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NCT01687179 · ClinicalTrials.gov registry record · Phase 1
Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis
A Phase 1 study, sponsored by Brigham and Women's Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 14
- Enrollment target
NCT01687179: Completed Phase 1 study, sponsored by Brigham and Women's Hospital.
NCT01687179 is a Phase 1 study that has completed, run by Brigham and Women's Hospital. The registered enrollment target is 14 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (77% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01687179, a Phase 1 study, has completed, sponsored by Brigham and Women's Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 14 participants
- Enrollment target
Study 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.
Primary Outcome
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".
Interventions
- DRUG "Sirolimus" and "Hydroxychloroquine" 200 mg
- DRUG "Sirolimus" and "Hydroxychloroquine" 400 mg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2012-09 |
| Est. Completion | 2015-08 |
| Phase | Phase 1 |
What the finished NCT01687179 record still lists
NCT01687179 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 14 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (77% lower).
The record links to 0 conditions, and to 2 interventions - of which "Sirolimus" and "Hydroxychloroquine" 200 mg is the first listed.
NCT01687179 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01687179 about?
NCT01687179 is a clinical study titled "Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis". 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 i...
What is the current status of trial NCT01687179?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 14 participants. The study started on 2012-09. Estimated completion is 2015-08.
What interventions are being tested in trial NCT01687179?
The interventions under investigation include: "Sirolimus" and "Hydroxychloroquine" 200 mg (DRUG), "Sirolimus" and "Hydroxychloroquine" 400 mg (DRUG).
Who is sponsoring clinical trial NCT01687179?
This trial is sponsored by Brigham and Women's Hospital, which has 937 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01687179's enrollment target sits among peer trials
14 1844th of 2000 higher than 154 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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