Phase I, Part B study in adults with Primary Ciliary Dyskinesia (PCD) with RCT1100
Research type
Research Study
Full title
A Phase 1, Randomized, Double-Blind, Placebo Controlled, Single Ascending Dose Study in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults with Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100
IRAS ID
1008307
Contact name
Rachael Cavanagh
Contact email
Sponsor organisation
ReCode Therapeutics
Clinicaltrials.gov Identifier
Research summary
The aim of this clinical study being conducted in PCD (primary ciliary dyskenesia) patients who have a specific pathogenic mutation (on the DNAI1 gene) is to investigate the safety and tolerability of a potential new medicine called RCT1100 being developed as a treatment for PCD. There is currently no treatment for this condition, other than management of its symptoms with antibiotics and reducing exposure to inflammatory triggers. This study will evaluate how well it is tolerated in patients, when given at different doses. Patients will receive RCT1100, where both the patient and the investigators will know what treatment they are receiving (open-label). This study will be conducted at one site in England and is part B of a two part (A and B) study. The design of part B will be modified depending upon the outcome of part A. In part A, healthy volunteers will receive either RCT1100 or a matching dummy medicine called a placebo, and neither the patients nor the investigators will know which treatment they are receiving (this is called a double-blind study). In part A, participants will be given, RCT1100 or placebo by inhalation via an inhaler, with doses changing according to the group (cohort) to which a participant is allocated. In part B, participants will be given RCT1100 by inhalation via an inhaler with doses changing according to the group (cohort) to which the participant has been allocated. Participants will undergo physical and clinical examinations to assess their general health and their diagnosis of PCD by genetic testing prior to receiving the new medicine, and blood samples will be taken from participants as part of the health checks. The study is open to adults 18-65 who meet the criteria.The length of the study is up to 17 weeks, including screening (12 wks), dosing (1 day), observational period (48 hrs post dose) and post-treatment visits (Day 8, 15 and 29 post-dose). The study is not funded outside of Recode.
REC name
North East - York Research Ethics Committee
REC reference
23/NE/0177
Date of REC Opinion
21 Nov 2023
REC opinion
Further Information Favourable Opinion