RecruitingPhase 2ACTRN12619001643167

A Phase 2 study to evaluate the safety and tolerability of ACT001 in treating adults with Progressing Fibrosing Interstitial Lung Disease (PF-ILD), including Idiopathic Pulmonary Fibrosis (IPF)

A Phase 2 prospective multi-centre, parallel group study to evaluate the safety, tolerability, and activity of ACT001 in Adult participants with Progressing Fibrosing Interstitial Lung Disease (PF-ILD), including Idiopathic Pulmonary Fibrosis (IPF) when used alone or in combination with standard of care therapy.

Sponsor

Accendatech Au Pty Ltd

Enrollment

40 participants

Start Date

Jul 29, 2020

Study Type

Interventional

Conditions

Progressing Fibrosing Interstitial Lung Disease (PF-ILD)Idiopathic Pulmonary Fibrosis (IPF)

Summary

The current treatment options for patients with progressing Fibrosing Interstitial Lung Disease (PF-ILD), including Idiopathic Pulmonary Fibrosis (IPF) is limited and specific cohorts of patients cannot tolerate the approved treatments. Following the demonstration of activity in animal models for respiratory fibrotic diseases, this study will evaluate the safety and biological activity of ACT001 in adult patients. The study design will utilise a daily dose of ACT001, either alone or in combination with standard of care anti-fibrotic therapy, with evaluate safety, anti-inflammatory and anti-fibrotic activity in patients with stable disease state and evaluate any change in their respiratory function of a 12 month treatment period. The outcomes of this study are intended to support the biological activity of ACT001 so as to provide outcomes that can be translated into future research.

Eligibility

Sex: Both males and femalesMin Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

Fibrosing interstitial lung disease (ILD) is an umbrella term for a group of conditions where the lung tissue gradually becomes scarred and stiff, making it increasingly difficult to breathe. The most common form is Idiopathic Pulmonary Fibrosis (IPF), for which there is currently no cure. Existing treatments can slow the scarring but can cause significant side effects, and some patients cannot tolerate them. ACT001 is an experimental drug that has shown promise in reducing inflammation and fibrosis (scarring) in animal studies of respiratory disease. This Phase 2 study is evaluating whether ACT001 is safe and has any beneficial biological effect — such as reducing inflammation and slowing the scarring process — in adults with progressing fibrosing ILD, either alone or combined with standard anti-fibrotic therapy. Participants will take the medication daily for 12 months and have regular lung function tests and monitoring. You may be eligible if you are 18 or older and have been diagnosed with fibrosing interstitial lung disease confirmed on a high-resolution CT scan, with lung function above certain minimum thresholds (FVC >45%, DLCO >30%). Patients with COPD, severe heart disease, significant kidney or liver impairment, or a recent stroke would not be eligible. Women of childbearing potential must use reliable contraception.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

The investigational product is ACT001, dimethylamino michael adduct of micheliolide (DMAMCL), the drug product is an immediate release capsule containing 100mg of DMAMCL and is yellow and grey in colour. Each size 0 capsule also contains gelatine starch, magnesium stearate and silicon dioxide. Using a three-step synthetic sequence, ACT001 was obtained from parthenolide, which can be isolated from western medical herb tanacetum parthenium (feverfew), or Chinese medical herb magnolia delavayi franch. Cohort 1 will recruit participants that have been diagnosed with pulmonary fibrosing interstitial lung disease (PF-ILD), and are not receiving antifibrotic therapy. Cohort 2 will recruit participants that have been diagnosed with PF-ILD, and are currently receiving antifibrotic therapy. Patients will be assigned after consent to be administered either ACT001 orally (400mg/day) alone, or in combination with existing antifibrotic treatments. Each oral daily dose will be taken as two capsules in the morning and two in the evening after meal, with each dose recorded, for up to 52 weeks at investigator's discretion based on clinical assessment of safe use. Participants will be required to return any unused doses of study drug and/or the original container to site. The number of capsules of study product returned to the site will be counted and recorded as a measure of compliance. The investigator must account for all used Investigational Product containers. The completed dispending and inventory record will be provided to the sponsor and copies filed in the Investigator Site File (ISF). At the completion or termination of the study, a final drug accountability review and reconciliation must be completed, and any discrepancies must be investigated, and their resolution documented.