A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD

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

This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

Study Overview

• Status: Completed
• Conditions: Primary Ciliary Dyskinesia
• Intervention / Treatment: Drug: RCT1100

Detailed Description

The primary objective of this study is to assess the safety, tolerability, ciliary rescue, pharmacodynamic biomarkers, and preliminary efficacy of RCT1100 following multiple doses of inhaled RCT1100 administered via nebulizer to participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene.

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom, SW3 6NP
    • Royal Brompton Hospital
  • Southampton, United Kingdom, SO16 6YD
    • University Hospital Southampton NHS Foundation Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Major Inclusion Criteria:

• Healthy, adult, male or female of, 18-70 years of age, inclusive, at screening.
• Participant has clinical diagnosis of PCD and disease-causing mutations in the DNAI1 gene
• Participant has a forced expiratory volume in one second (FEV1) of at least 50% predicted.

Major Exclusion Criteria:

• History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
• History of cancer, with exception of adequately treated basal cell or squamous cell carcinoma of the skin.
• Predisposition to bleeding or clinically meaningful hemorrhagic event in the 12 months prior
• Medically significant hemoptysis.
• Anticoagulation therapy for the treatment of a pulmonary embolus or has had a pulmonary embolus in the last 6 months of screening.
• Active tuberculosis infection.
• 12-lead ECG with QT interval >450 msec (or >480 msec for BBB)

• Laboratory abnormalities in clinical laboratory tests at screening:

  1. Serum creatinine level
  2. Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
  3. Hematological or coagulation values outside the normal reference range
• Any medical history of disease that has the potential to cause a rise in total bilirubin over the ULN.
• COVID-19 infection within 4 weeks of Screening or receipt of COVID-19 vaccine within 2 weeks prior to first dose of RCT1100.
• Receipt of vaccine with live virus, attenuated live virus, or live viral components within 2 weeks prior to first dose of RCT1100 or to receive these vaccines during treatment or within 8 weeks of completion of study treatment.

Other protocol defined inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PCD Participants; RCT1100 mRNA therapy supplied to eligible participants with with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene	Drug: RCT1100; RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).	Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), as well as an adverse event of special interest (AESI): "Fever", which will include body temperature and any associated symptoms (chills, myalgia).	From Baseline Through Week 24

Collaborators and Investigators

• Sponsor: ReCode Therapeutics
• Investigators: Study Chair: John Matthews, MBBS, MCRP, PhD, ReCode Therapeutics, Inc.; Principal Investigator: Michael Loebinger, MD, Royal Brompton & Harefield NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Actual): September 20, 2024
• Primary Completion (Actual): June 6, 2025
• Study Completion (Actual): August 27, 2025

Study Registration Dates

• First Submitted: September 6, 2024
• First Submitted That Met QC Criteria: September 16, 2024
• First Posted (Actual): September 19, 2024

Study Record Updates

• Last Update Posted (Estimated): October 8, 2025
• Last Update Submitted That Met QC Criteria: October 2, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Primary Ciliary Dyskinesia; Kartagener Syndrome; PCD
• Additional Relevant MeSH Terms: Ciliopathies; Cardiovascular Diseases; Heart Diseases; Genetic Diseases, Inborn; Respiratory Tract Diseases; Bronchial Diseases; Congenital Abnormalities; Otorhinolaryngologic Diseases; Cardiovascular Abnormalities; Heart Defects, Congenital; Abnormalities, Multiple; Respiratory System Abnormalities; Bronchiectasis; Dextrocardia; Situs Inversus; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliary Motility Disorders; Kartagener Syndrome
• Other Study ID Numbers: RCT1100-102

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes