ReCode PCD Bronchoscopy Study
Research type
Research Study
Full title
A Phase 1b, Single-Center, Open-label Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults with Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene
IRAS ID
1009008
Contact name
Jennifer Scrivens
Contact email
Sponsor organisation
ReCode Therapeutics Inc.
Research summary
The study is being conducted in people with primary ciliary dyskinesia (PCD), caused by a change in a gene (DNAI1 gene), this change affects about 10% of PCD patients. In this type of PCD, the tiny hairs (cilia) that line the lungs and help move mucus out of the lungs, do not move properly, causing PCD symptoms. There is currently no cure for PCD and ReCode Therapeutics Inc, have developed a potential new treatment (RCT1100) for PCD. Up to 10 participants will take part and receive RCT1100, via a nebuliser, 3 times a week for 12 weeks. This study will investigate how safe and well tolerated RCT1100 is and if it can restore cilia movement and therefore mucociliary clearance in PCD.
The study will be ran at a hospital in England with expertise in the population and study procedures. Its open to adults with PCD aged 18-70, meeting the criteria. Subjects enrolled in RCT1100-101 (IRAS 1008307) may participate in this study. Participants will have a bronchoscopy (procedure to look inside the lungs) before receiving their first dose of RCT1100 in the study, during which lung tissue samples will be collected and analysed. Participants will stay over night in hospital for observation after having the 1st dose of RCT1100. The next 3 doses of RCT1100 will be given at the hospital, participants will stay for at least 1hr observation. Further doses will be given in the hospital clinic or at the patients home by a nurse. Participants will have another bronchoscopy at either Week 4 or Week 12 but not both and more lung samples will be collected and analysed. Participants will undergo blood tests, physical and clinical examinations to assess general health and specialist lung function tests to see if RCT1100 helps people with PCD. Salbutamol may be given before dosing with RCT1100 or if a participant is already prescribed a bronchodilator they may use their regular bronchodilator instead. The study will last about 7 months, with screening, 12 weeks treatment and 12 week follow up.REC name
South Central - Oxford A Research Ethics Committee
REC reference
24/SC/0062
Date of REC Opinion
5 Apr 2024
REC opinion
Further Information Favourable Opinion