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

Phase 1

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Combination Product: SP-101 and doxorubicin Cohort 2; Combination Product: SP-101 and doxorubicin Cohort 1

• Registration Number: NCT06526923
• Lead Sponsor: Spirovant Sciences, Inc.

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.

Study Timeline

• Study First Submitted: 2024-07-18
• Study First Submitted QC: 2024-07-24
• Study First Posted: 2024-07-30
• Study Start: 2024-09-16
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-21
• Last Update Posted: 2024-11-25
• Primary Completion: 2026-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 2	SP-101 and doxorubicin Cohort 2	Single inhalational administration of SP-101 and doxorubicin Dose 2
Cohort 1	SP-101 and doxorubicin Cohort 1	Single inhalational administration of SP-101 and doxorubicin Dose 1
Dose Expansion	SP-101 and doxorubicin Cohort 1	Single inhalational administration of SP-101 and doxorubicin Selected Dose
Dose Expansion	SP-101 and doxorubicin Cohort 2	Single inhalational administration of SP-101 and doxorubicin Selected Dose

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (4)

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States

Boston Children's Hospital, Brigham & Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

Columbia University; 🇺🇸 New York, New York, United States

Hospital at University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States