Clinical trial NCT06633757: Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance
Official title: A Phase 1b, Open-label, Study of RCT1100 in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance and Other Measures of Pharmacodynamic Activity
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: Primary Ciliary Dyskinesia (PCD)
Keywords: Primary ciliary Dyskinesia
PCD
Kartagener Syndrome
Lead sponsor: ReCode Therapeutics
Sponsor class: INDUSTRY
First posted: 2024-10-09
Enrollment: 12
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 a multi-dose 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 determine the impact of multiple doses of inhaled RCT1100, administered via nebulizer, on MCC with adult participants with Primary Ciliary Dyskinesia caused by pathogenic mutations in the DNAI1 Gene.

Eligibility criteria: Major Inclusion Criteria:

* The participant is a male or female, 18 to 75 years of age, inclusive, at the time of consent.
* Participant has disease-causing (pathogenic and/or likely pathogenic) mutations in the DNAI1 gene.
* The participant has a percent predicted forced expiratory volume in 1 second (FEV1pp) of at least 40% 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.
* 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: To Determine the impact of multiple doses of RCT1100 on MCC
timeFrame: Baseline through Week 12
Secondary outcomes:
Other outcomes:
Locations:
city: Chapel Hill
country: United States
facility: UNC
state: North Carolina
city: Copenhagen
country: Denmark
facility: Copenhagen University Hospital - Rigshospitalet
city: Münster
country: Germany
facility: Münster University Hospital, Albert-Schweitzer-Campus 1
state: North Rhine-Westphalia
Study officials:
affiliation: ReCode Therapeutics, Inc.
name: John Matthews, MBBS, MCRP, PhD
role: STUDY_CHAIR
affiliation: University Hospital Muenster
name: Heymut Omran, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Rigshospitalet, Denmark
name: Kim G Nielsen, Dr Med Sci
role: PRINCIPAL_INVESTIGATOR
References: