Trial Outcomes & Findings for Spironolactone Versus Prednisolone in DMD (NCT NCT03777319)

Last Updated: 2023-10-23

Results Overview

The determination of whether spironolactone has similar efficacy to glucocorticoids in improving muscle strength in steroid naïve DMD patients. This will be determined by measuring the time to complete a 100 meter timed test (100M).

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

2 participants

Primary outcome timeframe

6 months

Results posted on

2023-10-23

Participant Flow

Participant milestones

Participant milestones
Measure	Spironolactone An anticipated twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension. Spironolactone: Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.	Prednisolone An anticipated twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing per sites standard of care. The prednisolone will be provided will be provided as suspension. Prednisolone: Prednisolone will be prescribed for 6 months as the clinical standard of care.
Overall Study STARTED	1	1
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Spironolactone Versus Prednisolone in DMD

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Spironolactone n=1 Participants An anticipated twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension. Spironolactone: Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.	Prednisolone n=1 Participants An anticipated twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing per sites standard of care. The prednisolone will be provided will be provided as suspension. Prednisolone: Prednisolone will be prescribed for 6 months as the clinical standard of care.
Efficacy: Change in Time to Complete a 100 Meter Timed Test.	-0.6 sec	-5.3 sec

PRIMARY outcome

Timeframe: 6 months

Electrolytes (Sodium, Potassium, Cloride and Carbon dioxide, mmol/L) will be measured on a monthly basis following initiation of either spironolactone or prednisolone.

Outcome measures

Outcome measures
Measure	Spironolactone n=1 Participants An anticipated twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension. Spironolactone: Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.	Prednisolone n=1 Participants An anticipated twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing per sites standard of care. The prednisolone will be provided will be provided as suspension. Prednisolone: Prednisolone will be prescribed for 6 months as the clinical standard of care.
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Month 4	103 mmol/L	105 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Month 5	103 mmol/L	106 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Month 6	101 mmol/L	105 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Baseline	29 mmol/L	22 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Month 1	22 mmol/L	24 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Month 2	25 mmol/L	24 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Month 3	27 mmol/L	24 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Month 4	28 mmol/L	25 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Month 5	28 mmol/L	26 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. CO2-Month 6	26 mmol/L	26 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Baseline	142 mmol/L	140 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Month 1	142 mmol/L	140 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Month 2	141 mmol/L	139 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Month 3	142 mmol/L	141 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Month 4	139 mmol/L	139 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Month 5	139 mmol/L	139 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Sodium-Month 6	140 mmol/L	143 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Baseline	4.5 mmol/L	3.8 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Month 1	4.7 mmol/L	4 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Month 2	4.2 mmol/L	4.5 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Month 3	4.1 mmol/L	3.9 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Month 4	4.5 mmol/L	4.6 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Month 5	4.5 mmol/L	4.2 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Potassium-Month 6	4.3 mmol/L	3.9 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Baseline	103 mmol/L	105 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Month 1	109 mmol/L	105 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Month 2	107 mmol/L	104 mmol/L
Safety Will be Monitored Through Regular Review of Electrolytes. Chloride-Month 3	103 mmol/L	105 mmol/L

SECONDARY outcome

Timeframe: 6 months

Secondary outcome measures will be Dynamometry score, which is a summation of maximum voluntary isometric contraction test values for knee flexion, knee extension, elbow flexion, and elbow extension

Outcome measures

Outcome measures
Measure	Spironolactone n=1 Participants An anticipated twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension. Spironolactone: Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.	Prednisolone n=1 Participants An anticipated twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing per sites standard of care. The prednisolone will be provided will be provided as suspension. Prednisolone: Prednisolone will be prescribed for 6 months as the clinical standard of care.
Efficacy: Dynamometry Score Elbow Flexion (Left)-Month 6	3.5 kg	3.4 kg
Efficacy: Dynamometry Score Elbow Flexion (Right)-Baseline	0 kg	3.6 kg
Efficacy: Dynamometry Score Elbow Flexion (Left)-Baseline	0 kg	4.1 kg
Efficacy: Dynamometry Score Elbow Extension (Right)-Baseline	0 kg	5.3 kg
Efficacy: Dynamometry Score Elbow Extension (Left)-Baseline	0 kg	4.1 kg
Efficacy: Dynamometry Score Knee Flexion (Right)-Baseline	4.1 kg	3.3 kg
Efficacy: Dynamometry Score Knee Flexion (Left)-Baseline	2.8 kg	3.4 kg
Efficacy: Dynamometry Score Knee Extension (Right)-Baseline	3.8 kg	4.8 kg
Efficacy: Dynamometry Score Knee Extension (Left)-Baseline	5.9 kg	5.2 kg
Efficacy: Dynamometry Score Elbow Flexion (Right)-Month 6	3.1 kg	2.9 kg
Efficacy: Dynamometry Score Elbow Extension (Right)-Month 6	2.4 kg	4.3 kg
Efficacy: Dynamometry Score Elbow Extension (Left)-Month 6	2.5 kg	3.8 kg
Efficacy: Dynamometry Score Knee Flexion (Right)-Month 6	4.3 kg	4.1 kg
Efficacy: Dynamometry Score Knee Flexion (Left)-Month 6	4.1 kg	3.9 kg
Efficacy: Dynamometry Score Knee Extension (Right)-Month 6	7.2 kg	6 kg
Efficacy: Dynamometry Score Knee Extension (Left)-Month 6	8.3 kg	5.1 kg

Adverse Events

Spironolactone

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Prednisolone

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Spironolactone n=1 participants at risk Twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension. Spironolactone: Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.	Prednisolone n=1 participants at risk Twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing per sites standard of care. The prednisolone will be provided will be provided as suspension. Prednisolone: Prednisolone will be prescribed for 6 months as the clinical standard of care.
Gastrointestinal disorders Viral Illness	100.0% 1/1 • Number of events 2 • Data was collected for each participant over a 7 month duration.	0.00% 0/1 • Data was collected for each participant over a 7 month duration.
Gastrointestinal disorders Loose Stool	100.0% 1/1 • Number of events 1 • Data was collected for each participant over a 7 month duration.	0.00% 0/1 • Data was collected for each participant over a 7 month duration.
Musculoskeletal and connective tissue disorders Muscle Cramps	0.00% 0/1 • Data was collected for each participant over a 7 month duration.	100.0% 1/1 • Number of events 3 • Data was collected for each participant over a 7 month duration.
Gastrointestinal disorders Dental Cavity	0.00% 0/1 • Data was collected for each participant over a 7 month duration.	100.0% 1/1 • Number of events 1 • Data was collected for each participant over a 7 month duration.

Additional Information

Dr. Kevin Flanigan

The Abigal Wexner Research Institute at Nationwide Children's Hospital

Phone: 614-722-5615

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Spironolactone n=1 Participants Twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension. Spironolactone: Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.	Prednisolone n=1 Participants Twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing per sites standard of care. The prednisolone will be provided will be provided as suspension. Prednisolone: Prednisolone will be prescribed for 6 months as the clinical standard of care.	Total n=2 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants