Phase 2 N=24 Treatment

A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis

Lymphangioleiomyomatosis

Bottom Line

View on ClinicalTrials.gov: NCT01059318 ↗

Enrolled (actual)

Serious AEs

33.3%

Results posted

Nov 2020

Primary outcome: Primary: Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations — -464.3; -1113.2; -1771.7 pg/mL

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Everolimus (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: Female
Sponsor: Novartis Pharmaceuticals
Primary completion: Jun 2012

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	—

Summary

This was an exploratory study to determine whether escalating doses of RAD001 (everolimus) were safe and effective in patients with Lymphangioleiomyomatosis

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

View full record on ClinicalTrials.gov →

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.