A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
NCT ID: NCT01059318
Last Updated: 2020-11-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
24 participants
INTERVENTIONAL
2010-01-31
2012-06-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Everolimus
All patients received a starting dose of everolimus 2.5mg/day for 4 weeks, followed by a dose of 5 mg/day for 4 weeks and finally a dose of 10mg/day for 18 weeks.
The 26 week treatment period was followed by an optional extension period wherein patients continued therapy until the last patient had completed 26-weeks of treatment. The longest period a patient participated in the study was 62 weeks.
Everolimus
Everolimus was formulated as tablets in strengths of 2.5mg, 5mg and 10mg.
Interventions
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Everolimus
Everolimus was formulated as tablets in strengths of 2.5mg, 5mg and 10mg.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Pulmonary function abnormalities as follows:
* FEV1 of ≤ 80% of the predicted value following administration of a standard dose of a short acting β2-agonist (\*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) OR
* FEV1 \< 90% of the predicted value of bronchodilator following administration of a standard dose of a short acting β2-agonist (\*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) and DLco (uncorrected) \<80% predicted.
* Female patients including those of childbearing potential will be included in this study.
* Negative pregnancy test at screening and baseline
Exclusion Criteria
* Change in FVC (ml) \> ± 15% of screening value at baseline visit (not less than 14d after screening visit).
* Use of any medicine containing estrogen in the 4 months prior to the screening visit and for the duration of the study
* Significant hematologic, renal, hepatic laboratory abnormality or amylase \> 1.5x the upper limit of the normal range at the screening or baseline visits
* Fasting blood glucose \> 126mg/dl or random blood glucose \>200mg/dl at screening and/or baseline
* Recent surgery (involving entry into a body cavity or requiring sutures) within 2 months of the screening visit or any evidence of unhealed surgical wound.
* Uncontrolled hyperlipidemia (defined as persistent elevation of total cholesterol or triglycerides \>6.5nM/L) or a history of clinical atherosclerotic disease including heart attack, angina, peripheral vascular disease or stroke.
* Previous organ transplantation
* Inability to give informed consent
* Inability to perform pulmonary function or 6 minute walk tests and imaging assessments
18 Years
FEMALE
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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Center for LAM Research and Clinical Care
Boston, Massachusetts, United States
University of Cincinnati, Department of Internal Medicine, Pulmonary, Critical Care & Sleep Medicine,
Cincinnati, Ohio, United States
Novartis Investigative Site
Lyon, , France
Novartis Investigative Site
Milan, , Italy
Countries
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References
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McCormack FX, Inoue Y, Moss J, Singer LG, Strange C, Nakata K, Barker AF, Chapman JT, Brantly ML, Stocks JM, Brown KK, Lynch JP 3rd, Goldberg HJ, Young LR, Kinder BW, Downey GP, Sullivan EJ, Colby TV, McKay RT, Cohen MM, Korbee L, Taveira-DaSilva AM, Lee HS, Krischer JP, Trapnell BC; National Institutes of Health Rare Lung Diseases Consortium; MILES Trial Group. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. N Engl J Med. 2011 Apr 28;364(17):1595-606. doi: 10.1056/NEJMoa1100391. Epub 2011 Mar 16.
Goldberg HJ, Harari S, Cottin V, Rosas IO, Peters E, Biswal S, Cheng Y, Khindri S, Kovarik JM, Ma S, McCormack FX, Henske EP. Everolimus for the treatment of lymphangioleiomyomatosis: a phase II study. Eur Respir J. 2015 Sep;46(3):783-94. doi: 10.1183/09031936.00210714. Epub 2015 Jun 25.
Other Identifiers
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2010-019825-32
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CRAD001X2201
Identifier Type: -
Identifier Source: org_study_id