Trial · NCT01942590
Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
NCT01942590, Phase 1, Phase 2, Completed, Dwight Koeberl, M.D., Ph.D.; results first posted 2017-10-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy. Trials, record NCT01942590. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01942590
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
- NCT number
- NCT01942590
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Dwight Koeberl, M.D., Ph.D.
- Conditions
- Pompe Disease
- Interventions
- Clenbuterol, Placebo
- First posted
- 2013-09-16
- Last updated
- 2019-07-02
- Results first posted
- 2017-10-23
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement | 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 |
| Primary | Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity | 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 |
| Secondary | Change in 6 Minute Walk Test | Baseline, week 18 | 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. |
| Secondary | Change in 6 Minute Walk Test | Baseline, week 52 | 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. |
| Secondary | Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Baseline, Week 18 | Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. |
| Secondary | Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Baseline, Week 52 | Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. |
| Secondary | Change in Urinary Glc4 Biomarker | Baseline, Week 18 | 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. |
| Secondary | Change in Urinary Glc4 Biomarker | Baseline, Week 52 | |
| Secondary | GSGC (Gait, Stairs, Gowers, Arising From a Chair.) | Baseline, Week 18, and Week 52 | 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. |
| Secondary | Quick Motor Function Test (QMFT) | Baseline, Week 18, and Week 52 | 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. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement | Any point up to week 52 | participants | NUMBER | |
| PRIMARY | Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity | Any point up to week 52 | participants | NUMBER | |
| SECONDARY | Change in 6 Minute Walk Test | Baseline, week 18 | Participants who completed 6 minute walk test | meters | MEAN |
| SECONDARY | Change in 6 Minute Walk Test | Baseline, week 52 | Participants who completed 6 minute walk test | meters | MEAN |
| SECONDARY | Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Baseline, Week 18 | participants who completed FVC testing | change in FVC measured as % expected | MEAN |
| SECONDARY | Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Baseline, Week 52 | participants who completed FVC testing | change in FVC measured as % expected | MEAN |
| SECONDARY | Change in Urinary Glc4 Biomarker | Baseline, Week 18 | mmol/mol CN | MEAN | |
| SECONDARY | Change in Urinary Glc4 Biomarker | Baseline, Week 52 | participants who completed urinary Glc4 biomarker collection | mmol/mol CN | MEAN |
| SECONDARY | GSGC (Gait, Stairs, Gowers, Arising From a Chair.) | Baseline, Week 18, and Week 52 | participants who completed GSGC testing | units on a scale | MEAN |
| SECONDARY | Quick Motor Function Test (QMFT) | Baseline, Week 18, and Week 52 | units on a scale | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group43
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement | Clenbuterol | 1 | participants | ||
| Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement | Placebo Comparator | 0 | participants | ||
| Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity | Clenbuterol | 0 | participants | ||
| Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity | Placebo Comparator | 0 | participants | ||
| Change in 6 Minute Walk Test | Clenbuterol | 18.09 | Standard Deviation 24.85 | meters | |
| Change in 6 Minute Walk Test | Placebo Comparator | 6.878 | Standard Deviation 76.02 | meters | |
| Change in 6 Minute Walk Test | Clenbuterol | 16.42 | Standard Deviation 24.61 | meters | |
| Change in 6 Minute Walk Test | Placebo Comparator | -18.13 | Standard Deviation 40.9 | meters | |
| Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Clenbuterol | 1.575 | Standard Deviation 5.14 | change in FVC measured as % expected | |
| Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing | Placebo Comparator | 2.825 | Standard Deviation 11.78 | change in FVC measured as % expected |
1 to 10 of 43Source ClinicalTrials.gov
See all 43 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Clenbuterol | 8 | |
| Overall Study | STARTED | Placebo Comparator | 5 | |
| Overall Study | COMPLETED | Clenbuterol | 7 | |
| Overall Study | COMPLETED | Placebo Comparator | 4 | |
| Overall Study | NOT COMPLETED | Clenbuterol | 1 | |
| Overall Study | NOT COMPLETED | Placebo Comparator | 1 |
Source ClinicalTrials.gov
Adverse events22
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | anxiety | General disorders | Clenbuterol | 2 | 8 | |
| other | anxiety | General disorders | Placebo Comparator | 1 | 5 | |
| other | decreased appetite | General disorders | Clenbuterol | 2 | 8 | |
| other | decreased appetite | General disorders | Placebo Comparator | 0 | 5 | |
| other | Elevated creatine kinase (>3x baseline) | Musculoskeletal and connective tissue disorders | Clenbuterol | 1 | 8 | |
| other | Elevated creatine kinase (>3x baseline) | Musculoskeletal and connective tissue disorders | Placebo Comparator | 0 | 5 | |
| other | GI upset | Gastrointestinal disorders | Clenbuterol | 2 | 8 | |
| other | GI upset | Gastrointestinal disorders | Placebo Comparator | 0 | 5 | |
| other | weight gain | General disorders | Clenbuterol | 2 | 8 | |
| other | weight gain | General disorders | Placebo Comparator | 0 | 5 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Clenbuterol | 0 | 8 |
| Total, all-cause mortality | Placebo Comparator | 0 | 5 |
| Total, other adverse events | Clenbuterol | 8 | 8 |
| Total, other adverse events | Placebo Comparator | 5 | 5 |
| Total, serious adverse events | Clenbuterol | 1 | 8 |
| Total, serious adverse events | Placebo Comparator | 0 | 5 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD004364 | Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease | KOEBERL, DWIGHT D | DUKE UNIVERSITY | FOOD AND DRUG ADMINISTRATION | 2013 to 2014 |
Source NIH RePORTER
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Clenbuterol vs Placebo Comparator: deaths from any cause | Trial comparisons |
| Clenbuterol vs Placebo Comparator: Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity | Trial comparisons |
| Clenbuterol vs Placebo Comparator: Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement | Trial comparisons |
| Clenbuterol vs Placebo Comparator: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01942590
GET https://conductscience.org/api/trials/v1/NCT01942590/results?page=2