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Trial record 9 of 12 for:    NICHD Cushing's

Prospective, Open-Label, Multicenter, International Study of Mifepristone for Symptomatic Treatment of Cushing's Syndrome Caused by Ectopic Adrenal Corticotrophin Hormone (ACTH) Secretion

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ClinicalTrials.gov Identifier: NCT00422201
Recruitment Status : Terminated
First Posted : January 15, 2007
Results First Posted : October 14, 2013
Last Update Posted : November 11, 2013
Sponsor:
Information provided by (Responsible Party):
HRA Pharma

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cushing's Syndrome
Intervention Drug: Mifepristone
Enrollment 18
Recruitment Details 18 patients recruited. Recruitment terminated.
Pre-assignment Details  
Arm/Group Title Single Arm Mifepristone
Hide Arm/Group Description

Study medication was to be administered at a total daily dose of 600 mg (given as one 200 mg tablet tid, per os) starting on the day of inclusion.

This dose was to be maintained during the whole study except in case of suspicion of adrenal insufficiency, in which case the dose was temporally stopped for 2 to 3 days and restarted at a lower dose of 400 mg daily until the end of the study.

Period Title: Overall Study
Started 18
Completed 7
Not Completed 11
Arm/Group Title Mifepristone
Hide Arm/Group Description

Single arm. Study medication was to be administered at a total daily dose of 600 mg (given as one 200 mg tablet tid, per os) starting on the day of inclusion.

This dose was to be maintained during the whole study except in case of suspicion of adrenal insufficiency, in which case the dose was temporally stopped for 2 to 3 days and restarted at a lower dose of 400 mg daily until the end of the study.

Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
<=18 years
0
   0.0%
Between 18 and 65 years
16
  88.9%
>=65 years
2
  11.1%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants
53.9  (12.48)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
10
  55.6%
Male
8
  44.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 18 participants
France 2
United States 7
Netherlands 6
Germany 1
Italy 2
1.Primary Outcome
Title Glycemic Disorders Improved or Normalized
Hide Description

Criteria for improvement or normalization of glycemic disorders:

A. For diabetic patients (known or diagnosed at pre-inclusion visit)

  • Decrease in HbA1c > 0.3% B. For patients with IGT
  • Normalization of OGTT (2-hour glucose plasma level after 75 g OGTT < 7.8 mmol/L (140 mg/dL) D. For patients with IFG

If impaired fasting glucose is also associated with impaired glucose tolerance during OGTT at pre-inclusion:

- Normalization of OGTT (2-hour glucose plasma level after 75 g OGTT < 7.8 mmol/L (140 mg/dL)

If impaired fasting glycemia is associated with normal OGTT at pre-inclusion (except at T0):

- Normalization of fasting plasma glucose (fasting plasma glucose < 5.5 mmol/L (100 mg/dL)

Time Frame 8 weeks at steady dose
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
18 patients were recruited. 7 completed the study but only 3 according to the last protocol version (in which primary efficacy criteria were changed), so only these 3 patients were to be analysed
Arm/Group Title Mifepristone
Hide Arm/Group Description:

Single arm. Study medication was administered at a total daily dose of 600 mg (given as one 200 mg tablet tid, per os) starting on the day of inclusion.

This dose was to be maintained during the whole study except in case of suspicion of adrenal insufficiency, in which case the dose was temporally stopped for 2 to 3 days and restarted at a lower dose of 400 mg daily until the end of the study.

Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
2
2.Secondary Outcome
Title Features of Cushing's Syndrome
Hide Description [Not Specified]
Time Frame 8 weeks at steady dose
Outcome Measure Data Not Reported
Time Frame Adverse events were collected from enrollment to the end of the study
Adverse Event Reporting Description Number of patients at risk corresponds to the number of patients who have received study treatment. Among the 18 patients enrolled, 17 received study treament and are considered in the set of population at risk.
 
Arm/Group Title Mifepristone
Hide Arm/Group Description

Single arm. Study medication was to be administered at a total daily dose of 600 mg (given as one 200 mg tablet tid, per os) starting on the day of inclusion.

This dose was to be maintained during the whole study except in case of suspicion of adrenal insufficiency, in which case the dose was temporally stopped for 2 to 3 days and restarted at a lower dose of 400 mg daily until the end of the study.

All-Cause Mortality
Mifepristone
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Mifepristone
Affected / at Risk (%) # Events
Total   9/17 (52.94%)    
Cardiac disorders   
Myocardial infarction  1/17 (5.88%)  1
Endocrine disorders   
Adrenal insufficiency  1/17 (5.88%)  1
Gastrointestinal disorders   
Nausea  1/17 (5.88%)  1
General disorders   
Oedema  1/17 (5.88%)  1
Fatigue  1/17 (5.88%)  1
Infections and infestations   
Abscess limb  1/17 (5.88%)  2
Pneumocystis jiroveci  2/17 (11.76%)  2
Metabolism and nutrition disorders   
Hyperkalaemia  1/17 (5.88%)  1
Hypokalaemia  2/17 (11.76%)  2
Renal and urinary disorders   
Renal acute failure  1/17 (5.88%)  1
Renal impairment  1/17 (5.88%)  1
Reproductive system and breast disorders   
Uterine polyp  1/17 (5.88%)  1
Respiratory, thoracic and mediastinal disorders   
Pulmonary embolism  1/17 (5.88%)  1
Vascular disorders   
Hypotension  1/17 (5.88%)  1
1
Term from vocabulary, MedDRA 15.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Mifepristone
Affected / at Risk (%) # Events
Total   17/17 (100.00%)    
Blood and lymphatic system disorders   
Anaemia  3/17 (17.65%)  4
Thrombocytopenia  1/17 (5.88%)  1
Cardiac disorders   
Palpitations  1/17 (5.88%)  1
Endocrine disorders   
Adrenal insuffisiency  6/17 (35.29%)  14
Steroid withdrawal syndrome  1/17 (5.88%)  2
Hypothyroidism  1/17 (5.88%)  1
Eye disorders   
Diplopia  1/17 (5.88%)  1
Gastrointestinal disorders   
Nausea  7/17 (41.18%)  30
vomiting  4/17 (23.53%)  11
Diarrhoea  1/17 (5.88%)  2
Dry mouth  1/17 (5.88%)  1
General disorders   
Fatigue  7/17 (41.18%)  21
Pyrexia  3/17 (17.65%)  5
Oedema peripheral  2/17 (11.76%)  4
Oedema  2/17 (11.76%)  2
pain  1/17 (5.88%)  1
Infections and infestations   
Labyrinthitis  1/17 (5.88%)  1
Nasopharyngitis  1/17 (5.88%)  1
Parotitis  1/17 (5.88%)  1
Pneumonia  1/17 (5.88%)  1
Sputum purulent  1/17 (5.88%)  1
Upper respiratory tract infection  1/17 (5.88%)  1
Investigations   
C-reactive protein increased  3/17 (17.65%)  3
Blood creatinine increased  2/17 (11.76%)  3
Protein total decrased  2/17 (11.76%)  2
Blood albumin decreased  1/17 (5.88%)  2
Blood corticotrophin increased  1/17 (5.88%)  2
Blood cortisol increased  1/17 (5.88%)  2
Blood urea increased  1/17 (5.88%)  2
High density lipoprotein decreased  1/17 (5.88%)  2
Alanine aminotransferase increased  1/17 (5.88%) 
Blood pressure decreased  1/17 (5.88%)  1
Blood thyroid stimulating hormone increased  1/17 (5.88%)  1
Cortisol free urine  1/17 (5.88%)  1
Hepatic enzyme increased  1/17 (5.88%)  1
Laboratory test abnormal  1/17 (5.88%)  1
Low density lipoprotein increased  1/17 (5.88%)  1
Osteocalcin increased  1/17 (5.88%)  1
Proteinuria  1/17 (5.88%)  1
Transaminases increased  1/17 (5.88%)  1
urine potassium increased  1/17 (5.88%)  1
urine sodium increased  1/17 (5.88%)  1
Weight increased  1/17 (5.88%)  1
Metabolism and nutrition disorders   
Hypokalaemia  9/17 (52.94%)  29
Decreased apetite  4/17 (23.53%)  8
Hyperkalaemia  1/17 (5.88%)  3
Dehydratation  1/17 (5.88%)  2
Hypernatremia  1/17 (5.88%)  1
Hypertriglyceridaemia  1/17 (5.88%)  1
Hypocalcaemia  1/17 (5.88%)  1
Hypomagnesaemia  1/17 (5.88%)  1
Hyponatraemia  1/17 (5.88%)  1
Musculoskeletal and connective tissue disorders   
Back pain  2/17 (11.76%)  2
Muscular weakness  1/17 (5.88%)  1
osteoarthritis  1/17 (5.88%)  1
Pain in jaw  1/17 (5.88%)  1
Nervous system disorders   
Headache  5/17 (29.41%)  25
Tremor  2/17 (11.76%)  2
Ageusia  1/17 (5.88%)  1
Burning sensation  1/17 (5.88%)  1
Dizziness  1/17 (5.88%)  1
Hypersomnia  1/17 (5.88%)  1
Memory impairment  1/17 (5.88%)  1
Somnolence  1/17 (5.88%)  1
Psychiatric disorders   
Depression  1/17 (5.88%)  1
Renal and urinary disorders   
Microalbuminuria  1/17 (5.88%)  3
Nocturia  1/17 (5.88%)  2
Renal failure acute  1/17 (5.88%)  1
Haematuria  1/17 (5.88%)  1
Micturition frequency increased  1/17 (5.88%)  1
Pollakiuria  1/17 (5.88%)  1
Reproductive system and breast disorders   
Endometrial hyperplasia  1/17 (5.88%)  1
Endometrial hypertrophy  1/17 (5.88%)  1
Gynaecomastia  1/17 (5.88%)  1
Respiratory, thoracic and mediastinal disorders   
Chest pain  1/17 (5.88%)  1
Cough  1/17 (5.88%)  1
Dyspnoea  1/17 (5.88%)  1
oropharyngeal pain  1/17 (5.88%)  1
Rhinorrhoea  1/17 (5.88%)  1
Skin and subcutaneous tissue disorders   
Alopecia  1/17 (5.88%)  1
Pigmentation disorder  1/17 (5.88%)  1
Rash  1/17 (5.88%)  1
Vascular disorders   
Hypotension  1/17 (5.88%)  1
Orthostatic hypotension  2/17 (11.76%)  2
Hypertension  1/17 (5.88%)  1
1
Term from vocabulary, MedDRA 15.0
As a consequence of the premature study end, analyses performed were primarily descriptive due to the reduced number of enrolled and completed patients.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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
Name/Title: Head of Clinical Research
Organization: HRA Pharma
Phone: 0033140331130
Responsible Party: HRA Pharma
ClinicalTrials.gov Identifier: NCT00422201     History of Changes
Other Study ID Numbers: 070008
07-CH-0008
First Submitted: January 12, 2007
First Posted: January 15, 2007
Results First Submitted: August 8, 2013
Results First Posted: October 14, 2013
Last Update Posted: November 11, 2013