Pompe Disease (Late-onset)Phase 2NCT04094948
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
A Phase 2 study testing Clenbuterol in people with Pompe Disease (Late-onset), run by Duke University at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease (Late-onset)
- What is being tested?
- Clenbuterol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2023to 30 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- Continuous invasive ventilation (via tracheostomy or endotracheal tube)
- 6MWT 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
Show all 25Show fewer
- 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).
- Having started respiratory muscle strength training in the last 6 months prior to study day 1 or having discontinued respiratory muscle strength training in the 6-month period preceding study day 1, or having started respiratory strength training greater than 6 months prior to study day 1 and unwilling to continue for the first year of study participation.
- Received an investigational drug or participated in another interventional study within 90 days of Study Day 1.
- Anti-rhGAA IgG with sustained titer >1:25.600 for >6 months at time of enrollment.
- The use of the following concommitant meds is prohibited during the study:
- diuretics (water pill);
- digoxin (digitalis, Lanoxin);
- beta-blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal);
- tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor);
- Monoamine oxidase (MAO) inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or
- other bronchodilators such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer).
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27710
About this study
Official title: Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement Therapy
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)
- Being tested
- Clenbuterol, Placebos
- Keywords
- Pompe disease, LOPD, Late-onset Pompe Disease
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClenbuterolDrug
Also called Spiropent
20 mcg spiropent tablets will be overencapsulated (two 20 mcg tablets per capsule)
PlacebosDrug
dextrose-filled capsules
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in 6 minute walk test (MWT) distance
Measured: Baseline (week 0) through 52 weeks
Secondary outcomes (9)
Changes in pulmonary function tests: forced expiratory volume in 1 second (FEV1)
Measured: Baseline (week 0) through 52 weeks
Changes in pulmonary function tests: forced vital capacity (FVC)
Measured: Baseline (week 0) through 52 weeks
Changes in pulmonary function tests: maximum expiratory pressure (MEP)
Measured: Baseline (week 0) through 52 weeks
Changes in pulmonary function tests: Maximum inspiratory pressure (MIP)
Measured: Baseline (week 0) through 52 weeks
Changes in graded functional test: Gait, Stairs, Gower, Chair (GSCS)
Measured: Baseline (week 0) through 52 weeks
Changes in graded functional test: Quick Motor Function Test (QMFT)
Measured: Baseline (week 0) through 52 weeks
Changes in the concentration of alanine transaminase (ALT) in serum
Avoidance of liver toxicity as defined by a a persistent (sustained >2 weeks) >3x increase in aspartate aminotransferase (AST) or alanine aminotransferase (ALT) from the respective baseline values and/or an increase in direct, indirect or total bilirubin of >3x the upper limit of normal (no liver toxicity has been reported in association with clenbuterol administration and therefore repeat testing should be acceptable)
Measured: Baseline (week 0) through 52 weeks
Changes in the concentration of creatine kinase (CK) in serum
Avoidance of Worsening muscle involvement (i.e. muscle weakness, cramping, or fatigue) accompanied by a persistent (sustained >2 weeks) >3x increase in CK from baseline that is >2x the upper limit of normal (elevated CK is associated with LOPD and minor elevations of CK have been reported in association with clenbuterol administration, therefore repeat testing should be acceptable).
Measured: Baseline (week 0) through 52 weeks
Changes in the concentration of urinary glucose tetramer (Glc4)
Measured: Baseline (week 0) through 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2019
- Study start1 Jan 2023 (estimated)
- Primary completion30 Jun 2025 (estimated)
- Study completion30 Dec 2025 (estimated)
Why it stopped: hasn't got the funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease (Late-onset)Studies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dwight Koeberl, M.D., Ph.D., Duke University
- Investigators
- Dwight Koeberl, MD, PhD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04094948
- Sponsor's protocol ID
- Pro00102462
- Status last verified
- Aug 2022
- First submitted
- 17 Sep 2019
- Last updated
- 17 Aug 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT04094948
- Acronym / sponsor ID
- Pro00102462
- Conditions
- Pompe Disease (Late-onset)
- Keywords
- Pompe disease, LOPD, Late-onset Pompe Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: hasn't got the funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- 1 Jan 2023 (estimated)
- Primary completion
- 30 Jun 2025 (estimated)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 19 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| clenbuterol | Experimental | Drug: ClenbuterolThe 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. |
| placebo | Placebo comparator | Drug: PlacebosInitially, 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (25 listed)
- Continuous invasive ventilation (via tracheostomy or endotracheal tube)
- 6MWT 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ClenbuterolAlso: Spiropent20 mcg spiropent tablets will be overencapsulated (two 20 mcg tablets per capsule) | Drug | clenbuterol |
| Placebosdextrose-filled capsules | Drug | placebo |
Primary outcome
Changes in 6 minute walk test (MWT) distance
Time frame: Baseline (week 0) through 52 weeks
9 secondary outcomes
Changes in pulmonary function tests: forced expiratory volume in 1 second (FEV1)
Time frame: Baseline (week 0) through 52 weeks
Changes in pulmonary function tests: forced vital capacity (FVC)
Time frame: Baseline (week 0) through 52 weeks
Changes in pulmonary function tests: maximum expiratory pressure (MEP)
Time frame: Baseline (week 0) through 52 weeks
Changes in pulmonary function tests: Maximum inspiratory pressure (MIP)
Time frame: Baseline (week 0) through 52 weeks
Changes in graded functional test: Gait, Stairs, Gower, Chair (GSCS)
Time frame: Baseline (week 0) through 52 weeks
Changes in graded functional test: Quick Motor Function Test (QMFT)
Time frame: Baseline (week 0) through 52 weeks
Changes in the concentration of alanine transaminase (ALT) in serum
Time frame: Baseline (week 0) through 52 weeks
Changes in the concentration of creatine kinase (CK) in serum
Time frame: Baseline (week 0) through 52 weeks
Changes in the concentration of urinary glucose tetramer (Glc4)
Time frame: Baseline (week 0) through 52 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dwight Koeberl, MD, PhD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Clenbuterol in people with Pompe Disease (Late-onset), run by Duke University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 17 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in 6 minute walk test (MWT) distance (time frame: Baseline (week 0) through 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2022.