TABLE 1.
Overview of treatment trials presented at the European Respiratory Society Congress 2023
| Study | Treatment | Study population | Study phase | Outcomes |
| Kellogg et al. [18] (NCT02874989) | Dasatinib (100 mg daily) and quercetin (1250 mg daily) | 20 patients with IPF | Post hoc analysis of a first in human open-label pilot study | A significant improvement in senolitic markers was found, but no improvement in short-term clinical outcomes |
| Arora et al. [19] | LASN01 (a monoclonal antibody targeting IL-11R) | 58 healthy volunteers | Phase 1 | LASN01 inhibited >95% of the IL-11R signalling and was well-tolerated |
| Wuyts et al. [20] INTEGRIS-IPF (NCT04396756) | Bexotegrast 320 mg (dual-selective inhibitor of αvβ6 and αvβ1) | 29 patients with IPF | Phase 2a, multicentre RCT | Well-tolerated in participants with IPF up to 40 weeks of treatment; bexotegrast seemed to reduce FVC decline, and in a majority of patients QLF score improved |
| Glaspole et al. [21] (ACT001-AU-003) | ACT001 (targeting NF-κB and STAT3 signalling pathways) | 44 patients, 35 IPF, 9 fILD | Phase 2, multicentre, parallel group | ACT001 reduced FVC decline after 52 weeks |
| Borie et al. [22] ANDROTELO trial (NCT03710356) | Danazol (synthetic hormonal drug) | 25 patients with PF with a (likely) pathogenic TRG variant | Phase 2, open-label, multicentre | Danazol was poorly tolerated, only 10 patients completed 12-month treatment |
| West et al. [23] (ACTRN12618001838202) | Nebulised pirfenidone | 72 patients with IPF (41 from phase 1b, 31 newly enrolled) | Phase 2 open-label extension, multicentre | Reduction in FVC decline after 48 weeks, −165.5±242.6 mL in patients from 1b, −151.1±358.9 mL in newly enrolled patients |
| Corte et al. [24] (NCT04308681) | 30 or 60 mg of BMS-986278 (oral lysophosphatidic acid receptor 1 antagonist) | 125 patients with PPF | Phase 2 double-blind placebo-controlled RCT, multicentre | BMS-986278 reduced the rate of FVC decline after 26 weeks and was safe and well-tolerated; rates of FVC decline were 4.3% for placebo, −2.7% for 30 mg and −1.1% for 60 mg |
| Molyneaux et al. [25] PAciFy cough trial (NCT04429516) | Low-dose controlled-release morphine sulphate | 44 patients with IPF and chronic cough | Multicentre, double-blind, placebo-controlled, crossover phase 4 RCT | Morphine sulphate reduced objective cough frequency with 39.4% compared to placebo after 14 days; PROMs improved and treatment was well-tolerated |
| Myall et al. [26] | CPAP therapy in managing OSA | 44 patients with fILD and OSA, 12 started CPAP | Phase 4 study, non-randomised | Improvement in PROMs (K-BILD and PSQI) |
| Dhooria et al. [27] SARCORT trial (NCT03265405) | Prednisolone | 86 patients with pulmonary sarcoidosis | Single-centre, open-label, parallel-group RCT | Daily prednisolone dose of 40 mg is not superior to 20 mg in overall response; the frequency of relapse, treatment failure after 18 months and the mean time to these events were similar |
| Kinnersley et al. [28] (NCT03824392) | Efzofitimod (selective modulator of neuropilin-2) | 37 patients with pulmonary sarcoidosis (20 subtherapeutic dose, 17 therapeutic dose) | Phase 1b/2a randomised, double-blind, placebo-controlled, multiple ascending doses | In the therapeutic group a lower percentage of patients relapsed (7.7% versus 54.4%), FVC improved over time and health status improved in a significantly higher percentage |
| Bermudo Peloche et al. [29] FIBRO-COVID (NCT04607928) | Pirfenidone | 113 patients with post-COVID-19 pulmonary fibrosis | Phase 2, multicentre, placebo-controlled RCT | FVC improved with 12.74% in the pirfenidone group and 4.35% in the placebo group after 6 months, but differences were not statistically significant |
| Harari et al. [30] | Nintedanib | 30 patients with LAM | Phase 2, multicentre, open-label | Nintedanib stabilised lung function parameters (FVC, FEV1, DLCO) and was generally well-tolerated |
| Trapnell et al. [31] | Pulmonary macrophage transplantation | Not informed | Preclinical | Pulmonary macrophage transplantation corrected PAP and normalised disease biomarkers; approved to start a human trial |
| Bonella et al. [32] ASCEND trial (NCT02004691) | OAER therapy | 36 patients with acid sphingomyelinase deficiency | Phase 2/3, multicentre | Treatment with OAER therapy led to a persistent and progressive improvement of radiological infiltrates, FVC, DLCO and exercise capacity, and was well-tolerated |
IPF: idiopathic pulmonary fibrosis; IL-11R: interleukin 11 receptor; RCT: randomised controlled trial; FVC: forced vital capacity; QLF: Quantitative Lung Fibrosis score; fILD: fibrotic interstitial lung disease; PF: pulmonary fibrosis; TRG: telomere-related gene; PPF: progressive pulmonary fibrosis; PROMs: patient reported outcome measures; CPAP: continuous positive airway pressure; OSA: obstructive sleep apnoea; K-BILD: the King's brief interstitial lung disease questionnaire; PSQI: Pittsburgh sleep quality index; LAM: lymphangioleiomyomatosis; FEV1: forced expiratory volume in 1 s; DLCO: diffusing capacity of the lung for carbon monoxide; PAP: pulmonary alveolar proteinosis; OAER: olipudase alfa enzyme replacement.