Pompe DiseasePhase 1/2NCT01942590
Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
A Phase 1/2 study testing Clenbuterol in people with Pompe Disease, run by Dwight Koeberl, M.D., Ph.D. at 1 site in United States.
Trial summary
Funding Source- FDA OOPD The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy (ERT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease
- 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
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2013to 2 Sep 2016
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 assay and acid alpha-glucosidase gene sequencing,
- Age: 18+ years at enrollment,
- Receiving ERT at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks,
- Subjects are capable of giving written consent.
Who cannot join
- 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, arrythmia, cardiomyopathy)
- Tachycardia
- History of seizure disorder
- Hyperthyroidism
Show all 19Show fewer
- Pheochromocytoma
- Pregnancy
- History of diabetes
- History of hypersensitivity to beta 2-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.
- 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 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: A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
Funding Source- FDA OOPD
The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy (ERT).
Read the detailed description
This is a 52 week Phase I/II double-blind, randomized, placebo-controlled study of adjunctive clenbuterol in LOPD (Table 2, Section 6). All subjects will be evaluated at Week 0 and Week 6 to establish a baseline for motor function testing. At Week 6, subjects will be randomized 3:2 to clenbuterol or placebo, and evaluated for safety and efficacy during the Week 12 and 18 visits. The Investigational Drug Service will maintain double-blinding by providing either the study drug or placebo (over-encapsulated tablets) directly to subjects. The drug (or placebo) will be initiated at the Week 6 visit in a staged manner (first once daily and later BID), and the dose will be increased at the Week 12 visit in a similarly staged manner to minimize AEs and related attrition. All subjects will return for a final visit after a total of 52 weeks in the study.
In terms of standard of care, the subject will have two clinical visits (charged to the subject and/or the subject's insurance company), one at the initiation of the study drug (baseline) and one at the study completion (52 weeks). Study drug will be attempted to be initiated during the "off week", approximately one week following a dose of ERT, and ERT will continue throughout the duration of the study. Thereafter, study visits will be during the "off week". The 6, 12, and 18 week visits will be research visits (not charged to the subject and/or the subject's insurance company) in order to determine subject's overall health status and measure early signs of motor improvement. The initial dose of clenbuterol will be 40 mcg per oral each morning for one week, followed by 40 mcg BID for the next 5 weeks until the week 12 visit. 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 18 visit. If 80 mcg BID is tolerated at Week 18, the subject will continue on that dose until Week 52.
Compliance will be discussed at the Week 6, Week 12, and Week 18 visits. The subject will have phone visits during Week 1, 7, 13, 36, and 52, and compliance will be discussed then. We will call subjects daily during the first week following initiation of study drug (Week 7) and dosage escalation (Week 13) to support subjects through the early adverse effects of tachyphylaxis that may lead to premature termination. An interim call will occur during Week 36 to monitor compliance. Subjects who admit non-compliance, missing >6 doses of the study drug, will be considered non-compliant and withdrawn from the study. Subjects will be called during Week 1 and Week 52, 3 days following the muscle biopsy. All phone calls will review AEs (Table).
The efficacy of clenbuterol treatment during ERT in patients with LOPD will be evaluated with muscle and pulmonary function testing as the primary endpoints. A secondary endpoint, the urinary Glc4 biomarker, will be monitored when the subjects are evaluated at baseline, week 18 and week 52. The impact of enhanced CI-MPR-mediated uptake of GAA will be analyzed by comparing the muscle function, pulmonary function, and biochemical correction of muscle in subjects with LOPD treated with ERT, both prior to and during simultaneous β2 agonist therapy.
- Conditions
- Pompe Disease
- Being tested
- Clenbuterol, Placebo
- Keywords
- 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
PlaceboDrug
Also called Capsule
| Study group | Role | What they receive |
|---|---|---|
| Clenbuterol | Experimental | Initially, 40 mcg each morning for one week. Then, increase to 40 mcg BID for the next 5 weeks. If tolerated, will increase to 80 mcg in the morning and 40 mcg in the evening for one week. Then, last dose increase will be 80 mcg BID until week 52. Receives: Drug: Clenbuterol |
| Placebo Comparator | Placebo comparator | Initially, one capsule each morning for one week. Then, increase to one capsule BID for the next 5 weeks. If tolerated, will increase to two capsules in the morning and one capsule in the evening for one week. Then, last dose increase will be two capsules BID until week 52. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement
Worsening muscle involvement, as defined by >3x increase in CK from baseline that is >2x the upper limit of normal
Measured: Any point up to week 52
Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity
Liver toxicity, as defined by a >3x increase in AST or ALT from the respective baseline values and/or an increase in direct, indirect or total bilirubin of >3x the upper limit of normal
Measured: Any point up to week 52
Secondary outcomes (11)
Change in 6 Minute Walk Test
Assess exercise tolerance in study patients; test administered by physical therapist. Subjects were asked to walk for 6 minutes, unassisted. The distance walked was recorded in meters.
Measured: Baseline, week 18
Change in 6 Minute Walk Test
Assess exercise tolerance in study patients; test administered by physical therapist. Subjects were asked to walk for 6 minutes, unassisted. The distance walked was recorded in meters.
Measured: Baseline, week 52
Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing
Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test.
Measured: Baseline, Week 18
Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing
Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test.
Measured: Baseline, Week 52
Change in Urinary Glc4 Biomarker
The Glc4 biomarker is measured in urine and correlates with muscle glycogen content. It is a noninvasive measurement that serves as a biomarker for Pompe disease.
Measured: Baseline, Week 18
Change in Urinary Glc4 Biomarker
Measured: Baseline, Week 52
GSGC (Gait, Stairs, Gowers, Arising From a Chair.)
The GSGC is a criterion referenced assessment designed to measure functional status and change in gross motor function over time and, in particular, to measure clinically relevant change. Consists of 4 components: Gait, Climbing Stairs, Gower's Manuever, Arising From a Chair. Lowest score 4 = normal muscle function, highest score 27 = unable to perform motor function tests.
Measured: Baseline, Week 18, and Week 52
Quick Motor Function Test (QMFT)
The QMFT is a criterion referenced assessment designed to measure functional status and change in gross motor function over time and, in particular, to measure clinically relevant change. Consists of 16 motor function tests. Lowest score 0 = unable to perform motor function tests, highest score 64 = normal muscle function.
Measured: Baseline, Week 18, and Week 52
Late-Life Function and Disability Instrument (LLFDI)
The Late-Life Function \& Disability Instrument (Late-Life FDI) is an evaluative outcome instrument for community-dwelling older adults. Highest score 240 = normal function and no disability, lowest score 0 = low levels of frequency of participating in life tasks.
Measured: Baseline, Week 18, Week 52
Predicted Maximum Inspiration Pressure (MIP)
MIP is a measurement of inspiratory muscle weakness, including weakness of the diaphragm. MIP is decreased in Pompe disease and reflects weakness of respiratory muscles.
Measured: Baseline, Week 18, and Week 52
Maximum Expiratory Pressure (MEP)
MEP reflects the strength of the abdominal muscles and other expiratory muscles.
Measured: Baseline, Week 18, and Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2013 (estimated)
- Study startSep 2013
- Primary completion2 Sep 2016
- Study completion2 Sep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dwight Koeberl, M.D., Ph.D.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Dwight Koeberl, M.D., Ph.D., Duke University
- Investigators
- Dwight D Koeberl, MD, PhD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01942590
- Sponsor's protocol ID
- Pro00043680
- Other IDs
- R01FD004364
- Status last verified
- Jun 2019
- First submitted
- 11 Sep 2013
- Last updated
- 2 Jul 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Kamalakkannan G, Petrilli CM, George I, LaManca J, McLaughlin BT, Shane E, Mancini DM, Maybaum S. Clenbuterol increases lean muscle mass but not endurance in patients with chronic heart failure. J Heart Lung Transplant. 2008 Apr;27(4):457-61. doi: 10.1016/j.healun.2008.01.013.
PubMed 18374884Koeberl DD, Luo X, Sun B, McVie-Wylie A, Dai J, Li S, Banugaria SG, Chen YT, Bali DS. Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle. Mol Genet Metab. 2011 Jun;103(2):107-12. doi: 10.1016/j.ymgme.2011.02.006. Epub 2011 Feb 13.
PubMed 21397538Koeberl 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.
PubMed 22154081Koeberl DD, Case LE, Smith EC, Walters C, Han SO, Li Y, Chen W, Hornik CP, Huffman KM, Kraus WE, Thurberg BL, Corcoran DL, Bali D, Bursac N, Kishnani PS. Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease. Mol Ther. 2018 Sep 5;26(9):2304-2314. doi: 10.1016/j.ymthe.2018.06.023. Epub 2018 Jul 5.
PubMed 30025991
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT01942590
- Acronym / sponsor ID
- Pro00043680
- Conditions
- Pompe Disease
- Keywords
- Pompe disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Dwight Koeberl, M.D., Ph.D.University, hospital or other
- Study start
- Sep 2013
- Primary completion
- 2 Sep 2016 (actual)
- Study completion
- 2 Sep 2016 (actual)
- First posted
- 16 Sep 2013 (estimated)
- Results
- Posted 23 Oct 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Funding Source- FDA OOPD
The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy (ERT).
Detailed description
This is a 52 week Phase I/II double-blind, randomized, placebo-controlled study of adjunctive clenbuterol in LOPD (Table 2, Section 6). All subjects will be evaluated at Week 0 and Week 6 to establish a baseline for motor function testing. At Week 6, subjects will be randomized 3:2 to clenbuterol or placebo, and evaluated for safety and efficacy during the Week 12 and 18 visits. The Investigational Drug Service will maintain double-blinding by providing either the study drug or placebo (over-encapsulated tablets) directly to subjects. The drug (or placebo) will be initiated at the Week 6 visit in a staged manner (first once daily and later BID), and the dose will be increased at the Week 12 visit in a similarly staged manner to minimize AEs and related attrition. All subjects will return for a final visit after a total of 52 weeks in the study.
In terms of standard of care, the subject will have two clinical visits (charged to the subject and/or the subject's insurance company), one at the initiation of the study drug (baseline) and one at the study completion (52 weeks). Study drug will be attempted to be initiated during the "off week", approximately one week following a dose of ERT, and ERT will continue throughout the duration of the study. Thereafter, study visits will be during the "off week". The 6, 12, and 18 week visits will be research visits (not charged to the subject and/or the subject's insurance company) in order to determine subject's overall health status and measure early signs of motor improvement. The initial dose of clenbuterol will be 40 mcg per oral each morning for one week, followed by 40 mcg BID for the next 5 weeks until the week 12 visit. 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 18 visit. If 80 mcg BID is tolerated at Week 18, the subject will continue on that dose until Week 52.
Compliance will be discussed at the Week 6, Week 12, and Week 18 visits. The subject will have phone visits during Week 1, 7, 13, 36, and 52, and compliance will be discussed then. We will call subjects daily during the first week following initiation of study drug (Week 7) and dosage escalation (Week 13) to support subjects through the early adverse effects of tachyphylaxis that may lead to premature termination. An interim call will occur during Week 36 to monitor compliance. Subjects who admit non-compliance, missing >6 doses of the study drug, will be considered non-compliant and withdrawn from the study. Subjects will be called during Week 1 and Week 52, 3 days following the muscle biopsy. All phone calls will review AEs (Table).
The efficacy of clenbuterol treatment during ERT in patients with LOPD will be evaluated with muscle and pulmonary function testing as the primary endpoints. A secondary endpoint, the urinary Glc4 biomarker, will be monitored when the subjects are evaluated at baseline, week 18 and week 52. The impact of enhanced CI-MPR-mediated uptake of GAA will be analyzed by comparing the muscle function, pulmonary function, and biochemical correction of muscle in subjects with LOPD treated with ERT, both prior to and during simultaneous β2 agonist therapy.
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: ClenbuterolInitially, 40 mcg each morning for one week. Then, increase to 40 mcg BID for the next 5 weeks. If tolerated, will increase to 80 mcg in the morning and 40 mcg in the evening for one week. Then, last dose increase will be 80 mcg BID until week 52. |
| Placebo Comparator | Placebo comparator | Drug: PlaceboInitially, one capsule each morning for one week. Then, increase to one capsule BID for the next 5 weeks. If tolerated, will increase to two capsules in the morning and one capsule in the evening for one week. Then, last dose increase will be two capsules BID 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 (4 listed)
- Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing,
- Age: 18+ years at enrollment,
- Receiving ERT at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks,
- Subjects are capable of giving written consent.
Key exclusion criteria (19 listed)
- 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, arrythmia, cardiomyopathy)
- Tachycardia
- History of seizure disorder
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: Spiropent | Drug | Clenbuterol |
| PlaceboAlso: Capsule | Drug | Placebo Comparator |
Primary outcome
Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement
Time frame: Any point up to week 52
Worsening muscle involvement, as defined by >3x increase in CK from baseline that is >2x the upper limit of normal
Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity
Time frame: Any point up to week 52
Liver toxicity, as defined by a >3x increase in AST or ALT from the respective baseline values and/or an increase in direct, indirect or total bilirubin of >3x the upper limit of normal
11 secondary outcomes
Change in 6 Minute Walk Test
Time frame: Baseline, week 18
Change in 6 Minute Walk Test
Time frame: Baseline, week 52
Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing
Time frame: Baseline, Week 18
Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing
Time frame: Baseline, Week 52
Change in Urinary Glc4 Biomarker
Time frame: Baseline, Week 18
Change in Urinary Glc4 Biomarker
Time frame: Baseline, Week 52
GSGC (Gait, Stairs, Gowers, Arising From a Chair.)
Time frame: Baseline, Week 18, and Week 52
Quick Motor Function Test (QMFT)
Time frame: Baseline, Week 18, and Week 52
Late-Life Function and Disability Instrument (LLFDI)
Time frame: Baseline, Week 18, Week 52
Predicted Maximum Inspiration Pressure (MIP)
Time frame: Baseline, Week 18, and Week 52
Maximum Expiratory Pressure (MEP)
Time frame: Baseline, Week 18, and Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 18374884Kamalakkannan G, Petrilli CM, George I, LaManca J, McLaughlin BT, Shane E, Mancini DM, Maybaum S. Clenbuterol increases lean muscle mass but not endurance in patients with chronic heart failure. J Heart Lung Transplant. 2008 Apr;27(4):457-61. doi: 10.1016/j.healun.2008.01.013.
- Background: PubMed 21397538Koeberl DD, Luo X, Sun B, McVie-Wylie A, Dai J, Li S, Banugaria SG, Chen YT, Bali DS. Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle. Mol Genet Metab. 2011 Jun;103(2):107-12. doi: 10.1016/j.ymgme.2011.02.006. Epub 2011 Feb 13.
- Background: PubMed 22154081Koeberl 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.
- Derived from this study: PubMed 30025991Koeberl DD, Case LE, Smith EC, Walters C, Han SO, Li Y, Chen W, Hornik CP, Huffman KM, Kraus WE, Thurberg BL, Corcoran DL, Bali D, Bursac N, Kishnani PS. Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease. Mol Ther. 2018 Sep 5;26(9):2304-2314. doi: 10.1016/j.ymthe.2018.06.023. Epub 2018 Jul 5.
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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 D 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 1/2 study testing Clenbuterol in people with Pompe Disease, run by Dwight Koeberl, M.D., Ph.D. 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 4 inclusion and 19 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: finished, as of the sponsor's last update on 2 Jul 2019. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Yes. Results are posted in the registry (first posted 23 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement (time frame: Any point up to week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01942590, which also lists 4 linked publications.
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 2 Jul 2019.