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Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis

Primary Purpose

Lymphangioleiomyomatosis

Status

Recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

albuterol inhaler

albuterol nebulizer

PFT

About this trial

This is an interventional treatment trial for Lymphangioleiomyomatosis focused on measuring Albuterol, Bronchodilator, Nebulizer, Metered Dose Inhaler

Eligibility Criteria

18 Years - 100 Years (Adult, Older Adult)FemaleDoes not accept healthy volunteers

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).

Sites / Locations

National Institutes of Health Clinical CenterRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Arm Description

Nebullizer

Inhaler

Outcomes

Primary Outcome Measures

Greater improvement in lung function with nebulized albuterol.

Secondary Outcome Measures

Full Information

NCT ID

NCT01799538

First Posted

February 23, 2013

Last Updated

October 18, 2023

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

1. Study Identification

Unique Protocol Identification Number

NCT01799538

Brief Title

Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis

Official Title

Bronchodilator Effects of Nebulized Versus Inhaled Albuterol in Subjects With Lymphangioleiomyomatosis

Study Type

Interventional

2. Study Status

Record Verification Date

June 22, 2023

Overall Recruitment Status

Recruiting

Study Start Date

June 10, 2013 (Actual)

Primary Completion Date

November 1, 2026 (Anticipated)

Study Completion Date

November 1, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Lymphangioleiomyomatosis

Keywords

Albuterol, Bronchodilator, Nebulizer, Metered Dose Inhaler

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

Nebullizer

Arm Title

Arm Type

Active Comparator

Arm Description

Inhaler

Intervention Type

Drug

Intervention Name(s)

albuterol inhaler

Intervention Description

2 puffs of Metered Dose inhaler for Bronchodilation

Intervention Type

Drug

Intervention Name(s)

albuterol nebulizer

Intervention Description

Albuterol administered by nebulization will produce a greater increase in FEV1

Intervention Type

Procedure

Intervention Name(s)

PFT

Intervention Description

Breathing Test

Primary Outcome Measure Information:

Title

Greater improvement in lung function with nebulized albuterol.

Description

Greater improvement in lung function with nebulized albuterol.

Time Frame

3 days

10. Eligibility

Sex

Female

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

100 Years

Accepts Healthy Volunteers

Eligibility 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 (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).

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Tatyana Worthy, R.N.

Phone

(301) 827-1376

worthyt@mail.nih.gov

First Name & Middle Initial & Last Name or Official Title & Degree

Joel Moss, M.D.

Phone

(301) 496-1597

mossj@nhlbi.nih.gov

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Joel Moss, M.D.

Organizational Affiliation

National Heart, Lung, and Blood Institute (NHLBI)

Official's Role

Principal Investigator

Facility Information:

Facility Name

National Institutes of Health Clinical Center

City

Bethesda

State/Province

Maryland

ZIP/Postal Code

20892

Country

United States

Individual Site Status

Recruiting

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Citations:

PubMed Identifier

18252917

Citation

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

Results Reference

background

PubMed Identifier

16210669

Citation

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.

Results Reference

background

PubMed Identifier

10852420

Citation

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.

Results Reference

background

Links:

URL

https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2013-H-0051.html

Description

NIH Clinical Center Detailed Web Page

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Nebulized or Inhaled Albuterol for Lymphangioleiomyomatosis

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