Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis

Last updated: April 23, 2025
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Overall Status: Active - Recruiting

Phase

1/2

Condition

Lymphangioleiomyomatosis

Treatment

albuterol inhaler

PFT

albuterol nebulizer

Clinical Study ID

NCT01799538
130051
13-H-0051
  • Ages 18-100
  • Female

Study Summary

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.

Eligibility Criteria

Inclusion

  • INCLUSION CRITERIA:

  • Diagnosis of LAM either by tissue biopsy, evidence of lung and other organinvolvement (renal angiomyolipomas, chylous effusions, lymphangioleiomyomas), highserum levels of vascular endothelial growth factor D (VGEF-D)(1) or a diagnosis ofTSC associated with cystic lung lesions.

  • Age 18 years or over

  • Evidence of airflow obstruction: FEV1/VC ratio < fifth percentile of predictednormal and an FEV(1) <80% predicted of the normal values.

Exclusion

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 (chest x-ray and or CT scan procedure completedunder Protocol 95-H-0186)

  • History of seizures other than during infancy

  • 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 orurine pregnancy test under Protocol 95-H-0186).

  • Treatment with monoaminoxidase inhibitors, tricyclic antidepressants orBeta-adrenergic receptor antagonists or long acting anticholinergic bronchodilatorswho 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).

Study Design

Total Participants: 100
Treatment Group(s): 3
Primary Treatment: albuterol inhaler
Phase: 1/2
Study Start date:
June 10, 2013
Estimated Completion Date:
November 01, 2027

Study 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 100 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.

Connect with a study center

  • National Institutes of Health Clinical Center

    Bethesda, Maryland 20892
    United States

    Active - Recruiting

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