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Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis

NCT01687179 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2018-10-11

Study results available
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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.

Conditions

  • Lymphangioleiomyomatosis

Interventions

DRUG

"Sirolimus" and "Hydroxychloroquine" 200 mg

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 taken orally daily.

DRUG

"Sirolimus" and "Hydroxychloroquine" 400 mg

Once safety is established with the lower dose, (Sirolimus and Hydroxychloroquine 200 mg), subjects will receive Sirolimus 2 mg (adjusted to keep trough levels between 5 to 15 ng/ml) and hydroxychloroquine 200 mg twice a day.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Brigham and Women's Hospital

    lead OTHER

Principal Investigators

  • Elizabeth P Henske, MD · Brigham and Women's Hospital

  • Joel Moss, MD, PhD · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
85 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-09-30
Primary Completion
2015-08-31
Completion
2015-08-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01687179 on ClinicalTrials.gov