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

Phase 1

Active, not recruiting

• Conditions: Primary Ciliary Dyskinesia
• Interventions: Drug: RCT1100

• Registration Number: NCT06600425
• Lead Sponsor: ReCode Therapeutics

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.

Study Timeline

• Study First Submitted: 2024-09-06
• Study First Submitted QC: 2024-09-16
• Study First Posted: 2024-09-19
• Study Start: 2024-09-20
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-24
• Last Update Posted: 2025-04-29
• Primary Completion: 2025-06-30
• Study Completion: 2025-09-22

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 10

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).	From Baseline Through Week 24	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).

Trial Locations

Locations (2)

Royal Brompton Hospital; 🇬🇧 London, United Kingdom

University Hospital Southampton NHS Foundation Trust; 🇬🇧 Southampton, United Kingdom