MedTrace Logo

A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Adult Patients With Cystic Fibrosis

NCT06963008 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-05-08

No results posted yet for this study

Summary

The study adopts a prospective, single-center, open-label clinical study to evaluate the safety and efficacy of inhaled BMD003(CFTR mRNA) in Chinese adult patients with cystic fibrosis This study is a single dose escalation study of BMD003 nebulized inhalation in CF patients.

The study is divided into four cohorts, they are 2mg, 4mg, 8mg, and 16mg dose groups. The first dose group (2mg) is planned to enroll one patient. During the study, if there are ≥ 2 grade treatment-related adverse events (TRAEs) related to the investigational product that occur during the study period, 2 patients will be added at the current dose level. Each of the remaining groups will include 2-3 subjects, regardless of gender. Based on the safety data review of subjects in the previous dose group 4 weeks after administration, subjects in the next dose group will be allowed to receive the next higher dose, while patients in the previous dose group will complete the study.

The study includes three stages: screening period (2 weeks), administration period (1 day), and follow-up period (approximately 13 months). After passing the screening of the subjects, D1 was admitted to the hospital, D1 received a single nebulized inhalation investigational product (BMD003), and D2 completed safety observation before being discharged.

During the study, PK sample collection and safety assessment will be conducted at the corresponding visit points. After the treatment is completed, each patient will be followed up according to the schedule specified in the protocol.This study will conduct multiple dosing studies after the end of a single dose escalation. Multiple dosing doses, dosing intervals, etc. will be revised based on preclinical data and single dose data, and will be implemented after ethical approval.

Conditions

  • Cystic Fibrosis (CF)
  • Gene Therapy

Interventions

DRUG

inhaled BMD003(CFTR mRNA)

BMD003 is the world's first freeze-dried CFTR Mrna product (stored at 2-8 ℃), which is reconstituted with injection water before use, nebulized through a vibrating sieve atomizer, and inhaled into the lungs through the mouth and nose.

Sponsors & Collaborators

  • Peking Union Medical College Hospital

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-05-10
Primary Completion
2026-05-10
Completion
2027-05-10

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06963008 on ClinicalTrials.gov