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A Trial of PF-06252616 in Ambulatory Participants With LGMD2I

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ClinicalTrials.gov Identifier: NCT02841267
Recruitment Status : Completed
First Posted : July 22, 2016
Results First Posted : October 19, 2020
Last Update Posted : October 19, 2020
Sponsor:
Collaborator:
Pfizer
Information provided by (Responsible Party):
Kathryn Wagner, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition LGMD2I
Intervention Drug: PF 06252616
Enrollment 19
Recruitment Details  
Pre-assignment Details One participant in Cohort 2 was consented, but elected to withdraw from the study prior to assignment to a treatment arm.
Arm/Group Title Low Dose, Cohort 1 Middle Dose, Cohort 2 High Dose, Cohort 3
Hide Arm/Group Description

4 subjects will be enrolled in cohort 1 and will receive an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, if no stopping rules have been met, subjects will be receive an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.

PF 06252616

8 subjects will be enrolled in cohort 2 and receive 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.

PF 06252616

8 subjects will be enrolled in cohort 3 and receive 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.

PF 06252616

Period Title: Lead-in (Untreated)
Started [1] 4 7 8
Completed 4 7 8
Not Completed 0 0 0
[1]
Participants were observed for 16 weeks prior to treatment.
Period Title: Treatment Period
Started 4 [1] 7 8
Completed 4 7 8
Not Completed 0 0 0
[1]
Cohort 1 was invited to continue receiving 40mg/kg of study drug every 4 weeks for 28 weeks.
Period Title: Extension Study
Started 4 [1] 6 [2] 6 [3]
Completed 4 6 6
Not Completed 0 0 0
[1]
Cohort 1 was invited to continue receiving 40mg/kg of study drug every 4 weeks for 28 weeks.
[2]
Cohort 2 was invited to continue receiving 40mg/kg of study drug every 4 weeks for 40 weeks.
[3]
Cohort 3 was invited to continue receiving 40mg/kg of study drug every 4 weeks for 24 weeks.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose Total
Hide Arm/Group Description 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. Total of all reporting groups
Overall Number of Baseline Participants 4 7 8 19
Hide Baseline Analysis Population Description
Target enrollment was reached for Cohort 1 and Cohort 3. For Cohort 2, 8 subjects were screened, but 1 voluntarily withdrew prior to enrollment and was not replaced.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 7 participants 8 participants 19 participants
45  (8) 41  (18) 34  (9.9) 39  (13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 7 participants 8 participants 19 participants
Female
3
  75.0%
4
  57.1%
5
  62.5%
12
  63.2%
Male
1
  25.0%
3
  42.9%
3
  37.5%
7
  36.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 7 participants 8 participants 19 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
4
 100.0%
7
 100.0%
8
 100.0%
19
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Incidence of Dose Limiting or Intolerability Treatment Related Adverse Events
Hide Description Adverse events include subject-reported symptoms as well as clinically-significant changes in laboratory testing, vital signs, and suicide screening (based on the Columbia Suicide Severity Rating Scale).
Time Frame Baseline through 64 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled subjects are included in the analysis.
Arm/Group Title Cohort 1 Cohort 2, Middle Dose Cohort 3, High Dose
Hide Arm/Group Description:
4 subjects received 32 weeks of treatment with 5mg/kg PF 06252616 IV every 4 weeks followed by an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Measure Type: Number
Unit of Measure: events
Any adverse event 45 21 40
Serious adverse event 0 0 0
2.Secondary Outcome
Title Maximum Observed Serum Concentration at Steady State (Cmax, ss) of GDF-8
Hide Description The concentration of myostatin (GDF-8) was measured in serum 2 hours after dose administration at two visits (Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3)where drug concentration had reached steady state. The highest concentration from these two time points was averaged for each cohort.
Time Frame Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 40mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: ng/ml
9.02  (3.20) 8.96  (3.02) 9.67  (6.45)
3.Secondary Outcome
Title Minimum Observed Serum Trough Concentration at Steady State (Ctrough,ss) of GDF-8
Hide Description The concentration of myostatin (GDF-8) was measured in serum prior to dose administration at two time points (Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3) where drug concentration had reached steady state. The lowest concentration from these two time points was averaged for each cohort.
Time Frame Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 40mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: ng/ml
8.80  (2.96) 9.68  (2.21) 9.78  (7.61)
4.Secondary Outcome
Title Maximum Observed Serum Concentration (Cmax) of PF-06252616
Hide Description The peak concentration of study drug (PF-06252616) was measured in serum following dose administration at two time points (Day 113 and Day 169) where drug concentration had reached steady state. The higher concentration from these two time points was averaged for each cohort.
Time Frame Day 113 and Day 169
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants were analyzed. For Cohort 1, measurements are provided for 5mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: ng/ml
215306.5  (29490.4) 779015.7  (115450.0) 1625203  (271159.6)
5.Secondary Outcome
Title Minimum Observed Serum Trough Concentration (Ctrough) of PF-06252616
Hide Description The peak concentration of study drug (PF-06252616) was measured in serum prior to dose administration at two time points (Day 113 and Day 169) where drug concentration had reached steady state. The lower concentration from these two time points was averaged for each cohort.
Time Frame Day 113 and Day 169
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 5mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 7
Mean (Standard Deviation)
Unit of Measure: ng/ml
67016.8  (25887.3) 224024.1  (58734.5) 408814.1  (96607.6)
6.Secondary Outcome
Title Immunogenicity: Incidence of Anti-drug Antibody
Hide Description Blood samples were tested for the presence of anti-drug antibodies prior to the initiation of study drug, at dose escalation (Cohort 1 only), and at 3 separate time points after the last dose was given.
Time Frame Baseline through 96 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled subjects were included in the analysis.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
7.Secondary Outcome
Title Mean Change From Baseline in 10 Meter Walk/Run Time in Seconds
Hide Description Subjects are asked to run or walk as quickly as possible for 10 meters from a standing position. The total time to traverse 10 meters is recorded in seconds.
Time Frame Baseline through 32 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled subject were included in the analysis. For Cohort 1, data is provided for the 5mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: seconds
1.60  (2.95) -0.09  (0.11) 0.18  (0.31)
8.Secondary Outcome
Title Mean Change From Baseline of Forced Vital Capacity in Liters
Hide Description The total forced vital capacity was measured using a bedside spirometer. The best of 3 trials was recorded.
Time Frame Baseline through 32 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled subjects were included in the analysis. For Cohort 1, data is provided for the 5mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: liters
0.03  (0.14) 0.02  (0.15) -0.05  (0.14)
9.Secondary Outcome
Title Mean Change From Baseline in 2MWD in Meters
Hide Description Average change in distance (in meters) walked in 2 minutes.
Time Frame Baseline through 32 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 5mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: meters
-4.25  (9.91) 2.00  (7.77) 2.13  (5.17)
10.Secondary Outcome
Title Mean Change From Baseline in TUG in Seconds
Hide Description The timed-up-and-go test (TUG) is the total time it takes the subject to rise from a seated position, walk to a marker 3 meters away, return to the chair, and sit.
Time Frame Baseline through 32 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled subjects are included in the analysis. For Cohort 1, data is provided for the 5mg/kg dosing period.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: seconds
0.67  (1.15) 0.29  (1.11) -0.50  (1.51)
11.Secondary Outcome
Title Mean Change From Baseline in Muscle Strength as Measured by Modified MRC Scale
Hide Description Twenty-two muscle groups were measured on a modified MRC scale ranging from 1 through 12. The total scores from all 22 muscle groups were added to generate a summary score ranging from 12 to 264 with higher scores signifying greater strength. The change in summary score was calculated over the first 32 weeks of treatment.
Time Frame Baseline through 32 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants were analyzed after 32 weeks of treatment.
Arm/Group Title Cohort 1 - Low Dose Cohort 2 - Middle Dose Cohort 3 - High Dose
Hide Arm/Group Description:
4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks.
7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks.
8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks.
Overall Number of Participants Analyzed 4 7 8
Mean (Standard Deviation)
Unit of Measure: score on a scale
9.75  (6.18) 7.57  (8.62) 2.43  (4.54)
Time Frame Baseline through 124 weeks
Adverse Event Reporting Description Subject reported adverse events, as well as clinically relevant changes in laboratory screening, physical examination, suicide screening, cardiac screening are reported.
 
Arm/Group Title Lead-in Cohort 1 - Low Dose Cohort 1 - High Dose Cohort 1 Extension Cohort 2 Cohort 2 Extension Cohort 3 Cohort 3 Extension
Hide Arm/Group Description All subjects were observed in a 16-week untreated lead-in-phase prior to allocation to dosing cohorts. Subjects received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks for 32 weeks. Following the Low Dose period, subjects in Cohort 1 received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. Subjects enrolled in Cohort 1 were given the option to receive PF 06252616 at 40 mg/kg every 4 weeks for an additional 28 weeks. Subjects received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. Subjects enrolled in Cohort 2 were given the option to receive PF 06252616 at 40 mg/kg every 4 weeks for an additional 40 weeks. Subjects received 40 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. Subjects enrolled in Cohort 3 were given the option to receive PF 06252616 at 40 mg/kg every 4 weeks for an additional 24 weeks.
All-Cause Mortality
Lead-in Cohort 1 - Low Dose Cohort 1 - High Dose Cohort 1 Extension Cohort 2 Cohort 2 Extension Cohort 3 Cohort 3 Extension
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/19 (0.00%)      0/4 (0.00%)      0/4 (0.00%)      0/4 (0.00%)      0/7 (0.00%)      0/6 (0.00%)      0/8 (0.00%)      0/6 (0.00%)    
Hide Serious Adverse Events
Lead-in Cohort 1 - Low Dose Cohort 1 - High Dose Cohort 1 Extension Cohort 2 Cohort 2 Extension Cohort 3 Cohort 3 Extension
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/19 (5.26%)      0/4 (0.00%)      0/4 (0.00%)      0/4 (0.00%)      0/7 (0.00%)      1/6 (16.67%)      0/8 (0.00%)      0/6 (0.00%)    
Gastrointestinal disorders                 
Intestinal obstruction * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Musculoskeletal and connective tissue disorders                 
Stress fracture * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  4 0/8 (0.00%)  0 0/6 (0.00%)  0
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Lead-in Cohort 1 - Low Dose Cohort 1 - High Dose Cohort 1 Extension Cohort 2 Cohort 2 Extension Cohort 3 Cohort 3 Extension
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/19 (47.37%)      4/4 (100.00%)      4/4 (100.00%)      4/4 (100.00%)      6/7 (85.71%)      4/6 (66.67%)      7/8 (87.50%)      6/6 (100.00%)    
Blood and lymphatic system disorders                 
Anemia * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Leukopenia * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Cardiac disorders                 
Worsening cardiomyopathy * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 3/7 (42.86%)  3 0/6 (0.00%)  0 2/8 (25.00%)  2 1/6 (16.67%)  1
Ear and labyrinth disorders                 
Ear infection * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Ear pain * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Hearing loss * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Eye disorders                 
Blepharitis * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Vision abnormality - not otherwise specified * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Eye twitching * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Gastrointestinal disorders                 
Abdominal pain * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Diarrhea * 1  1/19 (5.26%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Gastroesophageal reflux * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  2 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Nausea * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 2/8 (25.00%)  2 0/6 (0.00%)  0
Hemoccult positive * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
New diagnosis - umbilical hernia * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
General disorders                 
Decreased appetite * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Fatigue * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 2/8 (25.00%)  2 0/6 (0.00%)  0
Syncope * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 1/6 (16.67%)  1
Hypoglycemia * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Hepatobiliary disorders                 
New diagnosis - hepatic steatosis * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Immune system disorders                 
Environmental allergies * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Infections and infestations                 
Upper respiratory symptoms * 1  1/19 (5.26%)  1 2/4 (50.00%)  2 1/4 (25.00%)  2 3/4 (75.00%)  3 3/7 (42.86%)  3 0/6 (0.00%)  0 2/8 (25.00%)  5 1/6 (16.67%)  1
Flu-like illness * 1  1/19 (5.26%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1 0/8 (0.00%)  0 1/6 (16.67%)  1
Infectious illness - not otherwise specified * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 1/6 (16.67%)  1
Cold sore * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Wound infection * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1 0/8 (0.00%)  0 0/6 (0.00%)  0
Dental infection * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 1/6 (16.67%)  1 0/8 (0.00%)  0 0/6 (0.00%)  0
Injury, poisoning and procedural complications                 
Pain associated with muscle biopsy * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Numbness following biopsy * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Lower extremity edema following biopsy * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Skin avulsion following biopsy * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Intravenous catheter infiltration * 1  0/19 (0.00%)  0 1/4 (25.00%)  2 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Ecchymosis at IV site * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Fall - not otherwise specified * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1 0/8 (0.00%)  0 1/6 (16.67%)  1
Musculoskeletal pain post fall * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 2/4 (50.00%)  2 0/4 (0.00%)  0 0/7 (0.00%)  0 2/6 (33.33%)  3 1/8 (12.50%)  1 3/6 (50.00%)  3
Fracture - pelvic * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  2 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Knee injury post fall * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Shoulder injury post fall * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Facial injury post fall * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 1/6 (16.67%)  1
Ankle injury post fall * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Dental injury * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Investigations                 
Ketones detected in urine * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Red blood cells in urine * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Elevated serum iron levels * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 2/4 (50.00%)  2 0/4 (0.00%)  0 0/7 (0.00%)  0 2/6 (33.33%)  2 0/8 (0.00%)  0 0/6 (0.00%)  0
Low serum iron levels * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 2/7 (28.57%)  2 0/6 (0.00%)  0 0/8 (0.00%)  0 1/6 (16.67%)  1
Elevated amylase * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Elevated serum ferritin * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Low serum ferritin * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Metabolism and nutrition disorders                 
Weight gain * 1 [1]  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Weight loss * 1 [1]  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 1/6 (16.67%)  1
Musculoskeletal and connective tissue disorders                 
Shoulder pain - not otherwise specified * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Knee pain - not otherwise specified * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Chest pain * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Neck pain - not otherwise specified * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1 0/8 (0.00%)  0 0/6 (0.00%)  0
Hip pain - not otherwise specified * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 1/6 (16.67%)  1 0/8 (0.00%)  0 0/6 (0.00%)  0
Nervous system disorders                 
Headache * 1  3/19 (15.79%)  3 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 2/8 (25.00%)  2 0/6 (0.00%)  0
New diagnosis - carpal tunnel syndrome * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Psychiatric disorders                 
Anxiety * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
New diagnosis - bipolar disorder * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
New diagnosis - personality disorder * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Renal and urinary disorders                 
Urinary tract infection * 1  2/19 (10.53%)  2 1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  2 0/6 (0.00%)  0
Urinary tract pain * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Nephrolithiasis * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Prostatic hypertrophy * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1 0/8 (0.00%)  0 0/6 (0.00%)  0
Hematuria * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 1/6 (16.67%)  1 1/8 (12.50%)  1 0/6 (0.00%)  0
Reproductive system and breast disorders                 
Vaginal yeast infection * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  2 0/6 (0.00%)  0
Skin and subcutaneous tissue disorders                 
Dermatitis * 1  1/19 (5.26%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  2 0/6 (0.00%)  0
Rash - not otherwise specified * 1  0/19 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Acne * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Cyst - not otherwise specified * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/7 (14.29%)  1 0/6 (0.00%)  0 0/8 (0.00%)  0 0/6 (0.00%)  0
Alopecia * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Pruritis * 1  0/19 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 0/7 (0.00%)  0 0/6 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
[1]
Greater than 5% of baseline weight
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Kathryn R. Wagner
Organization: Hugo W. Moser Research Institute at Kennedy Krieger Inc.
Phone: 443-923-9525
EMail: wagnerk@kennedykrieger.org
Layout table for additonal information
Responsible Party: Kathryn Wagner, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT02841267    
Other Study ID Numbers: WI203720
First Submitted: July 12, 2016
First Posted: July 22, 2016
Results First Submitted: January 30, 2020
Results First Posted: October 19, 2020
Last Update Posted: October 19, 2020