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The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis

PHASE2RECRUITING

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.

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Males and females aged >5 years, able to take capsules orally.

Fibrosing interstitial pneumonia (Idiopathic PF, idiopathic non-specific interstitial pneumonia, chronic hypersensitivity pneumonitis, pleuroparenchymal fibroelastosis, unclassifiable interstitial lung disease (ILD)) diagnosed according to the current international guidelines.

Age-adjusted peripheral blood leukocyte telomere length < 10th centile on Flow-FISH.

FVC > 40% predicted.

DLCO > 25% predicted.

If receiving background pirfenidone / nintedanib, stable dose for 28 days prior to screening.

Able to understand and sign a written informed consent form (or legally authorised representative).

Agreement to use a medically approved form of non-hormonal contraception (if of child-bearing potential) (noting that oral contraceptives are advised not to be used concurrently with danazol).

Exclusion criteria

Actively or imminently listed for lung transplantation.

Undergone, awaiting, or likely to require bone marrow transplantation within 12 months.

Concurrent enrolment in another study.

Females with a positive pregnancy test at screening or currently breastfeeding.

Pelvic infection.

Past jaundice with oral contraceptives.

Undiagnosed abnormal genital bleeding.

Undiagnosed ovarian/uterine masses

Any history of malignancy likely to result in significant disability or likely to require significant medical or surgical intervention within the next 12 months.

History of androgen-dependent tumour.

Any condition other than PF that, in the opinion of the investigator, is likely to result in the death of the participant within the next 12 months.

History of end-stage liver disease or ALT or AST > 3 times the upper limit of normal.

History of end-stage kidney disease requiring dialysis.

Markedly impaired cardiac function.

Known increased risk of or history of thromboembolism (e.g. Factor V Leiden, Protein C or S deficiency).

Uncontrolled hypertension.

Uncontrolled lipoprotein disorder.

Poorly-controlled diabetes mellitus.

History of marked or persistent androgenic reaction to previous gonadal steroid therapy.

History of epilepsy induced or worsened by previous gonadal steroid therapy.

History of raised intracranial pressure.

Known intolerance to danazol.

Porphyria.

Use of any of the following agents within 28 days before screening: danazol or other androgen therapy, warfarin or other anticoagulant, carbamazepine, phenytoin, investigational therapy, cytotoxic therapy, tacrolimus, cyclosporine.

Professional singer due to potential for voice change.

Competitive athletes.

Prostate specific antigen (PSA) above the upper limit of normal (adult males only).

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Eligibility

Age eligible for study : 5 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2021-09-07

Primary completion: 2024-12-01

Study completion finish: 2025-06-01

Study type

TREATMENT

Phase

PHASE2

Trial ID

NCT04638517

Intervention or treatment

DRUG: Danazol

DRUG: Placebo

Conditions

• Pulmonary Fibrosis
• Telomere Shortening
• Telomere Disease
• Dyskeratosis Congenita

Condition: Pulmonary Fibrosis, Telomere Shortening and more
DRUG: Danazol and other drugs
Newcastle, New South Wales, Australia and more
Sponsor: The University of Queensland

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

John Hunter Hospital

Research sites nearby

Select from list below to view details:

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 Prince Charles Hospital
Brisbane, Queensland, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
ACTIVE_COMPARATOR: Danazol 800mg daily in two divided doses orally for 12 months. In subjects who have difficulty tolerating danazol / placebo, the dose will be reduced by 200mg/day and side effects will be reassessed. If symptoms related to the study drug persist, subsequent 200mg/day dose reductions will be allowed until a tolerated dose is achieved. Background antifibrotic therapy is allowed.	DRUG: Danazol Danazol up to 800mg daily in two-divided doses.
PLACEBO_COMPARATOR: Placebo Matching placebo capsules.	DRUG: Placebo Matching placebo.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
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).	12 months

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Number of participants with treatment-emergent adverse events	Not Specified	12 months
Number of Participants With Death or Non-Elective Hospitalisation	Not Specified	12 months
Change in telomere length from baseline to 3, 6 and 9 months (base pairs)	Not Specified	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.	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.	6 and 12 months
Change in 6-minute walk distance from baseline	Not Specified	12 months
Change in Leicester cough questionnaire (LCQ) from baseline	Not Specified	12 months
Change in King's Brief Interstitial Lung Disease Questionnaire (K-BILD) from baseline	Not Specified	12 months
Change in Parent cough-specific quality of life (PCSQoL) from baseline	Not Specified	12 months

Frequently Asked Questions

Please note: some questions and answers are submitted by anonymous patients or using AI, and have not been verified by Clinrol

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

Clinical Trials Gov: The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis