Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

NCT04094948 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: Pro00102462
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The goals of this study are to determine safety and efficacy with regard to motor function of oral clenbuterol in combination with ERT in subjects with LOPD

Conditions

Pompe Disease (Late-onset)

Keywords

Pompe disease; LOPD; Late-onset Pompe Disease

Outcome Measures

Primary Outcomes (1)

• Changes in 6 minute walk test (MWT) distance

  Baseline (week 0) through 52 weeks

Secondary Outcomes (9)

• Changes in pulmonary function tests: forced expiratory volume in 1 second (FEV1)

  Baseline (week 0) through 52 weeks

• Changes in pulmonary function tests: forced vital capacity (FVC)

  Baseline (week 0) through 52 weeks

• Changes in pulmonary function tests: maximum expiratory pressure (MEP)

  Baseline (week 0) through 52 weeks

• Changes in pulmonary function tests: Maximum inspiratory pressure (MIP)

  Baseline (week 0) through 52 weeks

• Changes in graded functional test: Gait, Stairs, Gower, Chair (GSCS)

  Baseline (week 0) through 52 weeks

Study Arms (2)

clenbuterol

EXPERIMENTAL

The initial dose of clenbuterol will be 40 mcg per oral each morning for one week, followed by 40 mcg twice per day (BID) for the next 5 weeks until Week 6. If the 40 mcg BID per oral is well tolerated, the dose will be increased to 80 mcg each morning/40 mcg each evening for one week, followed by 80 mcg BID for the next 5 weeks until the Week 12 visit. If 80 mcg BID is tolerated at Week 12, the subject will continue on that dose until Week 52.

Drug: Clenbuterol

placebo

PLACEBO COMPARATOR

Initially, one capsule each morning for one week, followed by one capsule BID for the next 5 weeks until Week 6. If tolerated, the dose will be increased to two capsules each morning and 1 capsule each evening for one week, followed by two capsules BID for the next 5 weeks until the Week 12 visit. If two capsules BID is tolerated at Week 12, the subject will continue on that dose until Week 52.

Drug: Placebos

Interventions

Clenbuterol DRUG

20 mcg spiropent tablets will be overencapsulated (two 20 mcg tablets per capsule)

Also known as: Spiropent

clenbuterol

Placebos DRUG

dextrose-filled capsules

placebo

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 (GAA) assay and GAA gene sequencing,
• Age: 18+ years at enrollment,
• Receiving enzyme replacement therapy (ERT) at a stable dose for \>104 weeks,
• FVC \>15% of expected (supine).
• Subjects are capable of giving written consent.
• Able to walk at least 100 meters on the 6 minute walk test (6MWT) (with assistive devices permitted).

You may not qualify if:

• Continuous invasive ventilation (via tracheostomy or endotracheal tube)
• MWT distance \>90% of expected performance (% expected)
• FVC \>90% of expected (upright).
• Clinically relevant illness within two weeks of enrollment including fever \> 38.2o C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
• Chronic heart disease (Myocardial infarction, arrythmia, cardiomyopathy)
• Tachycardia
• History of seizure disorder
• Hyperthyroidism
• Pheochromocytoma
• Pregnancy
• History of diabetes
• History of hypersensitivity to β2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent),
• Patients on a non-standard schedule for ERT; for example, weekly infusions as opposed to infusions every two weeks.
• Treatment for asthma in the previous 12 months.
• Renal insufficiency (elevated serum creatinine).

Sponsors & Collaborators

• Duke University: lead

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

Clenbuterol

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

Study Officials

• Dwight Koeberl, MD, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: September 17, 2019
• First Posted: September 19, 2019
• Study Start: January 1, 2023
• Primary Completion: June 30, 2025
• Study Completion: December 30, 2025
• Last Updated: August 17, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)