June 2007 Miles Trial Update

Researcher Dr Liane Singer, at the Toronto General Hospital has applied to have Toronto as a site for the MILES trial.   At time of writing, (November 2007), the Toronto site has not yet been approved.

The MILES trial has already been recruiting LAM women at the Cincinnati site and the University of Southern Carolina (Charleston). Other sites will be added in the near future.

The MILES TRIAL (The Multicenter International Lymphangioleiomyomatosis Efficacy and Safety Trial: A Phase III, Randomized, Double-Blind, Placebo Controlled Study) DESCRIPTION

Group/Participating Institutions: The University of Cincinnati, The Children's Hospital Medical Center of Cincinnati, and 7-8 other universities affiliated with the Rare Lung Disease Consortium.
Investigator Name: Francis X. McCormack, M.D.
Main Contact Name: Francis X. McCormack, M.D.
Mailing Address: 231 Albert Sabin Way, MSB 6007, Cincinnati, OH 45267-0564
Telephone: 513-558-4831
Fax: 513-558-4858
E-mail: frank.mccormack@uc.edu

Why do this study?

Lymphangioleiomyomatosis (LAM) is an uncommon lung disease that affects women almost exclusively. LAM is associated with mutations in tuberous sclerosis genes, whether it occurs in patients with tuberous sclerosis complex or sporadically in patients without inherited disease. LAM has an insidious onset, and is typically slowly progressive. Infiltration of the tissues in the lung with atypical smooth muscle cells and destruction of the lung by cysts are hallmarks of the disease. The average survival of patients who see their physicians because of shortness of breath is thought to be in the range of 8.5 to 15 yrs. There are currently no cure or proven therapies for LAM.

Objectives:

1) To assess the safety of the drug Sirolimus (also called rapamycin or rapamune) given orally compared placebo (sugar pill) in patients with LAM

2) To assess the effect of Sirolimus on biological and clinical markers of lung function, including spirometry (pulmonary function tests), dyspnea (shortness of breath), quality of life, lung volumes, diffusion, oxygenation, and exercise tolerance.

Study Design: The study design will be a randomized, double-blind, placebo-controlled study of the safety and clinical effects of Sirolimus administered orally to patients with LAM. This means that LAM patients will be randomly assigned (similar to flipping a coin) to get either Sirolimus or a placebo. Neither the patient nor the treating physician will know which patients get the drug and which patients get the placebo.

Study subjects with LAM will be given Sirolimus 2 mg orally once per day or a placebo (that looks the same as the Sirolimus) for a period of 12 months and will be followed for months.

Investigators will be checking the results of patients' tests at 6, 12 and at 24 months. Eight visits to the clinical site will be required.

Population: The study population consists of women with LAM who are over 18 years of age and do not have other illnesses. Patients enter the trial with or without other treatments such as progesterone.

Number of Patients: Two hundred forty patients are wanted to enrol into the trial in total (120 active drug and 120 placebo). At least 10 patients will be required at each site in order for that site to participate.

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