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| [ ...Back to search results ] | [ Print-friendly version ] |
| Sirolimus Multicentre International Lymphangioleiomyomatosis Efficacy and Safety trial (the MILES trial): Canadian component |
| ISRCTN | ISRCTN13402678 |
| ClinicalTrials.gov identifier | NCT00414648 |
| Public title | Sirolimus Multicentre International Lymphangioleiomyomatosis Efficacy and Safety trial (the MILES trial): Canadian component |
| Scientific title | Sirolimus Multicentre International Lymphangioleiomyomatosis Efficacy and Safety: Canadian component - a placebo-controlled, randomised, parallel group trial |
| Acronym | MILES |
| Serial number at source | MCT-83051 |
| Study hypothesis |
1. Implement a double-blind, placebo-controlled, 'intention to treat' based, multicentre protocol for the determination of the safety and efficacy of sirolimus in patients with lymphangioleiomyomatosis (LAM)
2. Determine the correlation between changes in lung function and questionnaire-based assessments of dyspnoea, quality of life, fatigue, and degree of health impairment in LAM trial patients who are taking sirolimus or placebo |
| Lay summary | |
| Ethics approval |
1. Research Ethics Board of University Health Network, Toronto (Canada) approved on 07/12/2007 (ref: 07-095-A)
2. Institutional Review Board of Niigata University Medical and Dental Hospital (Japan) approved on the 24/07/2007 (ref: NH19-003) 3. Institutional Review Board (Federalwide Assurance # 00002988) of Cincinnati Children’s Hospital Medical Center, Cincinnati (USA) approved on 11/01/2008 (ref: 04-11-17) Ethics approval from Australia pending. |
| Study design | International, multicentre, placebo-controlled, randomised, parallel group trial |
| Countries of recruitment | Australia, Canada, Japan, United States of America |
| Disease/condition/study domain | Lymphangioleiomyomatosis (LAM) |
| Participants - inclusion criteria |
1. Women, aged 18 years and older
2. Signed and dated informed consent 3. Diagnosis of LAM as determined by biopsy (lung, abdominal mass, lymph node or kidney) and chest computed tomography [CT] scan findings compatible with LAM; or compatible chest CT scan findings in the setting of tuberous sclerosis, angiomyolipomata (diagnosed by CT, magnetic resonance imaging [MRI] or biopsy), or chylous pleural effusion (verified by thoracentesis) 4. Forced expiratory volume in one second (FEV1) less than or equal to 70% of predicted during baseline visit after bronchodilator |
| Participants - exclusion criteria |
1. History of myocardial infarction, angina or stroke related to atherosclerosis
2. Pregnancy or breast feeding, inadequate contraception 3. Significant haematologic or hepatic abnormality (i.e., transaminase levels greater than 3 times the upper limit of normal range, haematocrit [HCT] less than 30%, platelets less than 80,000/cumm, adjusted absolute neutrophil count less than 1,000/cumm, total white blood cell count [WBC] less than 3,000/cumm) 4. Intercurrent infection at initiation of sirolimus 5. Recent surgery 2 months (involving entry into a body cavity or requiring sutures) 6. Use of an investigational drug within the last 30 days 7. Uncontrolled hyperlipidaemia 8. Previous lung transplantation 9. Inability to attend scheduled clinic visits 10. Inability to give informed consent 11. Inability to perform pulmonary function testing 12. Creatinine greater than 2.5 mg/dl (greater than 220 µmol/L) 13. Chylous ascites sufficient to affect diaphragmatic function on pulmonary function tests (PFTs) 14. Pleural effusion sufficient to affect pulmonary function based on the opinion of the Site Investigator (generally greater than 500 cc) 15. Acute pneumothorax within the past 2 months (as pneumothorax can alter baseline PFT results) 16. History of malignancy in the past two years, other than squamous or basal cell skin cancer 17. Use of oestrogen containing medications |
| Anticipated start date | 01/06/2007 |
| Anticipated end date | 31/12/2010 |
| Status of trial | Completed |
| Patient information material | |
| Target number of participants | 260 |
| Interventions |
This is a multicentre, international, intention to treat, placebo-controlled, randomised parallel, drug/diagnostic, efficacy and safety trial with study participant, study investigator, caregiver, outcome assessor, and data analyst blinded.
1. Experimental group: sirolimus (rapamycin), daily dose of 2 mg during 12 months; follow up only for additional 12 months 2. Control group: matching placebo of active treatment, 1 pill daily for 12 months; follow up only for additional 12 months Contact for public queries same as below. Please note that you can also contact Dr Lianne Singer at lianne.singer@uhn.on.ca for details on this trial (scientific and public queries). |
| Primary outcome measure(s) | FEV1 slope response, measured at 12 months. |
| Secondary outcome measure(s) |
Measured at 12 months:
1. Six minute walk testing 2. Quantitative CT of the lung 3. Quality of life questionnaires (St George Respiratory Questionnaire, 36-item short form health survey [SF-36]) 4. Forced vital capacity (FVC) 5. Diffusing capacity of the lung for carbon monoxide (DLCO) |
| Sources of funding | Canadian Institutes of Health Research (CIHR) (Canada) - http://www.cihr-irsc.gc.ca (ref: MCT-83051) |
| Trial website | http://rarediseasesnetwork.epi.usf.edu/rldc/centers/uhn-toronto.htm |
| Publications | 2011 results in http://www.ncbi.nlm.nih.gov/pubmed/21410393 |
| Contact name | Dr Gregory Paul Downey |
| Address |
11C-1183
Toronto General Hospital |
| City/town | Toronto, Ontario |
| Zip/Postcode | M5G 1L7 |
| Country | Canada |
| Tel | +1 416 340 4996 |
| Fax | +1 416 340 3609 |
| gregory.downey@uhn.on.ca | |
| Sponsor | University Health Network (Toronto) (Canada) |
| Address | 200 Elizabeth Street |
| City/town | Toronto, Ontario |
| Zip/Postcode | M5G 2C4 |
| Country | Canada |
| Sponsor website: | http://www.uhn.ca/index.htm |
| Date applied | 04/02/2008 |
| Last edited | 31/05/2011 |
| Date ISRCTN assigned | 04/02/2008 |
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| © 2012 ISRCTN unless otherwise stated. | |
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