Open
Actively Recruiting
Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial
About
Brief Summary
This is a study to determine if early, long-term low dose sirolimus is effective for preventing progression to more advanced stages.
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Inclusion Criteria:
- Female, age 18 or over
- Signed and dated informed consent
- Diagnosis of LAM as determined by compatible lung CT and one of the following
- biopsy (lung, abdominal mass, lymph node or kidney) or cytology from thoracic or abdominal sources revealing LAM, or
- tuberous sclerosis, angiomyolipomata (diagnosed by CT, MRI by the site radiologist or biopsy) or chylous pleural effusion (verified by tap), or
- VEGF-D level ≥ 800 pg/ml.
- Post-bronchodilator forced expiratory volume in one second of > 70%
- Presence of markers of non-trivial burden of LAM or likely progression based on one of
the following:
- pretrial FEV 1 rate of decline of >60cc/yr, comparing enrollment FEV1 to any prior measurement in the past 3 years, or
- baseline supplemental oxygen requirement with exercise, or
- pre-menopausal and one of the following (if post-menopausal, must have a VEGF-D level ≥ 600 pg/ml and one of the following) baseline diffusing capacity for carbon monoxide ≤80% predicted, a) baseline residual volume ≥120% predicted, b) baseline desaturation by 4% or more on six minute walk testing on room air c) more than 20 cysts on the carinal cut of the CT
Exclusion Criteria:
- Existing or imminent (within 12-18 months) clinical indication for treatment with mTOR inhibitors, based on judgment of site investigator
- DLCO <60% predicted
- Resting room air saturation <90%
- Exercise induced desaturation nadir on room air < 85%
- History of myocardial infarction, angina or stroke related to atherosclerosis
- Pregnant, breast feeding, or plan to become pregnant in the next 2.5 years
- Inadequate contraception
- Significant hematologic, renal, metabolic or hepatic abnormality (i.e. transaminase
levels > three times the UL of normal range, HCT < 30%, platelets < 80,000/mm3,
adjusted absolute neutrophil count < 1,000/ mm3, total WBC < 3,000/ mm3), creatinine
2.5 mg/dl, uncontrolled hyperlipidemia
- Acute or chronic infection, such as (nontuberculous mucobacteria or active hepatitis B or C infections)
- Recent surgery (involving entry into a body cavity or requiring 3 or more sutures) within three weeks of initiation of study drug
- Use of sirolimus, everolimus or investigational treatment for LAM within the 30 days prior to randomization
- Previous lung transplantation or active on transplant list
- Inability to attend scheduled clinic visits, or perform pulmonary function testing
- Pleural effusion or chylous ascites sufficient to affect pulmonary function based on the opinion of the Site Investigator
- Acute pneumothorax within the past month
- History of malignancy in the past two years, other than squamous or basal cell skin cancer.
- Use of estrogen containing medications within the 30 days prior to randomization.
- Known allergy to sirolimus
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Study Stats
Protocol No.
20-000217
Category
Pulmonary Disease
Principal Investigator
Ariss Derhovanessian
Contact
Location
- UCLA Westwood