Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis

NCT02737202 | Terminated Phase 2 DMC

• 2 other identifiers: SLAM 76024UH3TR000961-02
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 5

Brief Summary

This study is being done to determine if there is a potential benefit of saracatinib in LAM subjects. Based on the information of this trial, additional clinical development trials will be needed. The study will also test the tolerability of 125 mg of saracatinib given once daily over a 9 month period.

Conditions

Pulmonary Lymphangioleiomyomatosis

Keywords

LAM

Outcome Measures

Primary Outcomes (1)

• FEV1

  9 months

Secondary Outcomes (3)

• Angiomyolipoma measured volumetrically on MRI

  12 months

• Lung Cyst size measured on chest CT

  9 months

• VEGF-D serum levels

  12 months

Other Outcomes (1)

• Pulmonary Function Testing

  12 months

Study Arms (1)

Saracatinib

EXPERIMENTAL

Saracatinib will be given orally at a dose of 125 milligrams once daily for 9 months. Saracatinib is provided as a pink tablet.

Drug: saracatinib

Interventions

saracatinib DRUG

Subjects will receive enough tablets for 90 days +/- 14 days at each visit. Subject will have visits every 90 days for drug accountability as well as safety and efficacy testing to include pulmonary function testing, laboratory testing to include liver and kidney profile, urine pregnancy testing at each visit, vital signs, physical examination - any medically significant changes from baseline visit will be recorded, all adverse events will be monitored until resolution.

Also known as: AZD0530

Saracatinib

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Female patients. It should be noted that LAM occurs almost exclusively in women.
• to 65 years of age.
• All patients must have a diagnosis of LAM as defined by compatible cystic changes on chest computed tomography (CT) and one of the following:
• Open lung, transbronchial or thoracic needle biopsy consistent with LAM
• Open or needle abdominal biopsy findings consistent with LAM
• Clinical findings of tuberous scleroma complex (TSC), renal angiomyolipoma, cystic abdominal lymphangiomas, or history of chylous effusion in the chest or abdomen
• Serum vascular endothelial growth factor D (VEGF-D) \> 800 pg/ml
• Subjects must have had a recent reduction in forced expiratory volume at 1-second (FEV1) of \> 50ml/year, as shown by at least two pulmonary function testing (PFT) measured at least 6 months apart in the last 24 months prior to enrolling study.

You may not qualify if:

• Current infection.
• Major surgery within the past 2 months
• Advanced hematologic, renal, hepatic, non-LAM lung disease or metabolic diseases; or BMI of \>35
• The use of another investigational drug within 30 days
• The use of mTOR (mammalian target of rapamycin) inhibitors within 30 days
• Previous lung transplantation.
• 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 status post successful excision or treatment.
• 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

Sponsors & Collaborators

• Baylor College of Medicine: lead
• University of Cincinnati: collaborator
• Brigham and Women's Hospital: collaborator
• Stanford University: collaborator
• Loyola University: collaborator
• University of South Florida: collaborator
• National Institutes of Health (NIH): collaborator

Study Sites (5)

Stanford University

Stanford, California, 94305, United States

Location

Loyola Medical Center

Chicago, Illinois, 90153, United States

Location

Laboratory of Translational Research NHLBI

Bethesda, Maryland, 20814, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Baylor College of Medicine - Ben Taub General Hospital

Houston, Texas, 77030, United States

Location

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MeSH Terms

Interventions

saracatinib

Study Officials

• Tony Eissa, MD

  Baylor College of Medicine

  STUDY CHAIR

• Nicola A Hanania, MD

  Ben Taub Hospital

  PRINCIPAL INVESTIGATOR

• Francis X McCormack, MD

  University of Cincinnati

  PRINCIPAL INVESTIGATOR

• Daniel Dilling, MD

  Loyola University

  PRINCIPAL INVESTIGATOR

• Stephen Ruoss, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

• Joel Moss, MD

  Laboratory of Translational Research - NHLBI

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 31, 2016
• First Posted: April 13, 2016
• Study Start: April 1, 2016
• Primary Completion: July 30, 2019
• Study Completion: July 30, 2019
• Last Updated: July 30, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)