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

NCT06633757 | Completed Phase 1 FDA Drug

• 1 other identifier: RCT1100-103
• Study Type: interventional
• Target: 14
• Locations: 3 countries
• Sites: 3

Brief Summary

This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

Conditions

Primary Ciliary Dyskinesia (PCD)

Keywords

Primary ciliary Dyskinesia; PCD; Kartagener Syndrome

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

PCD Participants

EXPERIMENTAL

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

Drug: RCT1100

Interventions

RCT1100 DRUG

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

PCD Participants

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

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 (3)

UNC

Chapel Hill, North Carolina, 27514, United States

Location

Copenhagen University Hospital - Rigshospitalet

Copenhagen, 2100, Denmark

Location

Münster University Hospital, Albert-Schweitzer-Campus 1

Münster, North Rhine-Westphalia, 48149, Germany

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

• Heymut Omran, MD

  University Hospital Muenster

  PRINCIPAL INVESTIGATOR

• Kim G Nielsen, Dr Med Sci

  Rigshospitalet, Denmark

  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: October 7, 2024
• First Posted: October 9, 2024
• Study Start: October 22, 2024
• Primary Completion: June 30, 2025
• Study Completion: February 10, 2026
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); DE (1); DK (1)