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

NCT06600425 | Completed Phase 1 FDA Drug

• 1 other identifier: RCT1100-102
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 2

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.

Conditions

Primary Ciliary Dyskinesia

Keywords

Primary Ciliary Dyskinesia; PCD; Kartagener Syndrome

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

PCD Participants

EXPERIMENTAL

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

Drug: RCT1100

Interventions

RCT1100 DRUG

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

PCD Participants

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• lead ECG with QT interval \>450 msec (or \>480 msec for BBB)
• Laboratory abnormalities in clinical laboratory tests at screening:
• Serum creatinine level
• Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
• 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.

Sponsors & Collaborators

• ReCode Therapeutics: lead

Study Sites (2)

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Ciliary Motility Disorders; Kartagener Syndrome

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Bronchiectasis; Bronchial Diseases; Respiratory System Abnormalities; Dextrocardia; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Situs Inversus

Study Officials

• John Matthews, MBBS, MCRP, PhD

  ReCode Therapeutics, Inc.

  STUDY CHAIR

• Michael Loebinger, MD

  Royal Brompton & Harefield NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2024
• First Posted: September 19, 2024
• Study Start: September 20, 2024
• Primary Completion: June 6, 2025
• Study Completion: August 27, 2025
• Last Updated: October 8, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2)