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Extension Study for Patients Entered Into Study Infacort 003

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ClinicalTrials.gov Identifier: NCT02733367
Recruitment Status : Completed
First Posted : April 11, 2016
Results First Posted : September 13, 2019
Last Update Posted : November 5, 2019
Sponsor:
Information provided by (Responsible Party):
Diurnal Limited

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.

Condition or disease Intervention/treatment Phase
Adrenal Insufficiency Drug: Infacort® Phase 3

Detailed Description:
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 AI who had completed study Infacort 003 (EudraCT number 2014-002265-30). All subjects who had satisfactorily completed study Infacort 003 wiere offered the opportunity to participate in study Infacort 004 at or after their final visit of study Infacort 003. Subjects received the usual clinically-appropriate dose (since bioequivalence has been demonstrated with conventional hydrocortisone), as determined by the Investigator, which was administered according to usual clinical practice - generally 3 or 4 times a day. Subjects could continue to be treated in this study until they met the study withdrawal criteria, until Infacort® was commercially available locally (which has now been achieved), or until the Sponsor decided to discontinue the study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label, Long-term Follow-up of Safety and Biochemical Disease Control of Infacort® in Neonates, Infants and Children With Congenital Adrenal Hyperplasia and Adrenal Insufficiency Previously Enrolled in the Infacort 003 Study
Actual Study Start Date : March 4, 2016
Actual Primary Completion Date : August 10, 2018
Actual Study Completion Date : August 10, 2018


Arm Intervention/treatment
Experimental: Infacort
Infacort® granules
Drug: Infacort®
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).




Primary Outcome Measures :
  1. Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs) [ Time Frame: 29 months ]
    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.


Secondary Outcome Measures :
  1. Growth Velocity [ Time Frame: 29 months ]
    Growth velocity standard deviation score (SDS). Body height/length (cm) was obtained at each visit by specially trained paediatric endocrine nurses or physicians using standard calibrated auxological methods.

  2. Cortisol Levels [ Time Frame: 29 months ]
    Cortisol levels measured from dried blood spots. The dried blood spots were analysed for multi-steroids, including cortisol (all subjects). Blood spot absolute laboratory values for the safety population are presented. A dried blood spot sample was collected at the initial and final visits, every month for the first 2 months of the study and thereafter every 6 months (unless required after 3 months).

  3. Number of Participants Exhibiting a Change in Tanner Development Stage [ Time Frame: 29 months ]
    The Tanner Development Stage was assessed as an additional analysis in this study. All assessments (breast, genitalia, and pubic hair) were Grade 1 (pre-pubertal) at baseline, with only 1 subject (in Cohort 2) showing a change during the study. Subject 018 showed progression to Grade 2 in the pubic hair category (sparse, pigmented hair mainly on labia).



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Ages Eligible for Study:   1 Month to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects successfully completing study Infacort 003, whose inclusion criteria were:

  1. Male and female children less than 6 years of age.
  2. A diagnosis of adrenal insufficiency (AI) as confirmed by an inappropriately low cortisol usually with other supporting tests.
  3. Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
  4. Adequately hydrated and nourished. In addition, the parents/carers must be able to understand and give written Informed Consent for this extension study.

Exclusion Criteria:

  1. Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)
  2. Inability of the child to take oral therapy
  3. Subjects with clinical signs of acute infection or fever on inclusion (Note: the subject can be re-evaluated for eligibility once the episode is over)
  4. 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
  5. Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons
  6. Subjects who are in a dependent relationship with the Investigator or the Sponsor

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02733367


Locations
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Germany
Charité-Universitätsmedizin Berlin, CVK
Berlin, Germany, 13353
Sponsors and Collaborators
Diurnal Limited
Investigators
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Principal Investigator: Wiegand Charite University, Berlin, Germany
  Study Documents (Full-Text)

Documents provided by Diurnal Limited:
Study Protocol  [PDF] July 20, 2017
Statistical Analysis Plan  [PDF] February 1, 2017


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Responsible Party: Diurnal Limited
ClinicalTrials.gov Identifier: NCT02733367     History of Changes
Other Study ID Numbers: Infacort 004
First Posted: April 11, 2016    Key Record Dates
Results First Posted: September 13, 2019
Last Update Posted: November 5, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases