Trial Outcomes & Findings for Treatment of Adrenal Insufficiency in Children (NCT NCT02720952)

NCT ID: NCT02720952

Last Updated: 2022-05-04

Results Overview

The primary endpoint will be the maximum levels of serum cortisol concentration up to 240 minutes after intake of study drug as determined by the central laboratory.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 24 participants
• Primary outcome timeframe: 240 minutes
• Results posted on: 2022-05-04

Participant Flow

Participant milestones

Measure	Infacort Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone
Overall Study STARTED	24
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Treatment of Adrenal Insufficiency in Children

Baseline characteristics by cohort

Measure	Infacort n=24 Participants Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone
Age, Categorical <=18 years	24 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	718.1 days n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	24 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	24 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Germany	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 240 minutes

The primary endpoint will be the maximum levels of serum cortisol concentration up to 240 minutes after intake of study drug as determined by the central laboratory.

Outcome measures

Measure	Infacort n=24 Participants Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone	Question 2 Percentage of parents/carers that agreed, or strongly agreed with the statement: My child showed a positive reaction after Infacort was given.	Question 3 Percentage of parents/carers that agreed, or strongly agreed with the statement: I would be happy to give my child Infacort in the future.	Question 4 Percentage of parents/carers that agreed, or strongly agreed with the statement: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication.
Serum Cortisol Concentration up to 240 Minutes	575.8 nmol/L Geometric Coefficient of Variation 47.9	—	—	—

SECONDARY outcome

Timeframe: 240 minutes

Serum cortisol concentration 240 minutes after intake of study drug as determined by the central laboratory

Outcome measures

Measure	Infacort n=24 Participants Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone	Question 2 Percentage of parents/carers that agreed, or strongly agreed with the statement: My child showed a positive reaction after Infacort was given.	Question 3 Percentage of parents/carers that agreed, or strongly agreed with the statement: I would be happy to give my child Infacort in the future.	Question 4 Percentage of parents/carers that agreed, or strongly agreed with the statement: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication.
Serum Cortisol Concentration up to 6 Hours	60.1 nmol/L Geometric Coefficient of Variation 131.7	—	—	—

SECONDARY outcome

Timeframe: 1 minute

Population: Out of the total population, one parent/carer did not complete the palatability questionnaire and one parent/carer did not complete questions 3 and 4, and thus were excluded from the analysis of the relevant questions.

Palatability of the investigational product as determined by parent/carer responses to the following questions:

Question 1: My child found swallowing easy. Question 2: My child showed a positive reaction after Infacort was given. Question 3: I would be happy to give my child Infacort in the future. Question 4: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication.

Outcome measures

Measure	Infacort n=23 Participants Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone	Question 2 n=23 Participants Percentage of parents/carers that agreed, or strongly agreed with the statement: My child showed a positive reaction after Infacort was given.	Question 3 n=22 Participants Percentage of parents/carers that agreed, or strongly agreed with the statement: I would be happy to give my child Infacort in the future.	Question 4 n=22 Participants Percentage of parents/carers that agreed, or strongly agreed with the statement: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication.
Subject Assessment of Taste of the Product	19 Participants	15 Participants	21 Participants	21 Participants

SECONDARY outcome

Timeframe: 7-10 days

Incidence of serious adverse events (SAEs) and adverse events (AE).

Outcome measures

Measure	Infacort n=24 Participants Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone	Question 2 Percentage of parents/carers that agreed, or strongly agreed with the statement: My child showed a positive reaction after Infacort was given.	Question 3 Percentage of parents/carers that agreed, or strongly agreed with the statement: I would be happy to give my child Infacort in the future.	Question 4 Percentage of parents/carers that agreed, or strongly agreed with the statement: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication.
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) Treatment-Emergent	12 Adverse Events	—	—	—
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) Serious TEAEs	0 Adverse Events	—	—	—
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) Severe TEAEs	0 Adverse Events	—	—	—
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) Moderate TEAEs	2 Adverse Events	—	—	—
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) Mild TEAEs	10 Adverse Events	—	—	—

Adverse Events

Infacort

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Infacort n=24 participants at risk Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. Infacort®: dry granule formulation of hydrocortisone
Gastrointestinal disorders Diarrhoea	12.5% 3/24 • Number of events 3 Adverse Events will be recorded from the time of first intake of Infacort® until Visit 4. Serious Adverse Events (SAEs) will be recorded from the time of first intake of Infacort® until 7 days following administration of Infacort®. Any SAEs experienced after this 7-day period will be reported to the sponsor if the investigator suspects a causal relationship to the study drug.
Gastrointestinal disorders Vomiting	8.3% 2/24 • Number of events 2 Adverse Events will be recorded from the time of first intake of Infacort® until Visit 4. Serious Adverse Events (SAEs) will be recorded from the time of first intake of Infacort® until 7 days following administration of Infacort®. Any SAEs experienced after this 7-day period will be reported to the sponsor if the investigator suspects a causal relationship to the study drug.
Gastrointestinal disorders Infantile spitting up	4.2% 1/24 • Number of events 2 Adverse Events will be recorded from the time of first intake of Infacort® until Visit 4. Serious Adverse Events (SAEs) will be recorded from the time of first intake of Infacort® until 7 days following administration of Infacort®. Any SAEs experienced after this 7-day period will be reported to the sponsor if the investigator suspects a causal relationship to the study drug.
Skin and subcutaneous tissue disorders Rash	8.3% 2/24 • Number of events 2 Adverse Events will be recorded from the time of first intake of Infacort® until Visit 4. Serious Adverse Events (SAEs) will be recorded from the time of first intake of Infacort® until 7 days following administration of Infacort®. Any SAEs experienced after this 7-day period will be reported to the sponsor if the investigator suspects a causal relationship to the study drug.
Skin and subcutaneous tissue disorders Hyperhidrosis	4.2% 1/24 • Number of events 2 Adverse Events will be recorded from the time of first intake of Infacort® until Visit 4. Serious Adverse Events (SAEs) will be recorded from the time of first intake of Infacort® until 7 days following administration of Infacort®. Any SAEs experienced after this 7-day period will be reported to the sponsor if the investigator suspects a causal relationship to the study drug.
General disorders Fatigue	4.2% 1/24 • Number of events 1 Adverse Events will be recorded from the time of first intake of Infacort® until Visit 4. Serious Adverse Events (SAEs) will be recorded from the time of first intake of Infacort® until 7 days following administration of Infacort®. Any SAEs experienced after this 7-day period will be reported to the sponsor if the investigator suspects a causal relationship to the study drug.

Additional Information

Dr John Porter

Diurnal Limited

Email: info@diurnal.co.uk

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place