Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis
Status:
Completed
Trial end date:
2015-08-01
Target enrollment:
Participant gender:
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.