Investigating SP-101 gene therapy for cystic fibrosis

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)

This study is testing a new gene therapy called SP-101 to see if it can help adults with cystic fibrosis who can't use standard treatments.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Spirovant Sciences, Inc. (industry)
• Drugs / interventions: doxorubicin
• Locations: 4 sites (Kansas City, Kansas and 3 other locations)
• Trial ID: NCT06526923 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 65 with a confirmed diagnosis of cystic fibrosis and stable disease who are ineligible for or intolerant to existing CFTR modulator therapies.

Not a fit: Patients with significant lung infections or those requiring new interventional treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for cystic fibrosis patients who currently have limited alternatives.

How similar studies have performed: While gene therapy approaches for cystic fibrosis are being explored, this specific trial represents a novel application of SP-101 and has not been previously tested in this context.

Eligibility criteria

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 this trial is running

Kansas City, Kansas and 3 other locations

• 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

Conditions: Cystic Fibrosis, gene therapy, SP-101, AAV, doxorubicin, inhaled, augmenter