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Danazol for Genetic Bone Marrow and Lung Disorders

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ClinicalTrials.gov Identifier: NCT01441037
Recruitment Status : Completed
First Posted : September 27, 2011
Results First Posted : July 11, 2018
Last Update Posted : August 15, 2018
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Aplastic Anemia
Intervention Drug: Danazol
Enrollment 27
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Danazol
Hide Arm/Group Description Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
Period Title: Overall Study
Started 27
Completed 27
Not Completed 0
Arm/Group Title Danazol
Hide Arm/Group Description Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
Overall Number of Baseline Participants 27
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
<=18 years
3
  11.1%
Between 18 and 65 years
22
  81.5%
>=65 years
2
   7.4%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
Female
15
  55.6%
Male
12
  44.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
Hispanic or Latino
3
  11.1%
Not Hispanic or Latino
22
  81.5%
Unknown or Not Reported
2
   7.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
   7.4%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   3.7%
White
18
  66.7%
More than one race
3
  11.1%
Unknown or Not Reported
3
  11.1%
1.Primary Outcome
Title Number of Patients Having Attenuation of Accelerated Telomere Attrition
Hide Description The primary efficacy end point was a 20% reduction in the annual rate of telomere attrition measured at 24 months. The biologic response at 24 months, was defined as a reduction in the telomere length attrition rate to 96 bp per year or less. The normal rate of telomere loss of approximately 60 bp per year. Telomere length was determined with a semiautomated, Clinical Laboratory Improvement Amendments (CLIA)–approved real-time quantitative PCR (qPCR) assay performed in triplicate and validated for human cells
Time Frame 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
The analyses included only those subjects who took Danazol
Arm/Group Title Danazol
Hide Arm/Group Description:
Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
Overall Number of Participants Analyzed 27
Measure Type: Count of Participants
Unit of Measure: Participants
12
  44.4%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Danazol
Hide Arm/Group Description Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
All-Cause Mortality
Danazol
Affected / at Risk (%)
Total   0/27 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Danazol
Affected / at Risk (%)
Total   3/27 (11.11%) 
Gastrointestinal disorders   
Diarrhoea   1/27 (3.70%) 
General disorders   
Joint swelling   1/27 (3.70%) 
Oedema peripheral   1/27 (3.70%) 
Injury, poisoning and procedural complications   
Femur fracture   1/27 (3.70%) 
Respiratory, thoracic and mediastinal disorders   
Pneumocystis jirovecii pneumonia   1/27 (3.70%) 
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%
Danazol
Affected / at Risk (%)
Total   24/27 (88.89%) 
Blood and lymphatic system disorders   
Anaemia   2/27 (7.41%) 
Pallor   1/27 (3.70%) 
Cardiac disorders   
Palpitations   1/27 (3.70%) 
Tachycardia   1/27 (3.70%) 
Ear and labyrinth disorders   
Otitis externa   1/27 (3.70%) 
Eye disorders   
Scleritis   1/27 (3.70%) 
Vitreous floaters   1/27 (3.70%) 
Gastrointestinal disorders   
Abdominal distension   2/27 (7.41%) 
Abdominal pain upper   1/27 (3.70%) 
Constipation   3/27 (11.11%) 
Decreased appetite   1/27 (3.70%) 
Dental caries   1/27 (3.70%) 
Diarrhoea   1/27 (3.70%) 
Gastrointestinal pain   1/27 (3.70%) 
Infection   1/27 (3.70%) 
Nausea   2/27 (7.41%) 
Proctalgia   1/27 (3.70%) 
Rectal haemorrhage   3/27 (11.11%) 
Stomatitis   1/27 (3.70%) 
Vomiting   2/27 (7.41%) 
General disorders   
Asthenia   2/27 (7.41%) 
Back pain   1/27 (3.70%) 
Cough   1/27 (3.70%) 
Face oedema   1/27 (3.70%) 
Fatigue   12/27 (44.44%) 
Feeling hot   2/27 (7.41%) 
Influenza like illness   1/27 (3.70%) 
Irritability   1/27 (3.70%) 
Oedema   2/27 (7.41%) 
Oedema peripheral   1/27 (3.70%) 
Pain   1/27 (3.70%) 
Peripheral swelling   1/27 (3.70%) 
Pyrexia   1/27 (3.70%) 
Toothache   1/27 (3.70%) 
Upper respiratory tract infection   1/27 (3.70%) 
Immune system disorders   
Sinus congestion   1/27 (3.70%) 
Infections and infestations   
Bronchitis   1/27 (3.70%) 
Cellulitis   1/27 (3.70%) 
Cystitis   1/27 (3.70%) 
Sinusitis   2/27 (7.41%) 
Tooth disorder   1/27 (3.70%) 
Upper respiratory tract infection   6/27 (22.22%) 
Urinary tract infection   2/27 (7.41%) 
Viral infection   1/27 (3.70%) 
Injury, poisoning and procedural complications   
Clavicle fracture   1/27 (3.70%) 
Contusion   2/27 (7.41%) 
Increased tendency to bruise   1/27 (3.70%) 
Lower limb fracture   1/27 (3.70%) 
Spinal fracture   1/27 (3.70%) 
Umbilical hernia   1/27 (3.70%) 
Investigations   
Alanine aminotransferase increased   1/27 (3.70%) 
Blood creatine phosphokinase increased   1/27 (3.70%) 
Blood creatinine increased   1/27 (3.70%) 
Neutrophil count decreased   2/27 (7.41%) 
Platelet count decreased   2/27 (7.41%) 
Transaminases increased   1/27 (3.70%) 
Weight increased   4/27 (14.81%) 
Metabolism and nutrition disorders   
Iron overload   1/27 (3.70%) 
Musculoskeletal and connective tissue disorders   
Arthralgia   1/27 (3.70%) 
Back pain   2/27 (7.41%) 
Chest discomfort   1/27 (3.70%) 
Joint effusion   1/27 (3.70%) 
Joint range of motion decreased   1/27 (3.70%) 
Joint swelling   1/27 (3.70%) 
Muscle hypertrophy   1/27 (3.70%) 
Muscle spasms   9/27 (33.33%) 
Myalgia   1/27 (3.70%) 
Pain   1/27 (3.70%) 
Pain in extremity   1/27 (3.70%) 
Nervous system disorders   
Disturbance in attention   1/27 (3.70%) 
Dizziness   5/27 (18.52%) 
Headache   4/27 (14.81%) 
Migraine   1/27 (3.70%) 
Seizure   1/27 (3.70%) 
Psychiatric disorders   
Depression   3/27 (11.11%) 
Hypersomnia   1/27 (3.70%) 
Renal and urinary disorders   
Nephrolithiasis   1/27 (3.70%) 
Pollakiuria   1/27 (3.70%) 
Reproductive system and breast disorders   
Libido increased   1/27 (3.70%) 
Vaginal discharge   1/27 (3.70%) 
Respiratory, thoracic and mediastinal disorders   
Cough   6/27 (22.22%) 
Dysphonia   1/27 (3.70%) 
Dyspnoea   3/27 (11.11%) 
Dyspnoea exertional   4/27 (14.81%) 
Epistaxis   2/27 (7.41%) 
Nasal congestion   2/27 (7.41%) 
Oropharyngeal pain   2/27 (7.41%) 
Pneumonia   1/27 (3.70%) 
Respiratory tract congestion   2/27 (7.41%) 
Rhinitis allergic   2/27 (7.41%) 
Sinusitis   2/27 (7.41%) 
Tachypnoea   1/27 (3.70%) 
Upper respiratory tract infection   1/27 (3.70%) 
Skin and subcutaneous tissue disorders   
Acne   1/27 (3.70%) 
Alopecia   4/27 (14.81%) 
Folliculitis   1/27 (3.70%) 
Hirsutism   1/27 (3.70%) 
Hot flush   1/27 (3.70%) 
Hypertrichosis   1/27 (3.70%) 
Night sweats   1/27 (3.70%) 
Petechiae   2/27 (7.41%) 
Rash   2/27 (7.41%) 
Rash maculo-papular   1/27 (3.70%) 
Rash pruritic   1/27 (3.70%) 
Skin hyperpigmentation   2/27 (7.41%) 
Skin hypopigmentation   1/27 (3.70%) 
Skin lesion   1/27 (3.70%) 
Subcutaneous abscess   1/27 (3.70%) 
Vascular disorders   
Haemangioma   1/27 (3.70%) 
Haematoma   1/27 (3.70%) 
Indicates events were collected by systematic assessment
After 27 patients were enrolled, the study was halted early, because telomere attrition was reduced in all 12 patients who could be evaluated for the primary end point.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Young, Neal
Organization: National Heart Lung and Blood Institute
Phone: +1 301 496 5093
EMail: youngns@mail.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01441037     History of Changes
Other Study ID Numbers: 110209
11-H-0209
First Submitted: September 24, 2011
First Posted: September 27, 2011
Results First Submitted: June 14, 2018
Results First Posted: July 11, 2018
Last Update Posted: August 15, 2018