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The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)

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ClinicalTrials.gov Identifier: NCT02116712
Recruitment Status : Completed
First Posted : April 17, 2014
Last Update Posted : November 30, 2016
Sponsor:
Collaborators:
University of Texas
University of Cincinnati
Information provided by (Responsible Party):
Tony Eissa, Baylor College of Medicine

Brief Summary:

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

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Saracatinib

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.

Drug: Saracatinib
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Other Outcome Measures:
  1. Efficacy exploration [ Time Frame: 8 weeks ]
    explore the efficacy measurements (e.g., pulmonary function test, six minute walk test, and VEGF-D)



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female patients. It should be noted, however, that LAM occurs predominantly in women.
  • 18 to 65 years of age.
  • All patients must have a diagnosis of LAM as defined by one of the following:

Open lung, transbronchial or thoracic needle biopsy consistent with LAM Open or needle abdominal biopsy findings consistent with LAM Computed tomography (CT) of chest or abdomen consistent with LAM in the setting of TSC, renal angiomyolipoma (AML), cystic abdominal lymphangiomas, or history of chylous effusion in the chest or abdomen CT of chest consistent with LAM plus serum vascular endothelial growth factor (VEGF-D) > 800 pg/ml In cases where the diagnosis of LAM is based on biopsy, review of the pathology specimens by pathologists who are experienced with LAM, such as those at the NIH or the Mayo Clinic, will be obtained (if not done so previously).

Exclusion Criteria:

  • Current infection.
  • Major surgery within the past 2 months
  • Advanced hematologic, renal, hepatic, or metabolic diseases
  • The use of another investigational drug within 30 days
  • The use of mammalian target of rapamycin (mTOR) inhibitors within 30 days
  • Previous lung transplantation or active on transplant list.
  • Inability to attend scheduled clinic visits
  • Inability to give informed consent
  • Inability to perform pulmonary function testing
  • History of malignancy in the past two years, other than squamous or basal cell skin cancer or mild cervical cancer.
  • Nursing mothers
  • Current or planned pregnancy.
  • Not using adequate contraception (in woman of childbearing potential).
  • Significant clinical change in health in the past 30 days

Information from the National Library of Medicine

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


Locations
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
Sponsors and Collaborators
Tony Eissa
University of Texas
University of Cincinnati
Investigators
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

Responsible Party: Tony Eissa, Professor of Medicine - Pulmonary, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02116712     History of Changes
Other Study ID Numbers: SLAM 7601
First Posted: April 17, 2014    Key Record Dates
Last Update Posted: November 30, 2016
Last Verified: November 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: The subject is aware of his/her data at the time of the study. They are told if they are/are not using the correct technique for MDI. There is no more data to share with the subject.

Keywords provided by Tony Eissa, Baylor College of Medicine:
LAM
saracatinib
Lymphangioleiomyomatosis

Additional relevant MeSH terms:
Lymphangioleiomyomatosis
Lymphangiomyoma
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Saracatinib
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action