Trial Outcomes & Findings for Danazol for Genetic Bone Marrow and Lung Disorders (NCT NCT01441037)
NCT ID: NCT01441037
Last Updated: 2018-08-15
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
27 participants
Primary outcome timeframe
24 months
Results posted on
2018-08-15
Participant Flow
Participant milestones
| Measure |
Danazol
Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
|
|---|---|
|
Overall Study
STARTED
|
27
|
|
Overall Study
COMPLETED
|
27
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Danazol for Genetic Bone Marrow and Lung Disorders
Baseline characteristics by cohort
| Measure |
Danazol
n=27 Participants
Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
|
|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
22 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
22 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: 24 monthsPopulation: The analyses included only those subjects who took Danazol
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
Outcome measures
| Measure |
Danazol
n=27 Participants
Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
|
|---|---|
|
Number of Patients Having Attenuation of Accelerated Telomere Attrition
|
12 Participants
|
Adverse Events
Danazol
Serious events: 3 serious events
Other events: 24 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Danazol
n=27 participants at risk
Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
3.7%
1/27
|
|
General disorders
Joint swelling
|
3.7%
1/27
|
|
General disorders
Oedema peripheral
|
3.7%
1/27
|
|
Injury, poisoning and procedural complications
Femur fracture
|
3.7%
1/27
|
|
Respiratory, thoracic and mediastinal disorders
Pneumocystis jirovecii pneumonia
|
3.7%
1/27
|
Other adverse events
| Measure |
Danazol
n=27 participants at risk
Oral administration of Danazol (800 mg daily divided into two doses per day) to be given for 2 years
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.4%
2/27
|
|
Blood and lymphatic system disorders
Pallor
|
3.7%
1/27
|
|
Cardiac disorders
Palpitations
|
3.7%
1/27
|
|
Cardiac disorders
Tachycardia
|
3.7%
1/27
|
|
Ear and labyrinth disorders
Otitis externa
|
3.7%
1/27
|
|
Eye disorders
Scleritis
|
3.7%
1/27
|
|
Eye disorders
Vitreous floaters
|
3.7%
1/27
|
|
Gastrointestinal disorders
Abdominal distension
|
7.4%
2/27
|
|
Gastrointestinal disorders
Abdominal pain upper
|
3.7%
1/27
|
|
Gastrointestinal disorders
Constipation
|
11.1%
3/27
|
|
Gastrointestinal disorders
Decreased appetite
|
3.7%
1/27
|
|
Gastrointestinal disorders
Dental caries
|
3.7%
1/27
|
|
Gastrointestinal disorders
Diarrhoea
|
3.7%
1/27
|
|
Gastrointestinal disorders
Gastrointestinal pain
|
3.7%
1/27
|
|
Gastrointestinal disorders
Infection
|
3.7%
1/27
|
|
Gastrointestinal disorders
Nausea
|
7.4%
2/27
|
|
Gastrointestinal disorders
Proctalgia
|
3.7%
1/27
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
11.1%
3/27
|
|
Gastrointestinal disorders
Stomatitis
|
3.7%
1/27
|
|
Gastrointestinal disorders
Vomiting
|
7.4%
2/27
|
|
General disorders
Asthenia
|
7.4%
2/27
|
|
General disorders
Back pain
|
3.7%
1/27
|
|
General disorders
Cough
|
3.7%
1/27
|
|
General disorders
Face oedema
|
3.7%
1/27
|
|
General disorders
Fatigue
|
44.4%
12/27
|
|
General disorders
Feeling hot
|
7.4%
2/27
|
|
General disorders
Influenza like illness
|
3.7%
1/27
|
|
General disorders
Irritability
|
3.7%
1/27
|
|
General disorders
Oedema
|
7.4%
2/27
|
|
General disorders
Oedema peripheral
|
3.7%
1/27
|
|
General disorders
Pain
|
3.7%
1/27
|
|
General disorders
Peripheral swelling
|
3.7%
1/27
|
|
General disorders
Pyrexia
|
3.7%
1/27
|
|
General disorders
Toothache
|
3.7%
1/27
|
|
General disorders
Upper respiratory tract infection
|
3.7%
1/27
|
|
Immune system disorders
Sinus congestion
|
3.7%
1/27
|
|
Infections and infestations
Bronchitis
|
3.7%
1/27
|
|
Infections and infestations
Cellulitis
|
3.7%
1/27
|
|
Infections and infestations
Cystitis
|
3.7%
1/27
|
|
Infections and infestations
Sinusitis
|
7.4%
2/27
|
|
Infections and infestations
Tooth disorder
|
3.7%
1/27
|
|
Infections and infestations
Upper respiratory tract infection
|
22.2%
6/27
|
|
Infections and infestations
Urinary tract infection
|
7.4%
2/27
|
|
Infections and infestations
Viral infection
|
3.7%
1/27
|
|
Injury, poisoning and procedural complications
Clavicle fracture
|
3.7%
1/27
|
|
Injury, poisoning and procedural complications
Contusion
|
7.4%
2/27
|
|
Injury, poisoning and procedural complications
Increased tendency to bruise
|
3.7%
1/27
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
3.7%
1/27
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
3.7%
1/27
|
|
Injury, poisoning and procedural complications
Umbilical hernia
|
3.7%
1/27
|
|
Investigations
Alanine aminotransferase increased
|
3.7%
1/27
|
|
Investigations
Blood creatine phosphokinase increased
|
3.7%
1/27
|
|
Investigations
Blood creatinine increased
|
3.7%
1/27
|
|
Investigations
Neutrophil count decreased
|
7.4%
2/27
|
|
Investigations
Platelet count decreased
|
7.4%
2/27
|
|
Investigations
Transaminases increased
|
3.7%
1/27
|
|
Investigations
Weight increased
|
14.8%
4/27
|
|
Metabolism and nutrition disorders
Iron overload
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.4%
2/27
|
|
Musculoskeletal and connective tissue disorders
Chest discomfort
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Joint range of motion decreased
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Muscle hypertrophy
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
33.3%
9/27
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Pain
|
3.7%
1/27
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
3.7%
1/27
|
|
Nervous system disorders
Disturbance in attention
|
3.7%
1/27
|
|
Nervous system disorders
Dizziness
|
18.5%
5/27
|
|
Nervous system disorders
Headache
|
14.8%
4/27
|
|
Nervous system disorders
Migraine
|
3.7%
1/27
|
|
Nervous system disorders
Seizure
|
3.7%
1/27
|
|
Psychiatric disorders
Depression
|
11.1%
3/27
|
|
Psychiatric disorders
Hypersomnia
|
3.7%
1/27
|
|
Renal and urinary disorders
Nephrolithiasis
|
3.7%
1/27
|
|
Renal and urinary disorders
Pollakiuria
|
3.7%
1/27
|
|
Reproductive system and breast disorders
Libido increased
|
3.7%
1/27
|
|
Reproductive system and breast disorders
Vaginal discharge
|
3.7%
1/27
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
22.2%
6/27
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
3.7%
1/27
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.1%
3/27
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
14.8%
4/27
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.4%
2/27
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
7.4%
2/27
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.4%
2/27
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
3.7%
1/27
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
7.4%
2/27
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
7.4%
2/27
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
7.4%
2/27
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
3.7%
1/27
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract infection
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Acne
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
14.8%
4/27
|
|
Skin and subcutaneous tissue disorders
Folliculitis
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Hirsutism
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Hot flush
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Hypertrichosis
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
7.4%
2/27
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.4%
2/27
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
7.4%
2/27
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
3.7%
1/27
|
|
Skin and subcutaneous tissue disorders
Subcutaneous abscess
|
3.7%
1/27
|
|
Vascular disorders
Haemangioma
|
3.7%
1/27
|
|
Vascular disorders
Haematoma
|
3.7%
1/27
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place