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Beta-2 Agonist

Clenbuterol for Pompe Disease

Phase 2
Waitlist Available
Led By Dwight Koeberl, MD, PhD
Research Sponsored by Duke University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of Pompe disease by blood Acid alpha-glucosidase (GAA) assay and GAA gene sequencing
Age: 18+ years at enrollment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (week 0) through 52 weeks
Awards & highlights

Study Summary

This trial is testing a combination of drugs to treat a disease called "LOPD". They want to know if it's safe and if it works.

Eligible Conditions
  • Pompe Disease

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (week 0) through 52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (week 0) through 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Changes in 6 minute walk test (MWT) distance
Secondary outcome measures
Changes in graded functional test: Gait, Stairs, Gower, Chair (GSCS)
Changes in graded functional test: Quick Motor Function Test (QMFT)
Changes in pulmonary function tests: Maximum inspiratory pressure (MIP)
+6 more

Side effects data

From 2021 Phase 2 trial • 25 Patients • NCT04245709
40%
Jitters/tremors
32%
Cramps/spasms
28%
Insomnia
24%
Stiffness/spasticity
12%
Elevated liver transaminases
8%
Increased work of breathing
8%
Decreased appetite/weight loss
8%
GI upset/nausea
4%
Dizziness/instability
4%
Anxiety
4%
ECG changes
4%
Elevated thyroxine (T4)
4%
Tachycardia
4%
Constipation
4%
Urinary tract infection
4%
Accelerated disease progression
4%
Increased fasciculations
4%
Tendinitis
4%
Nasal congestion
4%
Tooth removal
4%
Near-drowning accident
4%
Pulmonary emboli
4%
Headache
4%
Palpitations
4%
Elevated creatine kinase (CK)
4%
Hyperglycemia
4%
Elevated PSA
4%
Pruritis
4%
Foot pain
4%
Restless legs
4%
Hot flashes
100%
80%
60%
40%
20%
0%
Study treatment Arm
Open Label Arm

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: clenbuterolExperimental Treatment1 Intervention
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.
Group II: placeboPlacebo Group1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Clenbuterol
FDA approved

Find a Location

Who is running the clinical trial?

Duke UniversityLead Sponsor
2,351 Previous Clinical Trials
3,409,546 Total Patients Enrolled
Dwight Koeberl, MD, PhDPrincipal InvestigatorDuke University

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~0 spots leftby Jun 2025