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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01687179 |
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Date of registration:
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02/08/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis
SAIL |
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Scientific title:
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Targeting Autophagy for the Treatment of TSC and LAM: a Phase I Trial of Hydroxychloroquine and Sirolimus |
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Date of first enrolment:
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September 2012 |
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Target sample size:
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14 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01687179 |
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Study type:
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Interventional |
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Study design:
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Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Elizabeth P Henske, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Brigham and Women's Hospital |
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Name:
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Joel Moss, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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National Heart, Lung, and Blood Institute (NHLBI) |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Female age 18 or older
- Ability to give informed consent
- Diagnosis of LAM as defined as typical cystic change on CT plus:
- biopsy or cytology of any tissue demonstrating LAM
- angiomyolipoma, chylothorax, lymphangioleiomyoma, or tuberous sclerosis
- serum VEGFD greater or equal to 800pg/ml
- Post-bronchodilator FEV1 equal or less than 80% of predicted or DLCO equal equal or
less than 70% of predicted, or RV > 120% of predicted at baseline
- Women of childbearing potential must agree to use 2 forms of barrier contraception
during and for 8 weeks after the last dose of medication.
Exclusion Criteria:
- History of intolerance of mTOR inhibitors
- History of intolerance to hydroxychloroquine
- History of severe psoriasis
- History of porphyria cutanea tarda
- Uncontrolled intercurrent illness
- Pregnant, breast feeding, or plan to become pregnant in the next year
- Inadequate contraception
- Significant hematological or hepatic abnormalities
- Use of an investigational drug within 30 days of study start
- Inability to attend scheduled clinic visits
- Inability to perform PFTs
- Creatinine > 2.5mg/dL
- Recent pneumothorax within 8 weeks of screening
- History of malignancy in the last 2 years other than basal cell skin cancer
- Use of estrogen containing medication within 30 days of screening
- Abnormal G6PD levels at baseline
- Preexisting maculopathy or retinopathy
- Preexisting myopathy
- Currently taking doxycycline, metformin, lupron, simvastatin
- Unable to undergo CT or MRI
- History of seizure within last year
- Hepatitis B, C, HIV positive serology
- Use of alternative medical therapies for LAM for at least 6 weeks prior to study
participation
- History of myocardial infarct, angina, or stroke related to atherosclerosis
- History of cardiomyopathy
- Previous lung transplant
- Surgery (involving entry into a body cavity or requiring 3 or more stitches) within 2
months of initiation of study drug
- Uncontrolled cholesterol > 350mg/dL, triglycerides > 400mg/dL
Age minimum:
18 Years
Age maximum:
85 Years
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Lymphangioleiomyomatosis
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Intervention(s)
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Drug: "Sirolimus" and "Hydroxychloroquine" 200 mg
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Drug: "Sirolimus" and "Hydroxychloroquine" 400 mg
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Primary Outcome(s)
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Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients
[Time Frame: 48 weeks]
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Secondary ID(s)
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1ZIAHL002541-21
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SAIL-1100
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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