Clinical trial NCT05737485: Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Primary Ciliary Dyskinesia
Keywords: Primary Ciliary Dyskinesia
PCD
Kartagener Syndrome
Lead sponsor: ReCode Therapeutics
Sponsor class: INDUSTRY
First posted: 2023-02-21
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility criteria: Major Inclusion Criteria:

* Healthy, adult, male or female of, 18-75 years of age, inclusive, at screening.
* Participant has disease causing 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.

Primary outcomes:
measure: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
timeFrame: From Baseline Through Day 180
Secondary outcomes:
Other outcomes:
Locations:
city: Las Vegas
country: United States
facility: PPD - Las Vegas Research Unit
state: Nevada
city: Sydney
country: Australia
facility: Macquarie University
state: New South Wales
city: Auckland
country: New Zealand
facility: New Zealand Clinical Research
city: London
country: United Kingdom
facility: Royal Brompton Hospital
Study officials:
affiliation: ReCode Therapeutics, Inc.
name: John Matthews, MBBS, MCRP, PhD
role: STUDY_CHAIR
References: