A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
NCT ID: NCT06526923
Last Updated: 2024-11-25
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2024-09-16
2026-12-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis
NCT03229252
A Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis
NCT03056989
Safety Study of Inhaled 552-02 in Cystic Fibrosis Patients
NCT00274313
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT05668741
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis
NCT06429176
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Cohort 1
Single inhalational administration of SP-101 and doxorubicin Dose 1
SP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
Cohort 2
Single inhalational administration of SP-101 and doxorubicin Dose 2
SP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Dose Expansion
Single inhalational administration of SP-101 and doxorubicin Selected Dose
SP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
SP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
SP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
SP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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
18 Years
65 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Spirovant Sciences, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Jessica Lee, MPH
Role: STUDY_DIRECTOR
Spirovant Sciences
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
References
Explore related publications, articles, or registry entries linked to this study.
Excoffon KJDA, Smith MD, Falese L, Schulingkamp R, Lin S, Mahankali M, Narayan PKL, Glatfelter MR, Limberis MP, Yuen E, Kolbeck R. Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRDeltaR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis Ferrets. Hum Gene Ther. 2024 Sep;35(17-18):710-725. doi: 10.1089/hum.2024.064. Epub 2024 Sep 4.
Excoffon KJDA, Lin S, Narayan PKL, Sitaraman S, Jimah AM, Fallon TT, James ML, Glatfelter MR, Limberis MP, Smith MD, Guffanti G, Kolbeck R. SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis. Hum Gene Ther. 2024 Sep;35(17-18):695-709. doi: 10.1089/hum.2024.063. Epub 2024 Aug 29.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
CFAAV-001
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.