Access to Pirfenidone Solution for Inhalation (AP01)

• Research type

  Research Study

• Full title

  Access to Pirfenidone Solution for Inhalation (AP01) for Treatment of Progressive, Fibrosing Interstitial Lung Diseases, including Idiopathic Pulmonary Fibrosis

• IRAS ID

  293785

• Contact name

  Nazia Chaudhuri

• Contact email

  nazia.chaudhuri@nhs.net

• Sponsor organisation

  Avalyn Pharma, Inc.

• Eudract number

  2020-005103-39

• Clinicaltrials.gov Identifier

  n/a, n/a

• Duration of Study in the UK

  4 years, 4 months, 6 days

• Research summary

  The AP01-005 study allows participants to continue or start therapy with AP01 (inhaled pirferidone). Participants to be included are diagnosed with Interstitial Lung Diseases (ILD) including but not limited to Idiopathic Pulmonary Fibrosis (IPF), fibrosing phenotype ILD, pulmonary involvement of scleroderma, rheumatoid lung and silicosis. The rationale is to support such participants without treatment option and participants who are unable to enroll in an AP01 study to benefit of the AP01.

  Clinical studies of AP01 have demonstrated that doses up to 100 mg twice daily are safe and well-tolerated and show promise as an efficacious treatment for fibrosing ILDs. Participants will have an in clinic visit every 12 weeks to collect any safety and lung function tests that are performed as standard care.

  AP01 is a 100 mg in 8 millilitre (mL) aqueous solution delivered by oral inhalation twice daily following nebulisation (administration) by the eFlow (device that allows drug to be inhaled). Participants study duration will depend on how long the participant will benefit from the drug, either any side effects which will require study termination or sponsor or investigator decision.
  
  Currently, there are two drugs, pirfenidone and nintedanib, given orally used to help slow down development of scar tissue in the lungs. These oral medications have side-effects, which in some participants limit their ability to use them; these approved medications are not usually prescribed for participants with early stage IPF. Studies have shown participants may be under-treated due to these factors.

  In this study, AP01, a formulation of pirfenidone that is optimised for delivery directly to the lung will be inhaled via a nebuliser; a device that converts liquid into a mist that is breathed into the lungs. Earlier studies showed taking the medication this way led to less pirfenidone in the blood stream and fewer side effects that are known to occur with oral pirfenidone. This study will collect further data on side effects and tolerability.

• REC name

  London - City & East Research Ethics Committee

• REC reference

  21/LO/0190

• Date of REC Opinion

  23 Apr 2021

• REC opinion

  Further Information Favourable Opinion