This is a Phase 1/2 multicenter, open-label, single dose trial of SP-101 investigational gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy.
This multi-center study is a first-in-human, single ascending dose, Phase 1/2 trial to evaluate the safety, pharmacokinetics, and pharmacodynamics of various dose levels in people with CF who are ineligible or intolerant to CFTR modulator therapy.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Single inhaled dose of SP-101 and doxorubicin Dose 1
Single inhaled dose of SP-101 and doxorubicin Dose 2
University of Kansas Medical Center
Kansas City, Kansas, United States
RECRUITINGBoston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, United States
RECRUITINGColumbia University
New York, New York, United States
Incidence and severity of adverse events
Safety and tolerability of SP-101 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.
Time frame: 52 weeks
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Hospital at University of Pennsylvania
Philadelphia, Pennsylvania, United States
RECRUITING