A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

Recruiting now · Phase 1/Phase 2

Conditions studied: Cystic Fibrosis

In brief

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.

Key facts

Study ID

NCT06526923

Run by

Spirovant Sciences, Inc.

People needed

15

Starts

2024-09-16

Expected to finish

2026-12-31

Last updated by the study team

2024-11-25

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Kansas Medical Center — Kansas City, Kansas, United States (enrolling)
• Boston Children's Hospital, Brigham & Women's Hospital — Boston, Massachusetts, United States (enrolling)
• Columbia University — New York, New York, United States (enrolling)
• Hospital at University of Pennsylvania — Philadelphia, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.