Trial · NCT01687179

Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis

NCT01687179, Phase 1, Completed, Brigham and Women's Hospital; results first posted 2018-10-11

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis. Trials, record NCT01687179. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01687179

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis
NCT number: NCT01687179
Status: Completed
Phases: Phase 1
Enrollment: 14 participants, actual
Lead sponsor: Brigham and Women's Hospital
Conditions: Lymphangioleiomyomatosis
Interventions: "Sirolimus" and "Hydroxychloroquine" 200 mg, "Sirolimus" and "Hydroxychloroquine" 400 mg
First posted: 2012-09-18
Last updated: 2018-10-11
Results first posted: 2018-10-11

Source ClinicalTrials.gov

Registered outcomes1

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	48 weeks	The Primary endpoint of this study was safety. Safety was assessed based on the adverse events and serious adverse events that occurred in these patients when they were on this combination therapy. Percentage of adverse events in each system at a dose was calculated from the total adverse events at that dose. Subjects were closely monitored and adverse events were classified and graded according to the "Common Terminology Criteria for Adverse Events, (CTCAE) Version 4.0".

Source ClinicalTrials.gov

Outcomes1

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	48 weeks		Percentage of adverse events	NUMBER

Source ClinicalTrials.gov

Results by group34

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 200 mg	Cardiac Disorders	2.27	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 400 mg	Cardiac Disorders	1.11	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 200 mg	Eye Disorders	0	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 400 mg	Eye Disorders	1.11	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 200 mg	GI Disorders	18.18	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 400 mg	GI Disorders	22.22	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 200 mg	General Disorders, Administration site condistions	18.18	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 400 mg	General Disorders, Administration site condistions	6.67	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 200 mg	Immune System disorders	0	Percentage of adverse events
Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients	Sirolimus and Hydroxychloroquine 400 mg	Immune System disorders	0.56	Percentage of adverse events

1 to 10 of 34Source ClinicalTrials.gov

See all 34 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Sirolimus and Hydroxychloroquine 200 mg	3
Overall Study	STARTED	Sirolimus and HydroxyChloroquine 400 mg	10
Overall Study	COMPLETED	Sirolimus and Hydroxychloroquine 200 mg	2
Overall Study	COMPLETED	Sirolimus and HydroxyChloroquine 400 mg	6
Overall Study	NOT COMPLETED	Sirolimus and Hydroxychloroquine 200 mg	1
Overall Study	NOT COMPLETED	Sirolimus and HydroxyChloroquine 400 mg	4

Source ClinicalTrials.gov

Adverse events10

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Rash/ Acne	Skin and subcutaneous tissue disorders	Sirolimus and Hydroxychloroquine 200 mg	3	3
other	Rash/ Acne	Skin and subcutaneous tissue disorders	Sirolimus and Hydroxychloroquine 400 mg	5	10
other	Abnormal Investigations	Investigations	Sirolimus and Hydroxychloroquine 200 mg	1	3
other	Abnormal Investigations	Investigations	Sirolimus and Hydroxychloroquine 400 mg	6	10
other	Diarrhea	Gastrointestinal disorders	Sirolimus and Hydroxychloroquine 200 mg	2	3
other	Diarrhea	Gastrointestinal disorders	Sirolimus and Hydroxychloroquine 400 mg	6	10
other	Oral Mucositis	Gastrointestinal disorders	Sirolimus and Hydroxychloroquine 200 mg	2	3
other	Oral Mucositis	Gastrointestinal disorders	Sirolimus and Hydroxychloroquine 400 mg	6	10
serious	radiculitis	Nervous system disorders	Sirolimus and Hydroxychloroquine 200 mg	0	3	0
serious	radiculitis	Nervous system disorders	Sirolimus and Hydroxychloroquine 400 mg	1	10	1

Source ClinicalTrials.gov

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sirolimus and Hydroxychloroquine 200 mg	0	3
Total, all-cause mortality	Sirolimus and Hydroxychloroquine 400 mg	0	10
Total, other adverse events	Sirolimus and Hydroxychloroquine 200 mg	3	3
Total, other adverse events	Sirolimus and Hydroxychloroquine 400 mg	9	10
Total, serious adverse events	Sirolimus and Hydroxychloroquine 200 mg	0	3
Total, serious adverse events	Sirolimus and Hydroxychloroquine 400 mg	1	10

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
28192114	DERIVED	PubMed
30144422	DERIVED	PubMed
31299246	DERIVED	PubMed

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
ZIAHL002541	Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM)	MOSS, JOEL	NATIONAL HEART, LUNG, AND BLOOD INSTITUTE	NATIONAL HEART, LUNG, AND BLOOD INSTITUTE	2009 to 2025

Source NIH RePORTER

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Sirolimus and Hydroxychloroquine 200 mg vs Sirolimus and Hydroxychloroquine 400 mg: deaths from any cause	Trial comparisons
Sirolimus and Hydroxychloroquine 200 mg vs Sirolimus and Hydroxychloroquine 400 mg: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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