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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2016
• Main ID: NCT02116712
• Date of registration: 15/04/2014
• Prospective Registration: Yes
• Primary sponsor: Tony Eissa
• Public title: The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)
• Scientific title: The Tolerability of Saracatinib in Subjects With Lymphangioleiomyomatosis (LAM) (SLAM-1)
• Date of first enrolment: August 2014
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02116712
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Khalid Almoosa, MD
• Affiliation: The University of Texas Health Science Center, Houston

• Name: Frank McCormack, MD
• Affiliation: University of Cincinnati

• Name: Tony Eissa, MD
• Affiliation: Baylor College of Medicine

• Name: Nicola A Hanania, MD
• Affiliation: Baylor College of Medicine - Ben Taub Hospital

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Pulmonary Lymphangioleiomyomatosis

Intervention(s)

Drug: Saracatinib

Primary Outcome(s)

Dose Determination [Time Frame: Saracatinib will be given for 4 weeks, the subject will be followed for a total of 8 weeks.]

Secondary Outcome(s)

Safety Profile [Time Frame: 8 weeks]

Secondary ID(s)

SLAM 7601

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

University of Texas

University of Cincinnati