAT questionnaires
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=61 Completed CareCAT questionnaires
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Subject Acceptance of the Treatment (CareCAT)
Refusal
|
7 number of events
|
1 number of events
|
|
Subject Acceptance of the Treatment (CareCAT)
Swallows well
|
80 number of events
|
56 number of events
|
SECONDARY outcome
Timeframe: Up to 22 months based on age groupTotal number of subjects experiencing hypothyroidism's signs and symptoms
Outcome measures
| Measure |
Treatment
n=20 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=11 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Signs and Symptoms of Hypothyroidism
|
0 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Up to 22 months based on age groupTotal number of subjects experiencing hyperthyroidism's signs and symptoms
Outcome measures
| Measure |
Treatment
n=20 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=11 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Signs and Symptoms of Hyperthyroidism
|
5 Participants
|
3 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 28 daysPopulation: due to the limited number of subjects enrolled (only 4 neonates: 3 with in Treatment group and 1 in Control group) no statistical analysis has been performed
Time to normalize TSH into reference range (unit: days), computed as: (date of first TSH result into reference ranges - date of first LT4 dose intake + 1)
Outcome measures
| Measure |
Treatment
n=3 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=1 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Time to Normalize TSH in Neonates
|
13 days
Full Range 8.15 • Interval 11.0 to 26.0
|
24 days
Interval 24.0 to 24.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 6 monthsPopulation: due to the limited number of subjects enrolled (only 4 neonates: 3 with in Treatment group and 1 in Control group) no statistical analysis has been performed
Time to normalize FT4 into the upper half of the laboratory normal FT4 range (unit: days), computed as: (date of first FT4 result into the upper half of the laboratory reference ranges - date of first LT4 dose intake + 1)
Outcome measures
| Measure |
Treatment
n=3 Participants
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=1 Participants
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Time to Normalize FT4 in Neonates
|
13 days
Interval 8.0 to 144.0
|
15 days
Interval 15.0 to 15.0
|
Adverse Events
Treatment
Serious events: 2 serious events
Other events: 15 other events
Deaths: 0 deaths
Control
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment
n=20 participants at risk
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=11 participants at risk
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Infections and infestations
bronchiolitis
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
0.00%
0/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
gastroenteritis
|
0.00%
0/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
respiratory syncytial virus infection
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
0.00%
0/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Investigations
blood thyroid stimulating hormone increased
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
0.00%
0/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
Other adverse events
| Measure |
Treatment
n=20 participants at risk
Tirosint®-SOL (levothyroxine sodium) oral solution (IBSA Pharma Inc.) at the following strengths: 13, 25, 37.5, 44, 50, 62.5, 75, 88, 100 mcg.
|
Control
n=11 participants at risk
Crushed levothyroxine sodium tablets
|
|---|---|---|
|
Gastrointestinal disorders
constipation
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
18.2%
2/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Gastrointestinal disorders
diarrhoea
|
10.0%
2/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
0.00%
0/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
General disorders
pyrexia
|
25.0%
5/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
18.2%
2/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
covid-19
|
0.00%
0/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
18.2%
2/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
conjunctivitis
|
0.00%
0/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
ear infection
|
10.0%
2/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
18.2%
2/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
nasopharyngitis
|
10.0%
2/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
27.3%
3/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
otitis media
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
upper respiratory tract infection
|
15.0%
3/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
18.2%
2/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Musculoskeletal and connective tissue disorders
torticollis
|
0.00%
0/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory disorder
|
0.00%
0/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Skin and subcutaneous tissue disorders
dermatitis diaper
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Skin and subcutaneous tissue disorders
rash
|
5.0%
1/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
9.1%
1/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
|
Infections and infestations
gastroenteritis
|
0.00%
0/20 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
18.2%
2/11 • from enrolment until end of follow-up, up to 1 year of treatment
Adverse Events are not reported per each dose level, because dosage adjustments were made for each subject during the course of the study, based on periodic assessment of the clinical response and laboratory parameters.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place