Drug trial to assess a new inhaled treatment for women with LAM

A new study is planned to investigate a possible inhaled treatment, called LAM-001 for women with LAM. The study is planned to begin in April and will be conducted at sites in Nottingham, Manchester and London.

Women aged between 18 and 65 years with LAM, who are not being treated with sirolimus (also called rapamycin), may be eligible to participate in a new study. The purpose of this trial is to assess the safety of LAM-001 a new inhaled drug, which is being investigated for treatment of LAM. The study will assess how much of the drug is absorbed and how it is processed in the body.

About the study: Participants will receive the study drug, LAM-001 for 14 days.

The entire duration of the study is approximately 10 weeks. Up to 6 patients may be enrolled. The first part of the study is called the screening period, which may last up to 4 weeks. The second part of the study is called treatment period which lasts 2 weeks. In this part of the study, you will receive the study drug for 14 days. The last part of the study consists of an end-of-study visit which will occur approximately 4 weeks after the last dose of study drug. Five visits to the study centre will be required throughout the study and 8 additional blood samples will need to be taken outside of the visits listed above. The study centres offer the option for Home Health Care visits as an alternative to attending the unit to have these blood samples taken.

Expenses: All study procedures will be provided at no expense. Participants will receive a payment, based on the number of visits completed and these are intended to cover all out of pocket expenses including meals, as well as the participants time spent in the research unit and for undergoing the study procedures. This study is also offering a Travel Assistance Programme which will be paid for by the Sponsor. This program is designed to assist participants with their travel needs to and from the study appointments.

How do I get involved: If you are interested in participating in the study or in learning more information, please contact one of the study centres listed below.

Nottingham University Hospitals NHS Trust: – Nottingham, UK; Professor Tim Harrison/ Professor Simon Johnson (Lead Investigators in UK)

Contact: Nik Neesham

Email: Nicola.neesham@nuh.nhs.uk

Telephone: 0115 840 8444

Medicines Evaluation Unit – Manchester, UK; Dr. Catalina Panainte

Contact: Simon Pearson

Email: SPEARSON@MEU.ORG.UK

Telephone: 0800 655 6553 / 0161 946 4059

The Royal Brompton Hospital– London, UK; Dr. Toby Maher

Contact: Lyn Paul

Email: l.paul@rbht.nhs.uk

Telephone: 0207 352 8121 ext. 4151