NCT06526923 · Spirovant Sciences, Inc.
A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
(SAAVe)
What this study is about
This is a Phase 1/2 conducted at multiple hospitals, where both patients and doctors know the treatment given, single dose trial of SP-101 experimental gene therapy in adults with CF who are ineligible for or intolerant to CFTR modulator therapy.
View original scientific 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.
Interventions
COMBINATION_PRODUCT
SP-101 and doxorubicin Cohort 1
Single inhaled dose of SP-101 and doxorubicin Dose 1
COMBINATION_PRODUCT
SP-101 and doxorubicin Cohort 2
Single inhaled dose of SP-101 and doxorubicin Dose 2
Primary outcome measures
Incidence and severity of adverse events
Time frame: 52 weeks
Safety and tolerability of SP-101 following a single inhalation dose, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
Exclusion criteria
- 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
Where
- Kansas City, Kansas
- Boston, Massachusetts
- New York, New York
- Philadelphia, Pennsylvania
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Nov 25, 2024 · Source of record for eligibility and locations