ClinicalTrials.gov record NCT01799538

Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Bronchodilator Effects of Nebulized Versus Inhaled Albuterol in Subjects With Lymphangioleiomyomatosis

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
Age18 years old and older
SexFemale
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This randomized interventional study compares lung-function tests after albuterol given by nebulizer versus metered‑dose inhaler in adult women with LAM and airflow obstruction. Participants stay overnight for three consecutive days while receiving assigned treatment and having breathing tests before and after doses. People using long-acting inhalers will need to stop those drugs about one week before participation.

What it is studying. Whether nebulized albuterol produces a greater short-term improvement in lung function (FEV1) than albuterol given by a metered‑dose inhaler in women with LAM.

What the researchers hope to learn. To measure and compare bronchodilator response (change in FEV1) after albuterol delivered by nebulizer versus by metered‑dose inhaler over three days. The study also evaluates inhaler dosing differences (including higher puff counts) versus nebulization.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • albuterol inhaler
  • albuterol nebulizer
Procedure
  • PFT

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 (chest x-ray and or CT scan procedure completed under 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 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).

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

You would have a screening visit with a physical exam and medical history (women of childbearing potential will have a pregnancy test per protocol). Participants stay overnight for 3 consecutive days at the study site. Each day you would receive either nebulized albuterol or 2–4 puffs of an albuterol inhaler at about the same time, with breathing tests (pulmonary function tests) before and after each dose. The registry notes no routine lab tests are specified, but medication‑holding rules apply (see eligibility).

How the study is structured. Randomized, open‑label (no masking), parallel two‑arm trial. Participants are assigned to receive albuterol either by nebulizer or by metered‑dose inhaler (the protocol notes two or four puffs for the inhaler arm). Each participant spends three consecutive days in hospital for dosing and has lung function tests before and after each dose; the main comparison is change in FEV1 over the 3 days.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on lymphangioleiomyomatosis

Clinical trials10 trials listed for lymphangioleiomyomatosisEvery trial the registry lists for lymphangioleiomyomatosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in lymphangioleiomyomatosis researchLatest monthly update: September 2026. Includes: Pleural fluid drainage. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 10, 2013
Primary completion
Nov 1, 2027 (estimated)
Study completion
Nov 1, 2027 (estimated)
Target enrollment
100 participants (estimated)
Last updated on registry
Aug 31, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • National Institutes of Health Clinical CenterRecruiting

    Bethesda, Maryland, 20892 · United States

Terms used on this page · explanations by MedicalUpdates.ai

biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
CT scan
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
women of childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
INCLUSION CRITERIA
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
EXCLUSION CRITERIA
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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