TELO-SCOPE is a national, multi-centre, double-blind, placebo-controlled, randomised (2:1) trial which will test the hypothesis that, compared to placebo, the addition of danazol to standard of care in pulmonary fibrosis associated with short telomeres is safe and will result in reduced telomere attrition.
TELO-SCOPE is a national, multi-centre, double-blind, placebo-controlled, randomised trial which will be conducted in subjects aged \>5 years with a multi-disciplinary diagnosis of pulmonary fibrosis and with age-adjusted telomere length below the 10th centile in adults; and for children (age \< 16 years), a confirmed diagnosis of Dyskeratosis Congenita (DC). Consenting participants who meet all other inclusions and no exclusions will be randomised (n=50, 2:1 (danazol:placebo)) to receive danazol (maximum tolerated dose (up to 800mg daily, two-divided doses) or matched placebo, for 12 months in addition to standard of care background therapy. The primary outcome is change in telomere length at 12 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
29
John Hunter Hospital
Newcastle, New South Wales, Australia
Sydney Children's Hospital
Sydney, New South Wales, Australia
Royal Prince Alfred Hospital
Sydney, New South Wales, Australia
The Children's Hospital Westmead
Sydney, New South Wales, Australia
The Prince Charles Hospital
Brisbane, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
The Alfred
Melbourne, Victoria, Australia
The Austin
Melbourne, Victoria, Australia
Fiona Stanley Hospital
Perth, Western Australia, Australia
Change in absolute telomere length from baseline (base pairs)
Telomere length will be measured in absolute terms (base pairs) using the telomere shortest length assay (TeSLA).
Time frame: 12 months
Number of participants with treatment-emergent adverse events
Time frame: 12 months
Number of Participants With Death or Non-Elective Hospitalisation
Time frame: 12 months
Change in telomere length from baseline to 3, 6 and 9 months (base pairs)
Time frame: 3, 6 and 9 months
Change in forced vital capacity (FVC) at 6 and 12 months
FVC is measured as the volume of air exhaled during spirometry.
Time frame: 6 and 12 months
Change in diffusing capacity for carbon monoxide at 6 and 12 months
DLCO is a measurement of the of the lung's gas transfer ability.
Time frame: 6 and 12 months
Change in 6-minute walk distance from baseline
Time frame: 12 months
Change in Leicester cough questionnaire (LCQ) from baseline
Time frame: 12 months
Change in King's Brief Interstitial Lung Disease Questionnaire (K-BILD) from baseline
Time frame: 12 months
Change in Parent cough-specific quality of life (PCSQoL) from baseline
Time frame: 12 months
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