Clinical trial for Cystic Fibrosis
Official title A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
ClinicalTrials.gov ID: NCT06526923
What this study is testing
What is SP-101 and doxorubicin Cohort 1?
An investigational inhaled gene therapy combination being studied as a potential treatment for cystic fibrosis. It is designed to deliver a functional CFTR gene to airway cells to help restore normal chloride transport.
Also referred to as SP-101, doxorubicin.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to learn whether SP-101 gene therapy is safe and shows early signs of benefit in adults with cystic fibrosis who are not eligible for or cannot tolerate CFTR modulator drugs.
- Who it's for
- Adults with cystic fibrosis who are ineligible for or intolerant to CFTR modulator therapy.
- What's involved
- Participants receive one dose of the investigational SP-101 gene therapy and attend clinic visits for monitoring, lab tests, and safety assessments.
- Time commitment
- A single treatment with several follow‑up visits over the next few weeks to months to check safety and response.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- Males or females, age 18 to 65 years at Screening Visit, inclusive
- Diagnosis of CF
- ppFEV1 value between 50-100% (inclusive)
- Resting oxygen saturation ≥94% on room air by pulse oximetry 5 . Clinically stable CF disease as assessed by the Investigator and not requiring any...
You likely can't join if
- Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening Visit.
- 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
- Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
- Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
- History of solid organ or hematological transplantation
- History of clinically significant cirrhosis with or without portal hypertension
See the full eligibility criteria
- Males or females, age 18 to 65 years at Screening Visit, inclusive
- Diagnosis of CF
- ppFEV1 value between 50-100% (inclusive)
- 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 treatment treatment within the last 3 months prior to...
- 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.
- 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
- Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
- Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
- History of solid organ or hematological transplantation
- History of clinically significant cirrhosis with or without portal hypertension
- History of pulmonary hypertension
- History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
- 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...
- History of allergic bronchopulmonary aspergillosis (ABPA)
- Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c \>9% at Screening
- Clinically significant laboratory abnormalities at Screening
- people with any medical condition or abnormal laboratory result that, in the opinion of the Investigator, will interfere with the safe completion of the study
- people who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
- people who have previously received any gene therapy agent
- people with known sensitivity to SP-101, doxorubicin or its excipients
The study team makes the final eligibility decision.
Where it is enrolling
- Kansas City, Kansas, United States
- Boston, Massachusetts, United States
- New York, New York, United States
- Philadelphia, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Kansas City, Kansas, United States; Boston, Massachusetts, United States; New York, New York, United States; Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.