SAAVe - a Phase 1/2 trial to evaluate SP-101 via nebulizer for treatment of CF
Research type
Research Study
Full title
A single ascending dose, Phase 1/2 trial to evaluate the safety, pharmacokinetics, and pharmacodynamics of SP-101 via nebulizer for the treatment of cystic fibrosis (CF)
IRAS ID
1008770
Contact name
Jessica Lee
Contact email
Sponsor organisation
Spirovant Sciences Inc.
Research summary
Cystic fibrosis (CF) is an inherited disease that mainly causes damage to the lungs and digestive system. It is caused by a faulty gene. SP-101 (the “study drug”) is an experimental investigational drug for the possible treatment of people with CF. "Investigational” means that the study drug is currently being tested and is not approved for use as a treatment for any disease by any health authority. The study drug is a type of gene therapy drug. Gene therapy is a medical approach that focuses on the correction of faulty genes using modified viruses that contain a "healthy" version of the gene related to the condition. It is thought that the study drug will introduce the healthy gene into the cells of the lungs. This will mean that a "healthier" version of the protein made by the faulty gene responsible for CF will be made, and lung function will be restored.
The main purpose of this study is to find out whether the study drug, with or without doxorubicin (a type of drug used in the treatment of certain types of cancer), is safe and tolerated for people living with CF.
This is a Phase 1/2 clinical research study. This means that this is the first time the study drug will be tested in people. Up to 30 participants in approximately 15-20 study centres globally will take part.
There will be 4 groups in the study and participants will be assigned to groups based on when they join the study. The groups are:
• Group 1: Study drug at a lower dose
• Group 2: Study drug at a higher dose
• Group 3: Study drug at a higher dose followed by doxorubicin at a lower dose
• Group 4: Study drug at a higher dose followed by doxorubicin at a higher dose
Participants will be told which group they are assigned to and which dose of study drug they will receive.
There will also be a dose-expansion group to gather additional information about the study drug's safety and the way the body processes the study drug.REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0978
Date of REC Opinion
7 Feb 2024
REC opinion
Further Information Unfavourable Opinion