Ph2 safety, biological activity, and PK study of ND-L02-s0201 for IPF
Research type
Research Study
Full title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Biological Activity, and PK of ND-L02-s0201 in Subjects with Idiopathic Pulmonary Fibrosis (IPF)
IRAS ID
243848
Contact name
Helen Parfrey
Contact email
Sponsor organisation
Nitto Denko Corporation
Eudract number
2017-004919-39
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 4 months, 9 days
Research summary
Idiopathic pulmonary fibrosis (IPF) is a progressive lung-scarring disease of unknown cause characterised by loss of lung function. The disease affects primarily older adults and typically has a survival of 2.5-3.5 years. Existing drug therapies are only able to slow down the process of scarring and new treatment options are needed.
ND-L02-s0201 is a new member of the small interfering RNA class of drugs that will be tested in the study. ND-L02-s0201 temporarily blocks HSP47 which is a protein involved in production of collagen. Interfering with the production of collagen which is involved in lung scarring may help treat IPF.
The main purpose of this study is to investigate the safety (side effects), tolerability (how well the body tolerates the drug), biological activity, and pharmacokinetics (what the body does to the drug) of ND-L02-s0201 in patients with IPF. Patients will be randomised (selected by chance) to receive either low dose ND-L02-s0201, high dose ND-L02-s0201, or placebo (pill that does not contain active drug). The drug will be given by drip every 2 weeks over a 24 week treatment period. The treatment period will be preceded by up to 6 weeks of screening. Patients who complete treatment will be followed up for 10 weeks.
Approximately 120 patients will take part in the study of which around 30 will participate in the Pharmacokinetics sub-study. Male or female IPF patients aged between 40 and 80 with adequate lung function and general state of health taking either pirfenidone or nintedanib, or none of these standard IPF drugs may be eligible for the study.
Study assessments include physical examinations, vital signs, ECG, evaluations of lung function, blood and urine tests, High Resolution CT scans, questionnaires.
Around 40 sites located in the US and Europe will participate in the study. Of these, 4 sites will be in the UK.Research Summary
The study was sponsored by Nitto Denko Corporation Medicine Studied: ND-L02-s0201 Study Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Biological Activity, and PK of ND-L02-s0201 in Subjects with Idiopathic Pulmonary Fibrosis (IPF).
Study number: ND-L02-s0201-005
When did this study take place? 08 October 2018 to 24 August 2022 Date of this report: 21 November, 2023 Why was this study done?
Idiopathic pulmonary fibrosis is a condition where the normal tissue in the lung thickens and turns into scar tissue over time. Treatments for IPF include prescription medications, oxygen therapy, pulmonary rehabilitation, and lung transplant. The two anti-scarring (antifibrotic) drugs approved by the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Ministry of Health, Labour, and Welfare (MHLW) of Japan for IPF are pirfenidone and nintedanib. Both have been shown to slow IPF progression.
ND-L02-s0201 is an experimental medication that has not been approved for the treatment of any medical condition by the regulatory authorities in any country and is being studied. ND-L02-s0201 was developed to treat fibrosis (scarring) of the lungs. It is made up of tiny particles of fat that contain a retinoid (a substance that acts like a vitamin). These tiny particles help carry the drug through the body and help the drug reach special cells in the lungs. These tiny particles also contain a small amount of RNA (ribonucleic acid) which is the active drug. RNA is used by the body to make new proteins. The RNA in ND-L02-s0201 is called an siRNA (small interfering RNA) and is designed to block the cells in the lungs from making a protein called “heat shock protein 47” (HSP47) for a limited period. It is thought that blocking the lungs from making HSP47 will help treat IPF because one of the roles of HSP47 is to help make collagen, which is involved in fibrosis (scarring).REC name
East of England - Cambridge South Research Ethics Committee
REC reference
18/EE/0249
Date of REC Opinion
16 Oct 2018
REC opinion
Further Information Favourable Opinion