What is SP-101 studying for cystic fibrosis?

SP-101 is being studied as a gene therapy for people with cystic fibrosis who cannot use or tolerate current CFTR modulator treatments. Researchers aim to evaluate its safety, how the body processes it, and its biological effects. The trial is sponsored by Spirovant Sciences, Inc.

Who may be eligible for this cystic fibrosis clinical trial?

Adults aged 18 to 65 with a confirmed diagnosis of cystic fibrosis may be eligible. Participants must have stable lung function and oxygen levels, and not have recent changes in lung treatments or prior gene therapy. Please review eligibility criteria for more details.

Is there any cost to participate in this cystic fibrosis clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly.

Actively Recruiting

Phase 1

Phase 2

Age: 18Years - 65Years

All Genders

ID06526923

Phase 12 Study of Single-Dose SP-101 Gene Therapy Delivered by Nebulizer to Treat Cystic Fibrosis in Adults Not Eligible for CFTR Modulators

Led by Spirovant Sciences, Inc. · Updated on 2024-11-25

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating SP-101, an investigational gene therapy, in adults with cystic fibrosis CF who cannot use or tolerate CFTR modulator therapy. This Phase 12, first-in-human study aims to assess the safety, how the body processes the drug, and its biological effects with varying doses. The trial is open-label and conducted at multiple centers. Participants receive a single inhaled dose of SP-101 combined with doxorubicin at different dose levels. The study includes three groups Dose 1, Dose 2, and a selected dose expansion group. Each participant will receive only one inhalation treatment during the trial. During the study, researchers will monitor participants for adverse events over 52 weeks to evaluate safety. They will also measure how the drug behaves in the body and its impact. Participants will have regular assessments including lung function tests and oxygen levels. The trial lasts about a year, during which participants will be closely observed and evaluated.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 65Years

All Genders

Eligibility Criteria

You may qualify if you...

Males or females aged 18 to 65 years at Screening Visit, inclusive
Diagnosis of cystic fibrosis
ppFEV1 value between 50-100% (inclusive)
Resting oxygen saturation of 94% or higher on room air by pulse oximetry
Clinically stable cystic fibrosis disease not requiring new interventional treatment within 3 months prior to Screening

You will not qualify if you...

Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening, except inhaled beta-agonists within 2 weeks prior
Clinically significant hemoptysis (>50 mL or 1/4 cup per day) within 12 weeks prior to dosing
Lung infection with Mycobacterium abscessus causing rapid decline
Active treatment for lung infection with Burkholderia cenocepacia or Burkholderia dolosa
History of solid organ or blood transplantation
History of clinically significant cirrhosis with or without portal hypertension
History of pulmonary hypertension
History of cardiotoxicity, coronary artery disease, or cardiomyopathy
Current active fungal infection, acute blood, lung, or bladder infection, significant liver or kidney dysfunction, or viral infection requiring new therapy within 30 days prior
History of allergic bronchopulmonary aspergillosis (ABPA)
Uncontrolled diabetes with hemoglobin A1c >9% at Screening
Clinically significant lab abnormalities at Screening
Any medical condition or lab result interfering with safe study completion
Receipt of any investigational products within 30 days or 5 half-lives prior to Screening
Prior gene therapy
Known sensitivity to SP-101, doxorubicin, or excipients

Research Team

Spirovant.ClinicalTrials

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Phase 12 Study of Single-Dose SP-101 Gene Therapy Delivered by Nebulizer to Treat Cystic Fibrosis in Adults Not Eligible for CFTR Modulators

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria