Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

NCT01885936 | Completed Phase 1 Results

• 1 other identifier: Pro00046020
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated in patients with Late Onset Pompe Disease.

Conditions

Pompe Disease

Keywords

LOPD; Pompe Disease

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events.

  All participants who experienced adverse events.

  52 weeks

Secondary Outcomes (2)

• Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.

  Baseline, Week 30, and Week 52

• Change in 6 Minute Walk Test

  Baseline, Week 6, and Week 52

Study Arms (2)

Albuterol

EXPERIMENTAL

Initially 4 mg daily for one week, then 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study.

Drug: Albuterol

Placebo Comparator

PLACEBO COMPARATOR

Initially one capsule daily for one week, then one capsule BID per oral daily for the next 5 weeks. If the one capsule BID per oral is well tolerated, the dose will be increased to two capsules each morning/one capsule each evening for one week, followed by two capsules BID per oral for the remainder of the study.

Drug: Placebo

Interventions

Albuterol DRUG

Initially 4 mg daily for one week, 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study.

Albuterol

Placebo DRUG

Initially one capsule daily for one week, then one capsule BID per oral daily for the next 5 weeks. If the one capsule BID per oral is well tolerated, the dose will be increased to two capsules each morning/one capsule each evening for one week, followed by two capsules BID per oral for the remainder of the study.

Placebo Comparator

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing,
• Age: 18+ years at enrollment.
• Receiving enzyme replacement therapy at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks.
• Subjects are capable of giving written consent.

You may not qualify if:

• Continuous invasive ventilation (via tracheostomy or endotracheal tube).
• Clinically relevant illness within two weeks of enrollment including fever \> 38.2 C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
• Chronic heart disease (Myocardial infarction in the past 2 months, arrhythmia, cardiomyopathy).
• History of seizure disorder.
• History of diabetes.
• Hypokalemia.
• History of hyperthyroidism.
• Pregnancy.
• Patients on a non-standard schedule for enzyme replacement therapy; for example, weekly infusions as opposed to infusions every two weeks.
• Anti-rhGAA antibody titer \> 1:100,000
• History of hypersensitivity to Beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent)..
• The use of the following medications:
• diuretics (water pill);
• digoxin (digitalis, Lanoxin);
• beta-blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal);

Sponsors & Collaborators

• Duke University: lead

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Related Publications (2)

• Koeberl DD, Li S, Dai J, Thurberg BL, Bali D, Kishnani PS. beta2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease. Mol Genet Metab. 2012 Feb;105(2):221-7. doi: 10.1016/j.ymgme.2011.11.005. Epub 2011 Nov 11.

  PMID: 22154081 BACKGROUND

• Koeberl DD, Case LE, Desai A, Smith EC, Walters C, Han SO, Thurberg BL, Young SP, Bali D, Kishnani PS. Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease. Mol Genet Metab. 2020 Feb;129(2):67-72. doi: 10.1016/j.ymgme.2019.12.008. Epub 2019 Dec 10.

  PMID: 31839530 DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

Albuterol

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Phenethylamines; Ethylamines

Results Point of Contact

• Title: Dwight Koeberl, M.D., Ph.D.
• Organization: Duke University

dwight.koeberl@duke.edu

919-681-9919

Study Officials

• Dwight d Koeberl, MD, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2013
• First Posted: June 25, 2013
• Study Start: June 1, 2013
• Primary Completion: December 16, 2016
• Study Completion: December 16, 2016
• Last Updated: July 15, 2019
• Results First Posted: February 6, 2018

Record last verified: 2019-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)