Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study, in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100

This is the first-in-human study with RCT1100 and is designed to provide initial safety and tolerability data for future clinical studies.

Study Overview

• Status: Recruiting
• Conditions: Primary Ciliary Dyskinesia
• Intervention / Treatment: Drug: RCT1100

Detailed Description

The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer to healthy participants and patients with Primary Ciliary Dyskinesia caused by a pathogenic mutation in the DNAI1 Gene.

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Priya Ryali, MBA; Phone Number: 650-629-7900; Email: clinicaltrials@recodetx.com
• Study Contact Backup: Name: Cathy Vo Buu; Phone Number: 650-629-7900; Email: clinicaltrials@recodetx.com

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2109
      • Recruiting
      • Macquarie University
• New Zealand
  • Auckland, New Zealand
    • Completed
    • New Zealand Clinical Research
• United Kingdom
  • London, United Kingdom, SW3 6NP
    • Recruiting
    • Royal Brompton Hospital
• United States
  • Nevada
    • Las Vegas, Nevada, United States, 89113
      • Recruiting
      • PPD - Las Vegas Research Unit
      • Contact: Phone Number: 877-362-2608
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Not yet recruiting
      • University of Pennsylvania

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Major Inclusion Criteria:

• Healthy, adult, male or female of, 18-65 years of age, inclusive, at screening.
• Participant has pathogenic mutations in the DNAI1 gene
• The 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.
• 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.

• 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
• History of alcohol abuse or drug addiction with the last year of screening.
• Active smoker (vaping included).

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: RCT1100; Drug: RCT1100 single dose	Drug: RCT1100; 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
The number of participants with Adverse Events (AEs) 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 Day 180

Collaborators and Investigators

• Sponsor: ReCode Therapeutics
• Investigators: Study Chair: John Matthews, MBBS, MCRP, PhD, ReCode Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 18, 2023
• Primary Completion (Estimated): September 21, 2024
• Study Completion (Estimated): December 21, 2024

Study Registration Dates

• First Submitted: January 20, 2023
• First Submitted That Met QC Criteria: February 20, 2023
• First Posted (Actual): February 21, 2023

Study Record Updates

• Last Update Posted (Actual): March 8, 2024
• Last Update Submitted That Met QC Criteria: March 7, 2024
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Primary Ciliary Dyskinesia; Kartagener Syndrome; PCD
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Respiratory Tract Diseases; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Otorhinolaryngologic Diseases; Movement Disorders; Abnormalities, Multiple; Ciliopathies; Dyskinesias; Ciliary Motility Disorders
• Other Study ID Numbers: RCT1100-101

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