Know Cancer

or
forgot password

An Exploratory, Open Label, Non-randomized, Within-patient Multiple Dose-escalation Safety, Tolerability, PK and Efficacy Trial of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis


Phase 2
18 Years
N/A
Not Enrolling
Female
Lymphangioleiomyomatosis

Thank you

Trial Information

An Exploratory, Open Label, Non-randomized, Within-patient Multiple Dose-escalation Safety, Tolerability, PK and Efficacy Trial of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis


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 < 90% of the predicted value of bronchodilator following administration of a
standard dose of a short acting β2-agonist (*200 µg Salbutamol, measured between 10
and 15 minutes of inhalation) and DLco (uncorrected) <80% predicted.

- Female patients including those of childbearing potential will be included in this
study.

- Negative pregnancy test at screening and baseline

Exclusion Criteria:

- FEV1<50% of predicted post-bronchodilator.

- Change in FVC (ml) > ± 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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Assess safety, pharmacokinetics (PK) and pharmacodynamics (PD), Measure: Safety (vital signs, ECGs, safety labs, AEs), PK (concentration of everolimus), PD (serum VEGF-D)

Outcome Time Frame:

26 weeks

Safety Issue:

Yes

Principal Investigator

Novartis

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CRAD001X2201

NCT ID:

NCT01059318

Start Date:

December 2009

Completion Date:

June 2012

Related Keywords:

  • Lymphangioleiomyomatosis
  • Lymphangioleiomyomatosis,
  • High Resolution CT scan,
  • chest x-ray,
  • 6 minute walk test,
  • pulse oximetry,
  • renal MRI,
  • Pikometer
  • Lymphangioleiomyomatosis

Name

Location

Center for LAM Research and Clinical Care Boston, Massachusetts  02115
University of Cincinnati, Department of Internal Medicine, Pulmonary, Critical Care & Sleep Medicine, Cincinnati, Ohio  45267