4D-710 in Adult Patients With Cystic Fibrosis
ConditionCystic Fibrosis Lung
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
Sponsor4D Molecular Therapeutics
About this trial
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
18 years and older
Sweat chloride ≥ 60 mmol/L
Mutation Status
Bi-allelic mutations in the CFTR gene, or
Disqualifiers
Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)
Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
Smoking (e.g. cigarettes, cigars, cannabis) or use of a vaping product during the 2 months prior to screening; must agree to not smoke or use vaping products during Screening and initial 24-month observation period.
Trial design
Design model
Sequential
Treatments tested in this trial
4D-710
Biological/Vaccine4D-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).
Treatment groups
30 Participants
are divided into 3 treatment groupsGroup A: 4D-710 Phase 1: Dose ExplorationExperimental treatment 1 intervention
Group B: 4D-710 Phase 2: Dose ExpansionExperimental treatment 1 intervention
Group C: 4D-710 Dose Exploration (Sub-Study)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence and severity of adverse events
Safety and tolerability of 4D-710 following a single dose via inhalation, 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.
Time frame
60 MonthsSponsors and contacts
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Source ClinicalTrials.gov