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
Treatment
Study Type
Interventional
Phase
Phase 3

Eligibility

Gender
Female
Healthy Volunteers
No
Minimum Age
18 Years
Maximum Age
N/A

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

Join this Trial

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Study Stats
Protocol No.
20-000217
Category
Pulmonary Disease
Contact
Chen Zheng
Location
  • UCLA Westwood
For Providers
NCT No.
NCT03150914
For detailed technical eligibility, visit ClinicalTrials.gov.