RecruitingPhase 2NCT05248230
4D-710 in Adult Patients With Cystic Fibrosis
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
Sponsor
4D Molecular Therapeutics
Enrollment
30 participants
Start Date
Mar 29, 2022
Study Type
INTERVENTIONAL
Conditions
Summary
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Eligibility
Min Age: 18 Years
Inclusion Criteria17
- years and older
- Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
- Sweat chloride ≥ 60 mmol/L
- Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease
- Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects.
- Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening
- Resting oxygen saturation ≥ 92% on room air at Screening
- years and older
- Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
- Sweat chloride ≥ 60 mmol/L
- Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease
- Currently on a stable dose of CFTR modulator therapy for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-month Observation Period
- FEV1 ≥ 40% and \< 70% predicted (per Global Lung Function Initiative) at Screening, AND/OR experienced at least 2 pulmonary exacerbations in the last year requiring intravenous antibiotics
Exclusion Criteria18
- 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.
- Contraindication to systemic corticosteroid therapy
- Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
- If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥ 6.5% at Screening
- If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C \> 7.5% at Screening
- Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment
- Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia
- Body Mass Index (BMI) \< 16
- Laboratory abnormalities at screening:
- ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
- Total bilirubin ≥ 2 × ULN
- Hemoglobin \< 10 g/dL
- Requirement for continuous or night-time oxygen supplementation
- Known CF liver disease with evidence of multilobular cirrhosis
- History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
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Interventions
BIOLOGICAL4D-710
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).
Locations(20)
View Full Details on ClinicalTrials.gov
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NCT05248230
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