Gene Therapy for Cystic Fibrosis
(SAAVe Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you should not have any changes in your established pulmonary treatment within 28 days before the screening visit. It's best to discuss your specific medications with the trial team.
How is the SP-101 treatment for cystic fibrosis different from other treatments?
What data supports the effectiveness of the treatment SP-101, Spirovant's AAV gene therapy, for cystic fibrosis?
Research on similar AAV-based gene therapies for cystic fibrosis has shown that while they are safe and well-tolerated, they have not yet demonstrated significant improvements in lung function. However, the approach holds promise as gene therapy is considered a potential way to correct the genetic defect causing cystic fibrosis.12467
Who Is on the Research Team?
Jessica Lee, MPH
Principal Investigator
Spirovant Sciences
Are You a Good Fit for This Trial?
This trial is for adults with Cystic Fibrosis who can't take or haven't had success with CFTR modulator therapy. Specific details about eligibility are not provided, but typically participants need to meet certain health standards and may be excluded based on factors that could impact the study's safety or results.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Single inhalational administration of SP-101 and doxorubicin at various dose levels
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- SP-101
Find a Clinic Near You
Who Is Running the Clinical Trial?
Spirovant Sciences, Inc.
Lead Sponsor