Clinical trial NCT06600425: A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD
Official title: A Phase 1b, Single-Center, Open-Label Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: Primary Ciliary Dyskinesia
Keywords: Primary Ciliary Dyskinesia
PCD
Kartagener Syndrome
Lead sponsor: ReCode Therapeutics
Sponsor class: INDUSTRY
First posted: 2024-09-19
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility 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 \&amp;gt;450 msec (or \&amp;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.

Other protocol defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: The number of participants with Adverse Events (AEs), including treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).
timeFrame: From Baseline Through Week 24
Secondary outcomes:
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: Royal Brompton Hospital
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
Study officials:
affiliation: ReCode Therapeutics, Inc.
name: John Matthews, MBBS, MCRP, PhD
role: STUDY_CHAIR
affiliation: Royal Brompton &amp; Harefield NHS Foundation Trust
name: Michael Loebinger, MD
role: PRINCIPAL_INVESTIGATOR
References: