Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance

A Phase 1b, Open-label, Study of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance and Other Measures of Pharmacodynamic Activity

This is a multi-dose 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 (PCD)
• Intervention / Treatment: Drug: RCT1100

Detailed Description

The primary objective of this study is to determine the impact of multiple doses of inhaled RCT1100, administered via nebulizer, on MCC with adult participants with Primary Ciliary Dyskinesia caused by pathogenic mutations in the DNAI1 Gene.

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 1

Contacts and Locations

Study Locations

• Denmark
  • Copenhagen, Denmark, 2100
    • Copenhagen University Hospital - Rigshospitalet
• Germany
  • North Rhine-Westphalia
    • Münster, North Rhine-Westphalia, Germany, 48149
      • Münster University Hospital, Albert-Schweitzer-Campus 1
• United States
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • UNC

Participation Criteria

Eligibility Criteria

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

Description

Major Inclusion Criteria:

• The participant is a male or female, 18 to 75 years of age, inclusive, at the time of consent.
• Participant has disease-causing (pathogenic and/or likely pathogenic) mutations in the DNAI1 gene.
• The participant has a percent predicted forced expiratory volume in 1 second (FEV1pp) of at least 40% 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.

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 Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene	Drug: 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
To Determine the impact of multiple doses of RCT1100 on MCC	The Change in Mucociliary and Cough Clearance through 1 hour after radiotracer administration by either the albumin-based PRMC or sulfur-colloid technique	Baseline through Week 12

Collaborators and Investigators

• Sponsor: ReCode Therapeutics
• Investigators: Study Chair: John Matthews, MBBS, MCRP, PhD, ReCode Therapeutics, Inc.; Principal Investigator: Heymut Omran, MD, University hospital Muenster; Principal Investigator: Kim G Nielsen, Dr Med Sci, Rigshospitalet, Denmark

Study record dates

Study Major Dates

• Study Start (Actual): October 22, 2024
• Primary Completion (Actual): June 30, 2025
• Study Completion (Actual): February 10, 2026

Study Registration Dates

• First Submitted: October 7, 2024
• First Submitted That Met QC Criteria: October 7, 2024
• First Posted (Actual): October 9, 2024

Study Record Updates

• Last Update Posted (Actual): April 30, 2026
• Last Update Submitted That Met QC Criteria: April 28, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Kartagener Syndrome; PCD; Primary ciliary Dyskinesia
• 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: RCT1100-103

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