The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)
Lymphangioleiomyomatosis (LAM) is a rare lung disease that mostly affects women of childbearing age. In LAM, abnormal, muscle-like cells begin to grow out of control in the lungs. As a result, air can't move freely in and out of the lungs. In some cases, this means the lungs can't supply the body's other organs with enough oxygen.
This study is being conducted to find out what dose of a drug called saracatinib is best tolerated by people with LAM. This drug has been tested in patients with certain types of cancer but is not currently approved by the United States Food and Drug Administration (FDA). Saracatinib may work in cancer by preventing the growth, movement and invasiveness of cancer cells. The use of saracatinib to treat LAM is considered experimental. Preliminary testing already completed suggests that the study drug, saracatinib, may suppress certain substances in the lungs of patients with LAM thus may be effective in slowing down the disease process
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)|
- Dose Determination [ Time Frame: Saracatinib will be given for 4 weeks, the subject will be followed for a total of 8 weeks. ] [ Designated as safety issue: Yes ]One of three escalating daily oral doses of saracatinib; 50, 125 and 175 mg. Saracatinib will be given orally once a day for four weeks. Adverse events will be monitored. The subject will receive only one of the doses as determined by the escalation of the study.
- Safety Profile [ Time Frame: 8 weeks ] [ Designated as safety issue: Yes ]This objective is to generate a safety profile utilizing the data collected along with the adverse events. Subjects will be followed closely during the 4 weeks while taking drug and again for four weeks after stopping the study drug.
- Efficacy exploration [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]explore the efficacy measurements (e.g., pulmonary function test, six minute walk test, and VEGF-D)
|Study Start Date:||March 2014|
|Estimated Study Completion Date:||April 2015|
|Estimated Primary Completion Date:||January 2015 (Final data collection date for primary outcome measure)|
Only one arm: Intervention is Saracatinib. We plan to study three escalating doses of oral saracatinib; 50, 125 and 175 mg. Saracatinib is given orally once a day.
More regarding dose escalation is included in intervention below.
Saracatinib is escalated as follows: Three dose levels will be administered for 1 month each: 50 mg, 125 mg and 175 mg. Three subjects will be treated at the lowest daily dose of 50 mg. If no subject experiences DLT (dose limiting toxicity), the dose level is escalated to 125 mg/day for the next cohort of 3 different subjects and so on to next dose.
Other Name: AZD0530
Show Detailed Description
Please refer to this study by its ClinicalTrials.gov identifier: NCT02116712
|Contact: Caryn O Pope, RRTfirstname.lastname@example.org|
|Contact: Tony Eissa, MDemail@example.com|
|United States, Ohio|
|University of Cincinnati||Not yet recruiting|
|Cincinnati, Ohio, United States, 45267|
|Contact: Tammy Roads 513-558-2148 firstname.lastname@example.org|
|Principal Investigator: Frank X McCormack, MD|
|United States, Texas|
|Baylor College of Medicine||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Caryn O Pope, RRT 713-873-2471 email@example.com|
|Contact: Nicola A Hanania, MD 713-873-2468 firstname.lastname@example.org|
|Principal Investigator: Nicola A Hanania, MD|
|University of Texas Health Science Center-Houston||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Stacy Burk, LVN 713-500-6851 Stacy.Burk@uth.tmc.edu|
|Contact: , MD|
|Principal Investigator: Khalid F Almoosa, MD|
|Study Chair:||Tony Eissa, MD||Baylor College of Medicine|
|Principal Investigator:||Nicola A Hanania, MD||Baylor College of Medicine - Ben Taub Hospital|
|Principal Investigator:||Khalid Almoosa, MD||The University of Texas Health Science Center, Houston|
|Principal Investigator:||Frank McCormack, MD||University of Cincinnati|