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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-18

Study Completion Date

2025-01-13

Brief Summary

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

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Detailed Description

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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.

Conditions

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Primary Ciliary Dyskinesia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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RCT1100

Drug: RCT1100 single dose

Group Type EXPERIMENTAL

RCT1100

Intervention Type DRUG

RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Interventions

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RCT1100

RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Intervention Type DRUG

Eligibility Criteria

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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.

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).

Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No