Treatment of Adrenal Insufficiency in Children

NCT02720952 | Completed Phase 3 Results DMC

• 1 other identifier: Infacort-003
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The study will be conducted in a total of 24 subjects before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism. The study will consist of three consecutive cohorts. Cohort 1 will include 12 subjects aged between 2 and \< 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to \<2 years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only if again no safety concerns emerge, then 6 neonates aged from birth to \<28 days will be enrolled (Cohort 3). The decision to continue after each cohort will be based on the recommendation of an Independent Data Monitoring Committee (IDMC).

Conditions

Adrenal Insufficiency

Outcome Measures

Primary Outcomes (1)

• Serum Cortisol Concentration up to 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.

  240 minutes

Secondary Outcomes (3)

• Serum Cortisol Concentration up to 6 Hours

  240 minutes

• Subject Assessment of Taste of the Product

  1 minute

• Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE)

  7-10 days

Study Arms (1)

Infacort

EXPERIMENTAL

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.

Drug: Infacort®

Interventions

Infacort® DRUG

dry granule formulation of hydrocortisone

Infacort

Eligibility Criteria

• Age: 1 Week - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male and female children less than 6 years of age.
• A diagnosis of adrenal insufficiency as confirmed by an inappropriately low cortisol usually with other supporting tests.
• Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
• Adequately hydrated and nourished.
• Ability of parents/carers to understand and give written Informed Consent

You may not qualify if:

• Clinically evident acute adrenal insufficiency (adrenal crisis).
• Inability of the child to take oral therapy.
• Concomitant therapy (other than that required to treat adrenal insufficiency, Vitamin D, Fluoride, Thyroxine and growth hormone).
• Subjects with clinical signs of acute infection or fever on Day 1.
• Any surgical or medical condition which in the opinion of the investigator may place the subject at higher risk from his/her participation in the study.
• Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons.
• Subjects who are dependent on the investigator or the sponsor.

Sponsors & Collaborators

• Neurocrine UK Limited: lead
• Treatment of Adrenal Insufficiency in Neonates consortium (TAIN): collaborator

Study Sites (1)

Charité-Universitätsmedizin Berlin, CVK

Berlin, 13353, Germany

Location

MeSH Terms

Conditions

Adrenal Insufficiency

Condition Hierarchy (Ancestors)

Adrenal Gland Diseases; Endocrine System Diseases

Results Point of Contact

• Title: Dr John Porter
• Organization: Diurnal Limited

info@diurnal.co.uk

Study Officials

• Susanna Wiegand, MD

  Charité-Universitätsmedizin Berlin, CVK

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2016
• First Posted: March 28, 2016
• Study Start: March 1, 2015
• Primary Completion: July 1, 2016
• Study Completion: July 1, 2016
• Last Updated: May 4, 2022
• Results First Posted: April 11, 2017

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)