4D-710 in Adult Patients With Cystic Fibrosis

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
4D Molecular Therapeutics
Study ID
NCT05248230
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Cystic Fibrosis Lung

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • 4D-710 — BIOLOGICAL
    4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR).

Study Details

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

Key Dates

Start date
Mar 29, 2022
Status verified
Mar 2026
Primary completion
Jun 30, 2027
Completion
Jun 30, 2031

Study Design

Enrollment
30 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: 4D-710 Phase 1: Dose Exploration
    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.
  • Experimental: 4D-710 Phase 2: Dose Expansion
    Participants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion.
  • Experimental: 4D-710 Dose Exploration (Sub-Study)
    Participants who are on currently available CFTR modulator therapy will receive a dose of 4D-710 at various dose levels.

Primary Outcome Measure

Incidence and severity of adverse events [ Time Frame: 60 Months ]

Central Contacts

Locations (20)

FacilityCityStateZIPSite coordinators
University of Alabama Child Health Research UnitBirminghamAlabama35233
Justin Anderson
[email protected]
George M. Solomon, MD (PRINCIPAL_INVESTIGATOR)
The University of ArizonaTucsonArizona85724
Elizabeth Ryan
[email protected]
Cori L Daines, MD (PRINCIPAL_INVESTIGATOR)
University of California San FranciscoSan FranciscoCalifornia94143
Legna Betancourt
[email protected]
Jonathan Singer, MD (PRINCIPAL_INVESTIGATOR)
National Jewish HealthDenverColorado80206
Jennifer Taylor-Cousar, MD
[email protected]
Michele Gaffigan
[email protected]
Jennifer Taylor-Cousar, MD (PRINCIPAL_INVESTIGATOR)
University of FloridaGainesvilleFlorida32610
Chrystal Bailey
[email protected]
Cesar Trillo-Alvarez, MD (PRINCIPAL_INVESTIGATOR)
University of Miami HospitalMiamiFlorida33136
Ivan Whitaker
[email protected]
305-799-9209
Maria G Tupayachi Ortiz, MD (PRINCIPAL_INVESTIGATOR)
Northwestern Memorial HospitalChicagoIllinois60611
Rachel Nelson
[email protected]
Manu Jain, MD (PRINCIPAL_INVESTIGATOR)
University of Kansas Medical CenterKansas CityKansas66160
Lawrence Scott
[email protected]
Joel Mermis, MD (PRINCIPAL_INVESTIGATOR)
Johns Hopkins HospitalBaltimoreMaryland21287
Kaia Houtman
[email protected]
Noah Lechtzin, MD (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
Monica Ulles
[email protected]
Alicia Casey, MD (PRINCIPAL_INVESTIGATOR)
Massachusetts General HospitalBostonMassachusetts02114
Margot Hardcastle
[email protected]
Allen Lapey, MD (PRINCIPAL_INVESTIGATOR)
University of North Carolina at Chapel HillChapel HillNorth Carolina27599-
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical CenterClevelandOhio44146
Alex Gifford, MD (PRINCIPAL_INVESTIGATOR)
Nationwide Children's HospitalColumbusOhio43205
Terri Johnson
[email protected]
Karen S. McCoy, MD (PRINCIPAL_INVESTIGATOR)
Penn State HealthHersheyPennsylvania17033
Judie A. Howrylak, MD, PhD (PRINCIPAL_INVESTIGATOR)
The Hospital of the University of PennsylvaniaPhiladelphiaPennsylvania19104
Melissa Molter
[email protected]
Daniel Dorgan, MD (PRINCIPAL_INVESTIGATOR)
Medical University of South CarolinaCharlestonSouth Carolina29425
Cameron Mathison
[email protected]
Patrick Flume, MD (PRINCIPAL_INVESTIGATOR)
The University of Texas Southwestern Medical CenterDallasTexas75390
Maria McLeod
[email protected]
Raksha Jain, MD (PRINCIPAL_INVESTIGATOR)
Virginia Commonwealth University Health SystemRichmondVirginia23298
Margaret K. Lessard
[email protected]
Nauman A. Chaudary, MD (PRINCIPAL_INVESTIGATOR)
University of Washington Medical CenterSeattleWashington98195
Roshni Prabhu
[email protected]
Christopher Goss, MD (PRINCIPAL_INVESTIGATOR)

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By research site
University of Alabama Child Health Research Unit· Birmingham, ALThe University of Arizona· Tucson, AZUniversity of California San Francisco· San Francisco, CANational Jewish Health· Denver, COUniversity of Florida· Gainesville, FLUniversity of Miami Hospital· Miami, FL

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