Active — Not RecruitingPhase 3

Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial

United States60 participantsStarted 2018-01-01

Plain-language summary

This is a study to determine if early, long-term low dose sirolimus is effective for preventing progression to more advanced stages.

Age range18 Years

SexFEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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:

✕. 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

Forced Expiratory Volume in 1 Second (FEV1 slope)

Timeframe: 2 years

NCT IDNCT03150914

SponsorUniversity of Cincinnati

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-06-30

Who can participate

Age range18 Years

SexFEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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:

✕. 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