NCT01799538

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P75+ for phase_1

Timeline
18mo left

Started Jun 2013

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress90%
Jun 2013Nov 2027

First Submitted

Initial submission to the registry

February 23, 2013

Completed
3 days until next milestone

First Posted

Study publicly available on registry

February 26, 2013

Completed
3 months until next milestone

Study Start

First participant enrolled

June 10, 2013

Completed
14.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2027

Last Updated

May 1, 2026

Status Verified

March 18, 2026

Enrollment Period

14.4 years

First QC Date

February 23, 2013

Last Update Submit

April 30, 2026

Conditions

Lymphangioleiomyomatosis

Keywords

AlbuterolBronchodilatorNebulizerMetered Dose Inhaler

Outcome Measures

Primary Outcomes (1)

  • Greater improvement in lung function with nebulized albuterol.

    Greater improvement in lung function with nebulized albuterol.

    3 days

Study Arms (2)

1

EXPERIMENTAL

Nebullizer

Drug: albuterol nebulizerProcedure: PFT

2

ACTIVE COMPARATOR

Inhaler

Drug: albuterol inhalerProcedure: PFT

Interventions

albuterol inhalerDRUG

2 puffs of Metered Dose inhaler for Bronchodilation

2
albuterol nebulizerDRUG

Albuterol administered by nebulization will produce a greater increase in FEV1

1
PFTPROCEDURE

Breathing Test

12

Eligibility Criteria

Age18 Years - 100 Years
Sexfemale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Publications (3)

  • McCormack FX. Lymphangioleiomyomatosis: a clinical update. Chest. 2008 Feb;133(2):507-16. doi: 10.1378/chest.07-0898.

    PMID: 18252917BACKGROUND

  • Ryu JH, Moss J, Beck GJ, Lee JC, Brown KK, Chapman JT, Finlay GA, Olson EJ, Ruoss SJ, Maurer JR, Raffin TA, Peavy HH, McCarthy K, Taveira-Dasilva A, McCormack FX, Avila NA, Decastro RM, Jacobs SS, Stylianou M, Fanburg BL; NHLBI LAM Registry Group. The NHLBI lymphangioleiomyomatosis registry: characteristics of 230 patients at enrollment. Am J Respir Crit Care Med. 2006 Jan 1;173(1):105-11. doi: 10.1164/rccm.200409-1298OC. Epub 2005 Oct 6.

    PMID: 16210669BACKGROUND

  • Costello LC, Hartman TE, Ryu JH. High frequency of pulmonary lymphangioleiomyomatosis in women with tuberous sclerosis complex. Mayo Clin Proc. 2000 Jun;75(6):591-4. doi: 10.4065/75.6.591.

    PMID: 10852420BACKGROUND

Related Links

MeSH Terms

Conditions

Lymphangioleiomyomatosis

Condition Hierarchy (Ancestors)

LymphangiomyomaNeoplasm, Lymphatic TissueNeoplasms by Histologic TypeNeoplasmsPerivascular Epithelioid Cell NeoplasmsNeoplasms, Connective and Soft TissueLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Study Officials

  • Joel Moss, M.D.

    National Heart, Lung, and Blood Institute (NHLBI)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Tatyana A Worthy, R.N.

CONTACT

(301) 827-1376worthyt@mail.nih.gov

Joel Moss, M.D.

CONTACT

(301) 496-1597mossj@nhlbi.nih.gov

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 23, 2013

First Posted

February 26, 2013

Study Start

June 10, 2013

Primary Completion (Estimated)

November 1, 2027

Study Completion (Estimated)

November 1, 2027

Last Updated

May 1, 2026

Record last verified: 2026-03-18

Locations

US(1)