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Trial record 3 of 103 for:    Pompe Disease

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01942590
Recruitment Status : Completed
First Posted : September 16, 2013
Results First Posted : October 23, 2017
Last Update Posted : July 2, 2019
Sponsor:
Information provided by (Responsible Party):
Dwight Koeberl, M.D., Ph.D., Duke University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Pompe Disease
Interventions Drug: Clenbuterol
Drug: Placebo
Enrollment 17
Recruitment Details 17 participants signed consent; 13 participants were randomized.
Pre-assignment Details  
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description

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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Period Title: Overall Study
Started 8 5
Completed 7 4
Not Completed 1 1
Reason Not Completed
Withdrawal by Subject             0             1
Adverse Event             1             0
Arm/Group Title Clenbuterol Placebo Comparator Total
Hide Arm/Group Description

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Total of all reporting groups
Overall Number of Baseline Participants 8 5 13
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 8 participants 5 participants 13 participants
52
(37 to 65)
32
(19 to 62)
51
(19 to 65)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 5 participants 13 participants
Female
3
  37.5%
3
  60.0%
6
  46.2%
Male
5
  62.5%
2
  40.0%
7
  53.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 5 participants 13 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
8
 100.0%
5
 100.0%
13
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 5 participants 13 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
8
 100.0%
5
 100.0%
13
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants 5 participants 13 participants
8 5 13
1.Primary Outcome
Title Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement
Hide Description Worsening muscle involvement, as defined by >3x increase in CK from baseline that is >2x the upper limit of normal
Time Frame Any point up to week 52
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 8 5
Measure Type: Number
Unit of Measure: participants
1 0
2.Primary Outcome
Title Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity
Hide Description 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
Time Frame Any point up to week 52
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 8 5
Measure Type: Number
Unit of Measure: participants
0 0
3.Secondary Outcome
Title Change in 6 Minute Walk Test
Hide Description 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.
Time Frame Baseline, week 18
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed 6 minute walk test
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: meters
18.09  (24.85) 6.878  (76.02)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0512
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.434
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
4.Secondary Outcome
Title Change in 6 Minute Walk Test
Hide Description 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.
Time Frame Baseline, week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed 6 minute walk test
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 6 4
Mean (Standard Deviation)
Unit of Measure: meters
16.42  (24.61) -18.13  (40.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0816
Comments Paired t test comparing each subject's performance at Week 52 to Baseline
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3318
Comments Paired t test comparing each subject's performance at Week 52 to Baseline
Method t-test, 1 sided
Comments [Not Specified]
5.Secondary Outcome
Title Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing
Hide Description Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test.
Time Frame Baseline, Week 18
Hide Outcome Measure Data
Hide Analysis Population Description
participants who completed FVC testing
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 8 4
Mean (Standard Deviation)
Unit of Measure: change in FVC measured as % expected
1.575  (5.14) 2.825  (11.78)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2074
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.332
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
6.Secondary Outcome
Title Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing
Hide Description Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
participants who completed FVC testing
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: change in FVC measured as % expected
-5.738  (20.04) 7.775  (11.98)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.233
Comments Paired t test comparing each subject's performance at Week 52 to Baseline
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.142
Comments Paired t test comparing each subject's performance at Week 52 to Baseline
Method t-test, 1 sided
Comments [Not Specified]
7.Secondary Outcome
Title Change in Urinary Glc4 Biomarker
Hide Description 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.
Time Frame Baseline, Week 18
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 3 4
Mean (Standard Deviation)
Unit of Measure: mmol/mol CN
-1.733  (0.6028) 0.0667  (0.666)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.019
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.366
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
8.Secondary Outcome
Title Change in Urinary Glc4 Biomarker
Hide Description [Not Specified]
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
participants who completed urinary Glc4 biomarker collection
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 6 3
Mean (Standard Deviation)
Unit of Measure: mmol/mol CN
-1.1  (1.857) -1.667  (2.401)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.161
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.43
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
9.Secondary Outcome
Title GSGC (Gait, Stairs, Gowers, Arising From a Chair.)
Hide Description 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.
Time Frame Baseline, Week 18, and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
participants who completed GSGC testing
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 7 participants 4 participants
17  (4.69) 7.5  (5.07)
Week 18 Number Analyzed 7 participants 4 participants
15.14  (5.61) 6.5  (3.7)
Week 52 Number Analyzed 6 participants 4 participants
13.8  (5.6) 6.5  (3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.01
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.004
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.154
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.211
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
10.Secondary Outcome
Title Quick Motor Function Test (QMFT)
Hide Description 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.
Time Frame Baseline, Week 18, and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 5 4
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 35  (16.4) 53.75  (13.7)
Week 18 40.6  (17.97) 54.75  (14.08)
Week 52 46.5  (15.1) 56.25  (14.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.006
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.007
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.297
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.196
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
11.Secondary Outcome
Title Late-Life Function and Disability Instrument (LLFDI)
Hide Description 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.
Time Frame Baseline, Week 18, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed LLFDI at the visit. Data was not collected from any participants in the placebo group.
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 4 0
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 4 participants 0 participants
103.75  (26.81)
Week 18 Number Analyzed 3 participants 0 participants
106.7  (39.3)
Week 52 Number Analyzed 4 participants 0 participants
112.5  (27.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3639
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0371
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
12.Secondary Outcome
Title Predicted Maximum Inspiration Pressure (MIP)
Hide Description 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.
Time Frame Baseline, Week 18, and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed the MIP testing
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: percentage of MIP
Baseline Number Analyzed 7 participants 4 participants
56.3  (34.64) 96.8  (17.23)
Week 18 Number Analyzed 7 participants 4 participants
47.4  (20.19) 83.8  (15.13)
Week 52 Number Analyzed 6 participants 4 participants
68.5  (26.86) 104.6  (13.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.268
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0036
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.286
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.279
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
13.Secondary Outcome
Title Maximum Expiratory Pressure (MEP)
Hide Description MEP reflects the strength of the abdominal muscles and other expiratory muscles.
Time Frame Baseline, Week 18, and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed the MEP testing
Arm/Group Title Clenbuterol Placebo Comparator
Hide Arm/Group Description:

Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: percentage of MEP
Baseline Number Analyzed 7 participants 4 participants
40.4  (17.5) 62.8  (14.5)
Week 18 Number Analyzed 7 participants 3 participants
40  (16.8) 83.3  (30.4)
Week 52 Number Analyzed 6 participants 4 participants
53.9  (7.8) 49.2  (11.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.479
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Clenbuterol
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.059
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
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Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 18
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.152
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo Comparator
Comments Baseline, Week 52
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.118
Comments [Not Specified]
Method t-test, 1 sided
Comments [Not Specified]
Time Frame baseline to 52 week
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Clenbuterol Placebo Comparator
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Initially, one capsule (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.

Clenbuterol

Initially, one capsule (placebo) 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 to two capsules until week 52.

Placebo

All-Cause Mortality
Clenbuterol Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)   0/5 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Clenbuterol Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   1/8 (12.50%)   0/5 (0.00%) 
Musculoskeletal and connective tissue disorders     
fall  [1]  1/8 (12.50%)  0/5 (0.00%) 
Indicates events were collected by systematic assessment
[1]
Dislocated artificial hip
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Clenbuterol Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   8/8 (100.00%)   5/5 (100.00%) 
Cardiac disorders     
Palpitations  2/8 (25.00%)  1/5 (20.00%) 
Gastrointestinal disorders     
GI upset  2/8 (25.00%)  0/5 (0.00%) 
General disorders     
anxiety  2/8 (25.00%)  1/5 (20.00%) 
decreased appetite  2/8 (25.00%)  0/5 (0.00%) 
weight gain  2/8 (25.00%)  0/5 (0.00%) 
Insomnia  5/8 (62.50%)  2/5 (40.00%) 
Tremors  4/8 (50.00%)  0/5 (0.00%) 
Musculoskeletal and connective tissue disorders     
Elevated creatine kinase (>3x baseline)  1/8 (12.50%)  0/5 (0.00%) 
Muscle spasma  4/8 (50.00%)  1/5 (20.00%) 
Renal and urinary disorders     
increased urination frequency  3/8 (37.50%)  0/5 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dwight Koeberl, M.D., Ph.D.
Organization: Duke University Health System
Phone: 919-684-2036
Responsible Party: Dwight Koeberl, M.D., Ph.D., Duke University
ClinicalTrials.gov Identifier: NCT01942590     History of Changes
Other Study ID Numbers: Pro00043680
R01FD004364 ( U.S. FDA Grant/Contract )
First Submitted: September 11, 2013
First Posted: September 16, 2013
Results First Submitted: August 25, 2017
Results First Posted: October 23, 2017
Last Update Posted: July 2, 2019