A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD

ReCode Therapeutics

Status and phase

Completed

Phase 1

Conditions

Primary Ciliary Dyskinesia

Treatments

Drug: RCT1100

Study type

Interventional

Funder types

Industry

Identifiers

NCT06600425

RCT1100-102

Details and patient eligibility

About

This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

Full description

The primary objective of this study is to assess the safety, tolerability, ciliary rescue, pharmacodynamic biomarkers, and preliminary efficacy of RCT1100 following multiple doses of inhaled RCT1100 administered via nebulizer to participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene.

Enrollment

7 patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Major Inclusion Criteria:

Healthy, adult, male or female of, 18-70 years of age, inclusive, at screening.
Participant has clinical diagnosis of PCD and disease-causing mutations in the DNAI1 gene
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.
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.
12-lead ECG with QT interval &gt;450 msec (or &gt;480 msec for BBB)
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.
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.