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

NCT ID: NCT06600425

Last Updated: 2025-10-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-09-20
• Study Completion Date: 2025-08-27

Brief Summary

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

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.

Conditions

Primary Ciliary Dyskinesia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PCD Participants

RCT1100 mRNA therapy supplied to eligible participants with with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene

Group Type: EXPERIMENTAL

RCT1100

Intervention Type: DRUG

RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Interventions

RCT1100

RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Intervention Type: DRUG

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ReCode Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John Matthews, MBBS, MCRP, PhD

Role: STUDY_CHAIR

ReCode Therapeutics, Inc.

Michael Loebinger, MD

Role: PRINCIPAL_INVESTIGATOR

Royal Brompton & Harefield NHS Foundation Trust

Locations

Royal Brompton Hospital

London, , United Kingdom

University Hospital Southampton NHS Foundation Trust

Southampton, , United Kingdom

Countries

United Kingdom

Other Identifiers

RCT1100-102

Identifier Type: -

Identifier Source: org_study_id