The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02116712|
Recruitment Status : Completed
First Posted : April 17, 2014
Last Update Posted : November 30, 2016
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
|Condition or disease||Intervention/treatment||Phase|
|Pulmonary Lymphangioleiomyomatosis||Drug: Saracatinib||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)|
|Study Start Date :||August 2014|
|Actual Primary Completion Date :||June 2015|
|Actual Study Completion Date :||July 2015|
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
- Dose Determination [ Time Frame: Saracatinib will be given for 4 weeks, the subject will be followed for a total of 8 weeks. ]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 ]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 ]explore the efficacy measurements (e.g., pulmonary function test, six minute walk test, and VEGF-D)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02116712
|United States, Ohio|
|University of Cincinnati|
|Cincinnati, Ohio, United States, 45267|
|United States, Texas|
|Baylor College of Medicine|
|Houston, Texas, United States, 77030|
|University of Texas Health Science Center-Houston|
|Houston, Texas, United States, 77030|
|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|