National Jewish Health | Clinical Research Services, Denver, CO
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This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with CF who are ineligible for or unable to tolerate CFTR modulator therapy.
Full description
This Phase 1/2 trial will evaluate the safety, tolerability, and preliminary efficacy of 4 dose levels of 4D-710, an investigational gene therapy, in adults with cystic fibrosis lung disease who are ineligible or unable to tolerate CFTR modulator therapy.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Key Inclusion Criteria:
18 years and older
Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
Sweat chloride ≥ 60 mmol/L
Mutation Status
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 ≤100% of predicted (per Global Lung Function Initiative) at Screening
Resting oxygen saturation ≥ 92% on room air at Screening
Key Exclusion Criteria:
Any prior gene therapy for any indication (Exception: mRNA-based 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
Two or more pulmonary exacerbations requiring treatment with intravenous (IV) antibiotics within 6 months prior to Screening
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:
Requirement for continuous or night-time oxygen supplementation
Known CF liver disease with evidence of cirrhosis
History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
Primary purpose
Allocation
Interventional model
Masking
24 participants in 5 patient groups
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Central trial contact
4DMT Patient Advocacy
Data sourced from clinicaltrials.gov
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