Phase 2
Completed N=24
A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
Source: ClinicalTrials.gov NCT01059318 ↗Enrolled (actual)
24
Serious AEs
33.3%
Results posted
Nov 2020
Primary outcomePrimary: Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations — -464.3; -1113.2; -1771.7 pg/mL
Summary
This was an exploratory study to determine whether escalating doses of RAD001 (everolimus) were safe and effective in patients with Lymphangioleiomyomatosis
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations |
-464.3; -1113.2; -1771.7 | — |
| PRIMARY Mean Trough (C0,ss) and Peak (C2,ss) Drug Concentration at Steady State |
3.1; 5.8; 11.0; 12.1; 22.6; 45.7 | — |
| SECONDARY Change From Baseline in Forced Vital Capacity (FVC) |
10 | — |
| SECONDARY Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) |
114 | — |
| SECONDARY Change From Baseline in Extended Pulmonary Function Testing |
0.199; 0.267; 0.169 | — |
| SECONDARY Change From Baseline in Carbon Monoxide Diffusing Capacity (DLCO) |
-0.782 | — |
| SECONDARY Change From Baseline in 6-minute Walk Test Score to Measure Exercise Capacity |
46.9 | — |
| SECONDARY Change From Baseline in Oxygen Saturation |
0.8 | — |
Eligibility Criteria
Inclusion Criteria
- Female aged >/= 18 years with a diagnosis of LAM
- Pulmonary function abnormalities as follows:
- FEV1 of ≤ 80% of the predicted value following administration of a standard dose of a short acting β2-agonist (*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) OR
- FEV1 ± 15% of screening value at baseline visit (not less than 14d after screening visit).
- Use of any medicine containing estrogen in the 4 months prior to the screening visit and for the duration of the study
- Significant hematologic, renal, hepatic laboratory abnormality or amylase > 1.5x the upper limit of the normal range at the screening or baseline visits
- Fasting blood glucose > 126mg/dl or random blood glucose >200mg/dl at screening and/or baseline
- Recent surgery (involving entry into a body cavity or requiring sutures) within 2 months of the screening visit or any evidence of unhealed surgical wound.
- Uncontrolled hyperlipidemia (defined as persistent elevation of total cholesterol or triglycerides >6.5nM/L) or a history of clinical atherosclerotic disease including heart attack, angina, peripheral vascular disease or stroke.
- Previous organ transplantation
- Inability to give informed consent
- Inability to perform pulmonary function or 6 minute walk tests and imaging assessments
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01059318). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.