Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2013 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:
NCT01799538
First received: February 23, 2013
Last updated: March 14, 2014
Last verified: November 2013
  Purpose

Background:

- Lymphangioleiomyomatosis (LAM) is a rare type of lung disease that occurs almost exclusively in women. In LAM, muscle tissue grows in the lungs and starts to block the flow of air. It is a progressive disease, and in severe cases may require a lung transplant. One possible treatment to improve breathing in people with LAM is inhaled albuterol. Albuterol can be given in a metered dose inhaler (MDI) or with a nebulizer. Researchers want to compare these methods to see which method best improves lung function in women with LAM.

Objectives:

- To see whether a nebulizer or MDI can better improve lung function in women with LAM.

Eligibility:

- Women at least 18 years of age who have impaired lung function because of LAM.

Design:

  • Participants will be screened with a physical exam and medical history. No lab tests will be needed for this study.
  • Participants will have a 3-day overnight stay at the National Institutes of Health. Those who are using long-acting inhalers will have to stop taking these drugs 1 week before the study.
  • Participants will receive either the nebulizer or two or four puffs of the inhaler. Four puffs of albuterol is a higher dose than is normally prescribed, and is being tested on this study.
  • Participants will have each treatment around the same time of day on each of the 3 days. Before and after taking the albuterol, participants will have lung function tests.

Condition Intervention Phase
Lymphangioleiomyomatosis
Drug: albuterol inhaler
Procedure: PFT
Drug: albuterol nebulizer
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Bronchodilator Effects of Nebulized Versus Inhaled Albuterol In Subjects With Lymphangioleiomyomatosis

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • Greater improvement in lung function with nebulized albuterol. [ Time Frame: 3 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 150
Study Start Date: December 2012
Estimated Study Completion Date: November 2015
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: albuterol inhaler
    N/A
    Procedure: PFT
    N/A
    Drug: albuterol nebulizer
    N/A
Detailed Description:

We have reported that approximately one third of patients with lymphangioleiomyomatosis (LAM) who have airflow obstruction respond to bronchodilators such as albuterol, a Beta2-adenergic receptor agonist, with an increase in forced expiratory flow in one second (FEV1) of 12% and 200 ml above baseline values. Others however, have questioned these findings, reporting instead, a low rate of response of only six percent. Contrasting with our study, in this study albuterol was administered with a metered dose inhaler whereas in ours it was given by nebulizer. We propose to measure changes in lung function after administration of albuterol, respectively by metered inhaler and nebulizer, for 3 consecutive days in 150 LAM subjects. Our hypothesis is that albuterol administered by nebulization will produce a greater increase in FEV1 than two puffs of inhaled albuterol. If this hypothesis is confirmed, then we may recommend that patients with LAM and airflow obstruction use as a method of drug administration a nebulizer, rather than a metered dose inhaler.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:
  • Diagnosis of LAM either by tissue biopsy, evidence of lung and other organ involvement (renal angiomyolipomas, chylous effusions, lymphangioleiomyomas), high serum levels of vascular endothelial growth factor D (VGEF-D)(1) or a diagnosis of TSC associated with cystic lung lesions.
  • Age 18 years or over
  • Evidence of airflow obstruction: FEV1/VC ratio < fifth percentile of predicted normal and an FEV(1) < 80% predicted of the normal values.

EXCLUSION CRITERIA:

Subjects will be excluded from the study if they meet one or more of the following criteria:

  • History of hypersensitivity to albuterol or any of its components.
  • Moderate or large pleural effusions
  • History of seizures
  • Inability to withhold bronchodilators for 24 hours
  • Cognitive Impairment
  • Age less than 18 years
  • Male sex
  • Status-post lung or kidney transplantation
  • Pregnant or breast feeding (women of childbearing potential will undergo a blood or urine pregnancy test under Protocol 95-H-0186).
  • Treatment with monoaminoxidase inhibitors, tricyclic antidepressants or Beta-adrenergic receptor antagonists or long acting anticholinergic bronchodilators who are unable to be discontinued for at least seven days before enrollment.
  • Patients with URI, uncontrolled hyperthyroidism or severe gastro-esophageal reflux. Major systemic diseases (i.e., malignancy; myocardial infarction or unstable angina; type 1 diabetes, severe hypertension; liver cirrhosis).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01799538

Contacts
Contact: Mary Haughey, R.N. (301) 496-3632 mhaughey@nhlbi.nih.gov
Contact: Angelo M Taveira-DaSilva, M.D. (301) 451-4950 dasilvaa@nhlbi.nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010    prpl@mail.cc.nih.gov   
Sponsors and Collaborators
Investigators
Principal Investigator: Angelo M Taveira-DaSilva, M.D. National Heart, Lung, and Blood Institute (NHLBI)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01799538     History of Changes
Other Study ID Numbers: 130051, 13-H-0051
Study First Received: February 23, 2013
Last Updated: March 14, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Albuterol
Bronchodilator
Nebulizer
Metered Dose Inhaler

Additional relevant MeSH terms:
Lymphangioleiomyomatosis
Immune System Diseases
Immunoproliferative Disorders
Lymphangiomyoma
Lymphatic Diseases
Lymphatic Vessel Tumors
Lymphoproliferative Disorders
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Connective and Soft Tissue
Perivascular Epithelioid Cell Neoplasms
Albuterol
Bronchodilator Agents
Adrenergic Agents
Adrenergic Agonists
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Anti-Asthmatic Agents
Autonomic Agents
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Peripheral Nervous System Agents
Pharmacologic Actions
Physiological Effects of Drugs
Reproductive Control Agents
Respiratory System Agents
Therapeutic Uses
Tocolytic Agents

ClinicalTrials.gov processed this record on October 23, 2014