Extension Study for Patients Entered Into Study Infacort 003

NCT02733367 | Completed Phase 3 Results DMC

• 1 other identifier: Infacort 004
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia who had completed study Infacort 003. All subjects who had satisfactorily completed study Infacort 003 were offered the opportunity to take part in Infacort 004.

Conditions

Adrenal Insufficiency

Outcome Measures

Primary Outcomes (1)

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

  The primary endpoint was the nature and occurrence of serious adverse events (SAEs) and adverse events (AEs) observed throughout the study. AEs were recorded from the time of the first intake of Infacort until the final visit.

  29 months

Secondary Outcomes (3)

• Growth Velocity

  29 months

• Cortisol Levels

  29 months

• Number of Participants Exhibiting a Change in Tanner Development Stage

  29 months

Study Arms (1)

Infacort

EXPERIMENTAL

Infacort® granules

Drug: Infacort®

Interventions

Infacort® DRUG

Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg).

Infacort

Eligibility Criteria

• Age: 1 Month - 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 (AI) 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. In addition, the parents/carers must be able to understand and give written Informed Consent for this extension study.

You may not qualify if:

• Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)
• Inability of the child to take oral therapy
• Any surgical or medical condition that 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 in a dependent relationship with the Investigator or the Sponsor

Sponsors & Collaborators

• Neurocrine UK Limited: lead

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

Limitations and Caveats

This study was designed as a safety study to evaluate the long-term use of Infacort in routine clinical practice. Efficacy results should be viewed as exploratory and interpreted with care.

Results Point of Contact

• Title: Information Line
• Organization: Diurnal

info@diurnal.co.uk

+44 (0) 2920 682069

Study Officials

• Wiegand

  Charite University, Berlin, Germany

  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: April 11, 2016
• Study Start: March 4, 2016
• Primary Completion: August 10, 2018
• Study Completion: August 10, 2018
• Last Updated: November 5, 2019
• Results First Posted: September 13, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)