Status:
RECRUITING
A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
Lead Sponsor:
Spirovant Sciences, Inc.
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
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 inel...
Eligibility Criteria
Inclusion
- Males or females, age 18 to 65 years at Screening Visit, inclusive
- Diagnosis of CF
- ppFEV1 value between 50-100% (inclusive)
- 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
- 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.
- 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
- Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
- Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
- History of solid organ or hematological transplantation
- History of clinically significant cirrhosis with or without portal hypertension
- History of pulmonary hypertension
- History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
- 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
- History of allergic bronchopulmonary aspergillosis (ABPA)
- Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c \>9% at Screening
- Clinically significant laboratory abnormalities at Screening
- 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
- Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
- Subjects who have previously received any gene therapy agent
- Subjects with known sensitivity to SP-101, doxorubicin or its excipients
Key Trial Info
Start Date :
September 16 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT06526923
Start Date
September 16 2024
End Date
December 31 2026
Last Update
November 25 2024
Active Locations (4)
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1
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
2
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, United States, 02115
3
Columbia University
New York, New York, United States, 10032
4
Hospital at University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104