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Gene Therapy

Gene Therapy for Cystic Fibrosis (SAAVe Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Spirovant Sciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of CF
FEV1 value between 50-100% (inclusive) of predicted mean for age, sex, race, ethnicity and height measured at Screening (per GLI standards)
Must not have
Subjects who have previously received any gene therapy agent
History of pulmonary hypertension
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new gene therapy called SP-101 in adults with cystic fibrosis who cannot take or do not tolerate CFTR modulator therapy.

Who is the study for?
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.
What is being tested?
The trial is testing SP-101 gene therapy in combination with doxorubicin, an existing medication. There are two different groups (cohorts) receiving varying doses or treatment regimens to evaluate how well the new therapy works and its safety.
What are the potential side effects?
Potential side effects aren't listed, but gene therapies like SP-101 might cause immune reactions, while doxorubicin is known to potentially cause heart damage, nausea, hair loss, and low blood cell counts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with cystic fibrosis.
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My lung function, measured by FEV1, is within 50-100% of the expected range for someone my age, sex, and height.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have never received gene therapy.
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I have a history of high blood pressure in the lungs.
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I am allergic to SP-101, doxorubicin, or similar medications.
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I am being treated for a lung infection caused by Burkholderia.
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I have had an organ or bone marrow transplant.
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I have a history of serious liver disease.
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I have a history of heart issues, including damage from past treatments, known heart disease, or weak heart muscle.
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My diabetes is not under control, with an A1c level over 9%.
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I have not coughed up a significant amount of blood (more than ¼ cup) in the last 3 months.
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I have a lung infection with Mycobacterium abscessus causing rapid health decline.
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I do not have any active infections or significant liver or kidney problems.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence and severity of adverse events

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: Dose ExpansionExperimental Treatment2 Interventions
Single inhalational administration of SP-101 and doxorubicin Selected Dose
Group II: Cohort 2Experimental Treatment1 Intervention
Single inhalational administration of SP-101 and doxorubicin Dose 2
Group III: Cohort 1Experimental Treatment1 Intervention
Single inhalational administration of SP-101 and doxorubicin Dose 1

Find a Location

Who is running the clinical trial?

Spirovant Sciences, Inc.Lead Sponsor
Jessica Lee, MPHStudy DirectorSpirovant Sciences
~10 spots leftby Dec 2026
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