Investigating SP-101 gene therapy for cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1/2 multicenter trial evaluates the safety and effectiveness of SP-101, an investigational gene therapy, in adults with cystic fibrosis who cannot tolerate or are ineligible for CFTR modulator therapy. The study involves administering single ascending doses of SP-101 alongside doxorubicin to assess pharmacokinetics and pharmacodynamics. Participants will be closely monitored for safety and clinical outcomes throughout the trial.

Why it matters

Who can join: the full rules

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

Where it's running

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

Study contacts

• Study coordinator: Spirovant.ClinicalTrials
• Email: clinicaltrials@spirovant.com
• Phone: (267) 805-6747

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.