A Single Ascending Dose, Phase 1/2 Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SP-101 Via Nebulizer for the Treatment of Cystic Fibrosis (CF)
Spirovant Sciences, Inc.
Summary
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.
Description
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.
Eligibility
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Males or females, age 18 to 65 years at Screening Visit, inclusive 2. Diagnosis of CF 3. ppFEV1 value between 50-100% (inclusive) 4. Resting oxygen saturation ≥94% on room air by pulse oximetry 5 . Clinically stable CF disease as assessed by the Investigator and not requiring any new class of interventional treatment within the last 3 months prior to Screening Exclusion Criteria: 1. Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening Visit. However, inhaled beta-agonists can be included within 2 weeks prior to Screen…
Interventions
- Combination ProductSP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
- Combination ProductSP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Locations (4)
- University of Kansas Medical CenterKansas City, Kansas
- Boston Children's Hospital, Brigham & Women's HospitalBoston, Massachusetts
- Columbia UniversityNew York, New York
- Hospital at University of PennsylvaniaPhiladelphia, Pennsylvania