A Phase 2 study of RXC007 in patients with IPF
Research type
Research Study
Full title
A Multi-Cohort, Randomised, Placebo-Controlled Phase 2a Study to Assess the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Ascending Doses of RXC007 in Patients with Idiopathic Pulmonary Fibrosis
IRAS ID
1005138
Contact name
Jane Robertson
Contact email
Sponsor organisation
RedX Pharma Plc
Eudract number
2022-000498-15
ISRCTN Number
ISRCTN60385283
Clinicaltrials.gov Identifier
N/A
Research summary
Summary of Research
The purpose of this study is to investigate the study drug RXC007. The main objectives of this study are as follows:
- To determine the safety and tolerability (degree to which side effects of a drug can be tolerated) of RXC007 when it is administered as twice daily doses over a period of up to 12 weeks (84 days).
- To investigate the levels of RXC007 in the blood, how this changes over a period of time and to evaluate whether there are differences in the levels between different dose strengths of RXC007.
- To investigate the effect of RXC007 on the body (known as pharmacodynamics) by analysing the levels of certain biomarkers in the body and to assess the effect of RXC007 on markers associated with Idiopathic Pulmonary Fibrosis (IPF).This study will comprise of 3 main study groups and 2 additional sub-study groups. The main study will consist of 3 planned groups of 16 patients with IPF: each group investigating a different dose strength of RXC007 starting at the lowest planned dose in Group 1 to the highest planned dose in Group 3. The sub-study will consist of 2 groups of up to 8 patients with IPF (each group respectively evaluating the same dose of RXC007 as evaluated in Group 1 and 3 of the main study). The sub-study groups will include evaluations of the levels of RXC007 found in fluid collected from the lungs.
The purpose of the data generated in this study is to provide further information and guidance to support the study sponsor in the development of the study drug RXC007 and to provide early data as to the potential effectiveness of RXC007 as a treatment for IPF.
Summary of Results
This study tested RXC007, an experimental oral treatment for idiopathic pulmonary fibrosis (IPF), a lung disease that causes scarring and breathing difficulty. The study looked at how safe RXC007 is, how the body handles it and whether in helps people with IPF. Some participants received RXC007 alone, while others took it with their usual IPF medicine (nintedanib or pirfenidone). The study was randomised and placebo-controlled, meaning some participants received RXC007 and others got a dummy capsule (placebo), and nobody knew which they were taking.
48 adults aged 40 to 80 years with IPF. Most were men (about 77%) and all were White. Participants were split into three groups: RXC007 20 mg twice daily, RXC007 50 mg twice daily, or placebo. Some participants continued using their usual IPF medicine.
RXC007 was given as a capsule either 20 mg or 50 mg twice a day. It was compared to a placebo capsule with no active medicine. RXC007 was tested both on its own and alongside approved IPF medicines (nintedanib or pirfenidone).
Most participants taking RXC007 had at least one side effect, similar to those taking placebo. No one died during the study. About participants17% stopped taking RXC007 due to side effects. One person had a serious side effect, but it was not caused by RXC007. Common side effects included cough, nausea, and diarrhoea. Overall RXC007 was well tolerated.
The study lasted 12 weeks, with some participants continuing for up to 24 weeks. RXC007 appeared to slow the decline in lung function compared with placebo, especially at the lower dose, although the difference was not statistically significant. Lung function was measured using a test called forced vital capacity (FVC). Participants taking RXC007 had smaller drops in FVC compared with those on placebo. There were also improvements in cough and quality of life scores, , but results varied.
The results suggest RXC007 might help slow down IPF. However, because this was asmall and early study, more research is needed to confirm these findings. No new safety concerns were found, and the results support further testing of RXC007.
A larger Phase 2b study will be planned to learn more about RXC007’s effects on lung function and safety in people with IPF.
More details are available on:
• EU Clinical Trials Register (EudraCT number 2022-000498-15)
• ClinicalTrials.gov (NCT05570058)REC name
Wales REC 2
REC reference
22/WA/0122
Date of REC Opinion
14 Jun 2022
REC opinion
Further Information Favourable Opinion