RECRUITING

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

Conditions

Cystic Fibrosis gene therapy SP-101 AAV doxorubicin inhaled augmenter

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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)

Quick Facts

Study Start:2024-09-16

Study Completion:2026-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06526923

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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	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

Inclusion Criteria

Exclusion 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

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

Contacts and Locations

Study Contact

Spirovant.ClinicalTrials

CONTACT

(267) 805-6747

clinicaltrials@spirovant.com

Principal Investigator

Jessica Lee, MPH

STUDY_DIRECTOR

Spirovant Sciences

Study Locations (Sites)

University of Kansas Medical Center

Kansas City, Kansas, 66160

United States

Boston Children's Hospital, Brigham & Women's Hospital

Boston, Massachusetts, 02115

United States

Columbia University

New York, New York, 10032

United States

Hospital at University of Pennsylvania

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: Spirovant Sciences, Inc.

Jessica Lee, MPH, STUDY_DIRECTOR, Spirovant Sciences

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-09-16

Study Completion Date2026-12-31

Study Record Updates

Study Start Date2024-09-16

Study Completion Date2026-12-31

Terms related to this study

Keywords Provided by Researchers

gene therapy
SP-101
AAV
doxorubicin
inhaled
augmenter

Additional Relevant MeSH Terms

Cystic Fibrosis