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 very soon and will be conducted at sites in Nottingham, Manchester and London.

Women aged between 18 and 70 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: This is an open-label study (meaning that participants will definitely receive the study drug, there is no placebo), divided into two Periods (phases):

Period 1: Participants will receive the study drug for 14 days.The entire duration of the study is approximately 10 weeks. Up to 6 patients may be enrolled. The first part of this period is called the screening period, which may last up to 4 weeks. The second part of this period is called treatment period with a duration of 2 weeks. In this part of the study, you will receive study drug for 14 days. The last part of the study consists of an end-of-period 1 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.

Period 2: Participants will receive the study drug up to 12 weeks. The entire duration of the study is approximately 20 weeks. Participants in this Period 2 may have participated in previous Period 1 if they still continue to be eligible for the study, or they can be included directly into this Period 2 of the study. 

This Period 2 also has 3 parts. The first part is the screening period, which may last up to 4 weeks. The second part of this period is also called treatment period with a duration of 12 weeks. In this part of the study, you will receive study drug for 84 days. The last part of the study consists of an end-of-period 2 visit which will occur approximately 4 weeks after the last dose of study drug. Up to seven visits to the study centre will be required throughout the study and 4 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 the additional blood samples taken in both Periods.

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 Program 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.

Confidentiality of your data and personal information: The study site will record basic personal details about the participants but they will only be identified by a code. Some people may access these records but to ensure privacy, the participants name and other directly identifying information will not be attached to records or samples released to the Sponsor and its service providers for research purposes. Participants have the right to access, through their study doctor, all the information collected about them and they also have the right to complain about how their information is handled to a supervisory authority that is responsible for enforcing data protection law. In the UK, this is the Office of the Information Commissioner. Participants can find more information about data protection contacting the study centres listed below.

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. Further written information about the study will then also be provided in a patient information sheet.

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

Contact: Liz Dark

Email: Elizabeth.Dark@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

In addition to the trials at the 3 UK centres, there will also shortly be a trial in the Netherlands, under Prof. Grutters at St. Antonius Ziekenhuis in Utrecht which it is hoped will be open by the end of November.