Lenticlair (TM) 1: A study to assess how well a new genetic therapy is tolerated and whether it improves lung function in people with CF

Full title

Lenticlair (TM) 1: A phase 1 study to assess how well a new inhaled genetic therapy, BI 3720931, is tolerated, and whether it improves lung function in adults with CF who are ineligible for CFTR modulator therapies.

Study details

Boehringer Ingelheim has made the decision to stop the investigation of the potential cystic fibrosis gene therapy (BI 3720931) and therefore terminated the Lenticlair^TM 1 trial (1504-0001). This decision is based on clinical data that - in the opinion of Boehringer Ingelheim - does not support continuing this trial and does not relate to any changes in the safety profile of the treatment. 

This study, called Lenticlair^TM 1, aims to find out how well a new genetic therapy is tolerated and whether it improves lung function in people with CF. This is the first time that the genetic therapy will be given to humans.

The therapy will be given as an inhaled therapy and is expected to add a healthy copy of the CFTR gene into the cells in the airways. This could help to normalise CFTR function and make the mucus less sticky, improving overall lung function.

Find out more about  genetic therapies and what’s involved in genetic therapy clinical trials.

This study is open to adults with CF who are not eligible to receive CFTR modulator therapies. Women who are of child-bearing potential (premenopausal women who have the potential to become pregnant) are not able to take part. 

Participants will be put in one of three groups, who will begin their part of the trial one after the other. Each group will receive a different dose of BI 3720931. Group 1 starts with the lowest dose, followed by group 2 with the middle dose, and group 3 with the high dose. 

The therapy will be inhaled through a nebuliser. Each participant will receive a single dose of BI 3720931. During the study, participants will continue taking their usual medicines.

Following this dose, doctors will closely monitor participants’ health at the study site for the first 3 days after receiving treatment. Participants will stay in hospital for a total of 6 days and 5 nights. 

After this, they will have regular visits to their usual CF care team. This will be weekly for two visits, then every two weeks for three visits, then every four weeks until the end of the trial participation period. They will check their overall health and note any potential side effects. Study participants will be asked to do regular lung function tests to measure how well their lungs are working. 

Participation in the study lasts for 7 months. After completion of this study, participants will be asked to take part in a long-term follow-up study. The main purpose of this study is to monitor the long-term health of participants who were treated with BI 3720931.

Participants will be in the long-term follow-up study for 15 years. This will involve about 20 site visits. For the first 2 years there will be visits every 3 months. After that, it will be a visit once a year. 

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