4D-710 in Adult Patients with Cystic Fibrosis

Phase 1

Recruiting

• Conditions: Cystic Fibrosis Lung
• Interventions: Biological: 4D-710

• Registration Number: NCT05248230
• Lead Sponsor: 4D Molecular Therapeutics

Brief Summary

This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.

Detailed Description

This Phase 1/2 trial will evaluate the safety, tolerability, and preliminary efficacy of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) advanced lung disease who are ineligible or unable to tolerate CFTR modulator therapy. A sub-study will evaluate 4D-710 in a cohort of adults with CF advanced lung disease and/or frequent pulmonary exacerbation (PE) while on currently available CFTR modulator therapy.

Study Timeline

• Study First Submitted: 2022-02-10
• Study First Submitted QC: 2022-02-10
• Study First Posted: 2022-02-21
• Study Start: 2022-03-29
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-28
• Last Update Posted: 2024-10-30
• Primary Completion: 2026-12
• Study Completion: 2030-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
4D-710 Phase 2: Dose Expansion	4D-710	Participants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion.
4D-710 Phase 1: Dose Exploration	4D-710	Participants who are ineligible for or intolerant of modulator therapy will receive one of various dose levels of 4D-710 to identify recommended phase 2 dose(s) for further evaluation.
4D-710 Dose Exploration (Sub-Study)	4D-710	Participants who are on currently available CFTR modulator therapy will receive a dose of 4D-710 at various dose levels.

Primary Outcome Measures

Name	Time	Method
Incidence and severity of adverse events	24 Months	Safety and tolerability of 4D-710 following a single inhalation dose, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.

Trial Locations

Locations (16)

University of Alabama Child Health Research Unit; 🇺🇸 Birmingham, Alabama, United States

University of California San Francisco; 🇺🇸 San Francisco, California, United States

National Jewish Health; 🇺🇸 Denver, Colorado, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Northwestern Memorial Hospital; 🇺🇸 Chicago, Illinois, United States

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Penn State Health; 🇺🇸 Hershey, Pennsylvania, United States

The Hospital of the University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

The University of Texas Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States

Virginia Commonwealth University Health System; 🇺🇸 Richmond, Virginia, United States

University of Washington Medical Center; 🇺🇸 Seattle, Washington, United States