Cystic Fibrosis
Conditions
Keywords
gene therapy, SP-101, AAV, doxorubicin, inhaled, augmenter
Brief 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.
Detailed 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.
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
Sponsors
Spirovant Sciences, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single ascending dose
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
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 Screening Visit. 2. Clinically significant episode of hemoptysis (\>50 mL or ¼ cup or 10 teaspoons per day) within 12 weeks prior to dosing with study drug on Day 1 3. Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status 4. Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa 5. History of solid organ or hematological transplantation 6. History of clinically significant cirrhosis with or without portal hypertension 7. History of pulmonary hypertension 8. History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy 9. Current active fungal infection (not just a positive culture), acute blood, lung, or bladder infection, clinically significant hepatic or renal dysfunction, and/or viral infection (including human immunodeficiency virus or hepatitis virus B or C) requiring the initiation of new therapy within 30 days prior to Screening 10. History of allergic bronchopulmonary aspergillosis (ABPA) 11. Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c \>9% at Screening 12. Clinically significant laboratory abnormalities at Screening 13. Subjects with any medical condition or abnormal laboratory result that, in the opinion of the Investigator, will interfere with the safe completion of the study 14. Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening 15. Subjects who have previously received any gene therapy agent 16. Subjects with known sensitivity to SP-101, doxorubicin or its excipients
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events | 52 weeks | 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. |
Countries
United States
Contacts
Primary ContactSpirovant.ClinicalTrials
Outcome results
None listed