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Brief Title: Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis
Official Title: Targeting Autophagy for the Treatment of TSC and LAM: a Phase I Trial of Hydroxychloroquine and Sirolimus
Study ID: NCT01687179
Brief Summary: Specific Aim 1: To investigate whether, in Lymphangioleiomyomatosis (LAM) patients, the combination of sirolimus and hydroxychloroquine is safe and well tolerated Specific Aim 2: To investigate whether, in LAM patients, 6 months of combination therapy with sirolimus and hydroxychloroquine results in improvement of indicators of disease, and whether the gains are sustained after stopping therapy. Specific Aim 3: To investigate the potential role of a LAM-specific peripheral blood signature to predict rates of disease progression and determine responsiveness to combination therapy. This will be a phase I dose escalation study of the combination of sirolimus (2 mg adjusted to keep trough levels between 5-15 ng/ml) and hydroxychloroquine (200 mg or 400 mg) taken orally daily. Up to 18 adult women with LAM will be enrolled.
Detailed Description: This will be a phase I dose escalation study of the combination of sirolimus (2 mg adjusted to keep trough levels between 5-15 ng/ml) and hydroxychloroquine (200 mg or 400 mg) taken orally daily for 6 months. The study is to be conducted at 2 sites. Up to 18 adult women with LAM will be enrolled, and each recruiting site will recruit between 8-12 subjects. The protocol will use the following eligibility criteria.
Minimum Age: 18 Years
Eligible Ages: ADULT, OLDER_ADULT
Sex: FEMALE
Healthy Volunteers: No
Brigham and Women's Hospital, Boston, Massachusetts, United States
Name: Elizabeth P Henske, MD
Affiliation: Brigham and Women's Hospital
Role: PRINCIPAL_INVESTIGATOR
Name: Joel Moss, MD, PhD
Affiliation: National Heart, Lung, and Blood Institute (NHLBI)
Role: PRINCIPAL_INVESTIGATOR