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

ReCode Therapeutics

Status and phase: Completed; Phase 1

Conditions: Primary Ciliary Dyskinesia

Treatments: Drug: RCT1100

Study type: Interventional

Funder types: Industry

Identifiers

NCT05737485

RCT1100-101

Details and patient eligibility

About

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

Full 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.

Enrollment: 9 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

RCT1100

Experimental group

Description:

Drug: RCT1100 single dose

Treatment:

Drug: RCT1100