KB407-02 A Phase 1 Study of Inhaled KB407, a Replication-Defective, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis

Status: Recruiting
Location: See all (5) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• The subject must have read, understood, and signed an Institutional Review Board/Ethics Committee (IRB/IEC) approved Informed Consent Form and must be able to and willing to follow study procedures and instructions

• Subjects aged 18 years or older at the time of Informed Consent

• A confirmed diagnosis of CF as defined by clinical signs and symptoms of CF and at least one of the following:

‣ A historical sweat chloride value \>60 mmol/L

⁃ Two copies of a disease causing mutation in the CFTR gene

• Clinically stable in the opinion of the Investigator

• Percent predicted FEV1 ≥40% and ≤100% of the predicted normal for age, gender, and height at Screening

• Resting oxygen saturation ≥92% on room air at Screening

Locations
United States
Florida
University of Florida, Gainesville
RECRUITING
Gainesville
Illinois
The Cystic Fibrosis Institute
RECRUITING
Northfield
North Carolina
Atrium Health Wake Forest Baptist Medical Center
RECRUITING
Winston-salem
New York
New York Medical College/ Boston Children's Health Physicians
RECRUITING
Hawthorne
Northwell Health Physicians
RECRUITING
New York
Contact Information
Primary
David Sweet, MD, PhD
dsweet@krystalbio.com
412-586-5830
Backup
Brittani Agostini, RN, CCRC
bagostini@krystalbio.com
412-586-5830
Time Frame
Start Date: 2023-06-30
Estimated Completion Date: 2026-01
Participants
Target number of participants: 12
Treatments
Experimental: Cohort 1 (open label)
A single administration of KB407
Experimental: Cohort 2 (open label)
Two administrations of KB407
Experimental: Cohort 3 (open label)
Four administrations of KB407
Related Therapeutic Areas
Sponsors
Leads: Krystal Biotech, Inc.

This content was sourced from clinicaltrials.gov