Table 1.

Ongoing clinical trials in familial pulmonary fibrosis.

Name/NCT Number	Aim	Method	Population	Intervention	Primary outcome	Enrolment	Start/End
ANDROTELO NCT03710356	Assess the safety and efficacy of danazol in patients with severe haematological or pulmonary disease related to telomeropathies.	Multi-centre Bayesian trial.	Subjects aged ≥15 years with mutation of a gene involved in telomere maintenance and severe haematological involvement and/or ILD.	Danazol for 12 months.	Haematological response or pulmonary response at 12 months.	40	2018-10-20 2022-10-20
TELO-SCOPE NCT04638517	Assess the safety and efficacy of danazol in adults and children with ILD associated with telomere shortening.	Multi-centre, double-blind, placebo-controlled, randomised trial.	Subjects aged >5 years with ILD associated with age-adjusted telomere length ≤10th centile.	Danazol or Placebo for 12 months.	Change in absolute telomere length in base pairs at 12 months.	50	2021-09-07 2025–06
EXG34217 Study NCT04211714	Assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders	Open label, single centre study	Age >18 years, with mild or moderate bone marrow failure and a diagnosis of telomere biology disorders.	Single infusion of autologous CD34+ cells contacted ex vivo with EXG-001.	Number of participants with treatment-related adverse events from baseline to 12 months.	12	2021-04-08 2027-04-08

Abbreviations: NCT, National Clinical Trail; ILD, interstitial lung disease.