Trial Outcomes & Findings for Weekly Steroids in Muscular Dystrophy (NCT NCT04054375)

NCT ID: NCT04054375

Last Updated: 2023-09-21

Results Overview

mg/dL, 0-unlimited, higher score indicates worse outcome

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 20 participants
• Primary outcome timeframe: Baseline and 6 months (Final Visit)
• Results posted on: 2023-09-21

Participant Flow

Patient Diagnosis Subtype: 19 with Limb-Girdle Muscular Dystrophy 1 with Becker Muscular Dystrophy

Participant milestones

Measure	Weekly Steroid Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Overall Study STARTED	20
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Weekly Steroids in Muscular Dystrophy

Baseline characteristics by cohort

Measure	Weekly Steroid n=20 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	35 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 6 months (Final Visit)

mg/dL, 0-unlimited, higher score indicates worse outcome

Outcome measures

Measure	Weekly Steroid n=16 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Fasting Glucose Baseline	93 mg/dL Standard Deviation 2
Fasting Glucose End	102 mg/dL Standard Deviation 4

PRIMARY outcome

Timeframe: Baseline and 6 months (Final Visit)

% , 0-100, higher score indicates worse outcome

Outcome measures

Measure	Weekly Steroid n=17 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
HbgA1c Baseline	5.2 % A1c Standard Deviation 0.08
HbgA1c End	5.3 % A1c Standard Deviation 0.09

PRIMARY outcome

Timeframe: Baseline and 6 months (Final Visit)

cholesterol levels - mg/dL, higher levels indicate worse outcomes

Outcome measures

Measure	Weekly Steroid n=17 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Fasting Lipid Profile Baseline	182 mg/dL Standard Deviation 10
Fasting Lipid Profile End	185 mg/dL Standard Deviation 9

PRIMARY outcome

Timeframe: Baseline and 6 months (Final Visit)

units/L, 0-unlimited, higher scores indicate worse outcome

Outcome measures

Measure	Weekly Steroid n=17 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Creatine Kinase Baseline	1574 U/L Standard Deviation 269
Creatine Kinase End	1047 U/L Standard Deviation 171

PRIMARY outcome

Timeframe: Baseline, 6 months

Force Vital Capacity (% of predicted value), decrease in FVC indicates declining respiratory function.

Outcome measures

Measure	Weekly Steroid n=11 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Respiratory Changes Baseline	80 % Expected Standard Deviation 8
Respiratory Changes End	79 % Expected Standard Deviation 8

SECONDARY outcome

Timeframe: Baseline, Month 6

Northstar Assessment for Dysferlinopathy

• score out of 58, range from 0 to 58, higher score indicates greater functional ability.

Outcome measures

Measure	Weekly Steroid n=16 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Functional Assessments - NSAD Change baseline	18.4 score on a scale Standard Deviation 17
Functional Assessments - NSAD Change 6 months	18.6 score on a scale Standard Deviation 17

SECONDARY outcome

Timeframe: Baseline, Month 6

number of meters walked in 6 minute period. Higher values indicate more motor function.

Outcome measures

Measure	Weekly Steroid n=8 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
6 Minute Walk Test 6 months	410 meters Standard Error 40
6 Minute Walk Test Baseline	386 meters Standard Error 37

SECONDARY outcome

Timeframe: Baseline, Month 6

time in seconds to walk/run 10 meters , less time to run indicates greater motor function

Outcome measures

Measure	Weekly Steroid n=9 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
10 Meter Run Timed Baseline	7.32 seconds Standard Error 0.92
10 Meter Run Timed 6 months	6.67 seconds Standard Error 0.77

SECONDARY outcome

Timeframe: Baseline, Month 6

upper extremity assessment, scoring between 1- 6, lower score indicates more upper extremity function

Outcome measures

Measure	Weekly Steroid n=18 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Brooke Scale Score baseline	3 scores on a scale Standard Error 1
Brooke Scale Score 6 months	3 scores on a scale Standard Error 1

SECONDARY outcome

Timeframe: Baseline, Month 6

Lower extremity assessment, score from 1-10, lower score indicates more function.

Outcome measures

Measure	Weekly Steroid n=20 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Vignos Scale Score baseline	5 scores on a scale Standard Error 1
Vignos Scale Score 6 months	5 scores on a scale Standard Error 1

SECONDARY outcome

Timeframe: Baseline, 6 months

Population: MRI of the muscles was collected on all baseline patients (20 participants). Due to clinical heterogeneity, different muscles had to be imaged for each patient. Five participants didn't get follow-up MRI scans due to COVID-19 pandemic.

MRI of leg muscles to measure changes in muscle fat percentage. The data point was collected by taking fat percentage at 6 months minus fat percentage at baseline with the following equation: (([final fat percentage - initial fat percentage]/initial fat percentage) * 100%)). All participants were included, both ambulatory and nonambulatory, with all genetic subtypes included. Five participants didn't have an MRI scan at 6 months and therefore were not included. Muscles imaged were analyzed for muscle fat changes from baseline to 6 months. Data is limited in interpretation due to various muscle groups in both ambulatory and non-ambulatory patients.

Outcome measures

Measure

Weekly Steroid n=15 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM

Muscle Imaging

-14 percent of change from baseline Standard Deviation 13

SECONDARY outcome

Timeframe: Baseline, 6 months

whole dexa body scan to assess bone density with Z scores (more negative z score indicates increased risk for fractures).

Z-score of 0 represents the population mean, and is the average bone density. Positive scores indicate greater bone density and negative scores indicate decreased bone density, which could be clinically correlated with osteoporosis.

Outcome measures

Measure	Weekly Steroid n=15 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Bone Density baseline	-1.64 z-score Standard Error 0.33
Bone Density 6 months	-1.65 z-score Standard Error 0.34

SECONDARY outcome

Timeframe: Baseline, 6 months

whole body dexa scans to assess lean mass % (0- 100 %). Increase lean mass % is the desired outcome.

Outcome measures

Measure	Weekly Steroid n=15 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Lean Mass % baseline	37.5 percentage Standard Error 2.5
Lean Mass % 6 months	38.1 percentage Standard Error 2.6

SECONDARY outcome

Timeframe: Baseline and 6 months

Grip strength of the total force (Newtons) in both hands.

Participants attempted 3 trials in the right hand that was then averaged to create a right-hand average force score.

Then, the participants attempted 3 trials in the left hand that was then averaged to create a left-hand average force score.

The right-hand average force score was added to the left-hand average force score to create a total grip strength score.

Outcome measures

Measure	Weekly Steroid n=17 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Functional Assessments - Upper Limb Strength Baseline	39 Force (Newtons) Standard Deviation 25
Functional Assessments - Upper Limb Strength 6 months	41 Force (Newtons) Standard Deviation 27

SECONDARY outcome

Timeframe: baseline, 6 months

Population: Manual muscle testing scores are from 0 to 5, with 5 being the strongest and 0 no muscle movement at all.

Manual motor testing of the right knee flexion muscle group.

Outcome measures

Measure	Weekly Steroid n=16 Participants Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM Prednisone: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Muscle Strength Test Baseline	3 Units on scale Interval 1.0 to 5.0
Muscle Strength Test 6 months	3 Units on scale Interval 1.0 to 5.0

Adverse Events

Weekly Steroid

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Senda Ajroud-Driss

Northwestern University School of Medicine

Phone: (312) 944-0063

Email: s-ajroud@northwestern.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place