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Trial record 52 of 107 for:    "21-hydroxylase deficiency"

Extension Study for Patients Entered Into Study Infacort 003

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Adrenal Insufficiency
Intervention Drug: Infacort®
Enrollment 18
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Infacort
Hide Arm/Group Description

Infacort® granules

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). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.

Period Title: Overall Study
Started 18
Completed 12
Not Completed 6
Reason Not Completed
Withdrawal by Subject             6
Arm/Group Title Infacort
Hide Arm/Group Description

Infacort® granules

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). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.

Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
Subjects recruited were those who had satisfactorily completed study Infacort 003 and agreed to participate in study Infacort 004. The investigator ensured that all subjects who were treated in study Infacort 003 were invited to participate.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
<=18 years
18
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Days
Number Analyzed 18 participants
1021.6  (650.84)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
8
  44.4%
Male
10
  55.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
18
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
18
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Germany Number Analyzed 18 participants
18
Body Mass Index  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 18 participants
17.02  (2.083)
Body Surface Area  
Mean (Standard Deviation)
Unit of measure:  M^2
Number Analyzed 18 participants
0.582  (0.1803)
1.Primary Outcome
Title Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)
Hide Description 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.
Time Frame 29 months
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Infacort
Hide Arm/Group Description:

Infacort® granules

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). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.

Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: adverse events
Treatment-Emergent Adverse Events (TEAE) - Overall 193
Serious TEAEs 9
Severe TEAEs 0
Moderate TEAEs 42
Mild TEAEs 151
2.Secondary Outcome
Title Growth Velocity
Hide Description 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.
Time Frame 29 months
Hide Outcome Measure Data
Hide Analysis Population Description
Reference data for growth velocity is only available for patients <=6 years old at time of assessment. Therefore n=2 & not 4 for C1 at Month 11. Subject 001 was re-enrolled so no Visit 10 data available and, therefore, n=1 for C1 at Month 23.
Arm/Group Title Infacort
Hide Arm/Group Description:

Infacort® granules

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). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.

Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: Standard Deviation Score
visit 6 - 11th Month Number Analyzed 10 participants
0.6343  (2.51996)
Visit 10 - 23rd Month Number Analyzed 9 participants
0.9214  (1.88757)
3.Secondary Outcome
Title Cortisol Levels
Hide Description 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).
Time Frame 29 months
Hide Outcome Measure Data
Hide Analysis Population Description
The reason why n=14 at Visit 1 is because of no result for Subjects 005, 010, 017 & 018. N=12 at Final Visit due to 12 completers.
Arm/Group Title Infacort
Hide Arm/Group Description:

Infacort® granules

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). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.

Overall Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: nmol/L
Visit 1 Number Analyzed 14 participants
43.329  (48.3217)
Final Visit Number Analyzed 12 participants
22.022  (27.1323)
4.Secondary Outcome
Title Number of Participants Exhibiting a Change in Tanner Development Stage
Hide Description 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).
Time Frame 29 months
Hide Outcome Measure Data
Hide Analysis Population Description
'Count of Participants' data below represents the number of subjects that exhibited a change from baseline in their Tanner Development Stage.
Arm/Group Title Infacort
Hide Arm/Group Description:

Infacort® granules

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).

Overall Number of Participants Analyzed 18
Measure Type: Count of Participants
Unit of Measure: Participants
1
   5.6%
Time Frame Adverse events were recorded from the time of the first intake of Infacort in this study until the final study visit (i.e. 29 months).
Adverse Event Reporting Description Details of any adverse events were collected, including date of onset, end date, frequency, severity, seriousness, relationship to Infacort, action taken, and outcome. Any adverse event was followed, whenever possible, until it returned to the baseline condition or became stable with no further change expected.
 
Arm/Group Title Infacort
Hide Arm/Group Description

Infacort® granules

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). This was a non-randomised, open-label, single-group study; all subjects who participated received Infacort. One subject was initially withdrawn from the study, but subsequently re-enrolled.

All-Cause Mortality
Infacort
Affected / at Risk (%)
Total   0/18 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Infacort
Affected / at Risk (%) # Events
Total   3/18 (16.67%)    
Gastrointestinal disorders   
Vomiting  1  1/18 (5.56%)  2
Infections and infestations   
Erysipelas  1  1/18 (5.56%)  1
Gastroenteritis  1  2/18 (11.11%)  4
Urinary Tract Infection  1  1/18 (5.56%)  1
Pyelonephritis acute  1  1/18 (5.56%)  1
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Infacort
Affected / at Risk (%) # Events
Total   14/18 (77.78%)    
Gastrointestinal disorders   
Abdominal pain  1  1/18 (5.56%)  7
Abdominal pain upper  1  1/18 (5.56%)  4
Constipation  1  1/18 (5.56%)  1
Dental caries  1  1/18 (5.56%)  1
Diarrhoea  1  2/18 (11.11%)  6
Nausea  1  1/18 (5.56%)  3
Vomiting  1  6/18 (33.33%)  12
General disorders   
Pyrexia  1  10/18 (55.56%)  45
Infections and infestations   
Bronchitis  1  2/18 (11.11%)  6
Conjunctivitis  1  5/18 (27.78%)  6
Enterovirus infection  1  1/18 (5.56%)  1
Gastroenteritis  1  8/18 (44.44%)  11
Gastroenteritis viral  1  1/18 (5.56%)  1
Hand-foot-and-mouth disease  1  1/18 (5.56%)  1
Influenza  1  1/18 (5.56%)  1
Laryngitis  1  1/18 (5.56%)  1
Molluscum contagiosum  1  1/18 (5.56%)  1
Oral candidiasis  1  1/18 (5.56%)  1
Otitis media  1  1/18 (5.56%)  2
Otitis media viral  1  3/18 (16.67%)  3
Pharyngitis  1  2/18 (11.11%)  2
Pharyngotonsilitis  1  1/18 (5.56%)  1
Respiratory tract infection  1  2/18 (11.11%)  2
Rhinitis  1  2/18 (11.11%)  2
Roseola  1  1/18 (5.56%)  1
Tonsilitis  1  3/18 (16.67%)  3
Viral infection  1  6/18 (33.33%)  6
Viral upper respiratory tract infection  1  7/18 (38.89%)  21
Scarlet fever  1  1/18 (5.56%)  1
Upper respiratory tract infection  1  1/18 (5.56%)  1
Injury, poisoning and procedural complications   
Laceration  1  1/18 (5.56%)  1
Thermal burn  1  1/18 (5.56%)  1
Tooth injury  1  1/18 (5.56%)  1
Venomous sting  1  1/18 (5.56%)  1
Injury  1  1/18 (5.56%)  1
Scar  1  1/18 (5.56%)  1
Investigations   
Body temperature increased  1  2/18 (11.11%)  6
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/18 (5.56%)  1
Synovitis  1  1/18 (5.56%)  2
Scoliosis  1  1/18 (5.56%)  1
Nervous system disorders   
Headache  1  1/18 (5.56%)  3
Paraesthesia  1  1/18 (5.56%)  2
Reproductive system and breast disorders   
Vulval disorder  1  1/18 (5.56%)  1
Respiratory, thoracic and mediastinal disorders   
Pharyngeal erythema  1  1/18 (5.56%)  1
Skin and subcutaneous tissue disorders   
Eczema  1  1/18 (5.56%)  1
Urticaria  1  1/18 (5.56%)  1
Dermatitis allergic  1  1/18 (5.56%)  1
Surgical and medical procedures   
Genitourinary operation  1  2/18 (11.11%)  2
Circumcision  1  1/18 (5.56%)  1
Vascular disorders   
Secondary hypertension  1  1/18 (5.56%)  1
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
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.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Information Line
Organization: Diurnal
Phone: +44 (0) 2920 682069
EMail: info@diurnal.co.uk
Layout table for additonal information
Responsible Party: Diurnal Limited
ClinicalTrials.gov Identifier: NCT02733367     History of Changes
Other Study ID Numbers: Infacort 004
First Submitted: March 16, 2016
First Posted: April 11, 2016
Results First Submitted: August 9, 2019
Results First Posted: September 13, 2019
Last Update Posted: November 5, 2019