Trial Outcomes & Findings for CoQ10 and Prednisone in Non-Ambulatory DMD (NCT NCT00308113)

Results Overview

Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

3 participants

Primary outcome timeframe

12 months

Results posted on

2013-11-08

Participant Flow

Recruitment started in April 2007 at two participating centers of the Cooperative International Neuromuscular Research Group (CINRG): University of Pittsburgh and University of Puerto Rico. Enrollment closed in December 2008 by the CINRG Data and Safety Monitoring Board (DSMB) due to changes in standard of care.

Participant milestones

Participant milestones
Measure	Coenzyme Q10 and Prednisone CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL. Prednisone : Prednisone 0/75 mg/kg/day.	Enhanced Standard of Care Enhanced standard of care.
Overall Study STARTED	1	2
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Coenzyme Q10 Alone CoenzymeQ10 taken once a day each morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL.	Prednisone Alone Prednisone taken once a day each morning by mouth Prednisone : Prednisone 0/75 mg/kg/day.	Coenzyme Q10 and Prednisone CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL. Prednisone : Prednisone 0/75 mg/kg/day.	Enhanced Standard of Care Enhanced standard of care.
Overall Study Protocol closure	0	0	0	1

Baseline Characteristics

CoQ10 and Prednisone in Non-Ambulatory DMD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Coenzyme Q10 Alone CoenzymeQ10 taken once a day each morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL.	Prednisone Alone Prednisone taken once a day each morning by mouth Prednisone : Prednisone 0/75 mg/kg/day.	Coenzyme Q10 and Prednisone n=1 Participants CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL. Prednisone : Prednisone 0/75 mg/kg/day.	Enhanced Standard of Care n=2 Participants Enhanced standard of care.	Total n=3 Participants Total of all reporting groups
Age, Customized 10-18 years	—	—	1 participants n=5 Participants	2 participants n=4 Participants	3 participants n=21 Participants
Gender Female	—	—	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=21 Participants
Gender Male	—	—	1 participants n=5 Participants	2 participants n=4 Participants	3 participants n=21 Participants

PRIMARY outcome

Timeframe: 12 months

Population: No analysis was performed as only 1 out of 3 participants completed all echocardiogram measurements before the protocol was closed.

Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 12 months

Population: No analysis was performed as only 1 out of 3 participants completed all the pulmonary function measurements before the protocol was closed.

Comparing change from baseline levels in pulmonary function (FEV1 and FVC) in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: No analysis was performed as the study was closed with N=3 out of 120 and side effects profile between groups could not be analyzed.

To compare side effect profiles of the three regimens to the enhanced standard of care group, to include height, weight, weight/height ratio, body mass index, cataract formation, blood glucose, blood pressure, and behavioral changes.

Outcome measures

Outcome data not reported

Adverse Events

Coenzyme Q10 Alone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Prednisone Alone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Coenzyme Q10 and Prednisone

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Enhanced Standard of Care

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Coenzyme Q10 Alone CoenzymeQ10 taken once a day each morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL.	Prednisone Alone Prednisone taken once a day each morning by mouth Prednisone : Prednisone 0/75 mg/kg/day.	Coenzyme Q10 and Prednisone n=1 participants at risk CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL. Prednisone : Prednisone 0/75 mg/kg/day.	Enhanced Standard of Care n=2 participants at risk Enhanced standard of care.
Surgical and medical procedures Tonsilectomy	— 0/0 • 12 months	— 0/0 • 12 months	100.0% 1/1 • 12 months	0.00% 0/2 • 12 months

Other adverse events

Other adverse events
Measure	Coenzyme Q10 Alone CoenzymeQ10 taken once a day each morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL.	Prednisone Alone Prednisone taken once a day each morning by mouth Prednisone : Prednisone 0/75 mg/kg/day.	Coenzyme Q10 and Prednisone n=1 participants at risk CoenzymeQ10 and prednisone each taken once a day in the morning by mouth. Coenzyme Q10 : serum levels of greater or equal to 2.5 micrograms/mL. Prednisone : Prednisone 0/75 mg/kg/day.	Enhanced Standard of Care n=2 participants at risk Enhanced standard of care.
Blood and lymphatic system disorders Limb edema	— 0/0 • 12 months	— 0/0 • 12 months	100.0% 1/1 • 12 months	0.00% 0/2 • 12 months
Cardiac disorders Cardiac arrhythmia	— 0/0 • 12 months	— 0/0 • 12 months	100.0% 1/1 • 12 months	0.00% 0/2 • 12 months
Infections and infestations Upper airway infection	— 0/0 • 12 months	— 0/0 • 12 months	100.0% 1/1 • 12 months	0.00% 0/2 • 12 months
General disorders Mood alteration and agitation	— 0/0 • 12 months	— 0/0 • 12 months	100.0% 1/1 • 12 months	0.00% 0/2 • 12 months
Gastrointestinal disorders Gastrointestinal Virus	— 0/0 • 12 months	— 0/0 • 12 months	0.00% 0/1 • 12 months	50.0% 1/2 • 12 months

Additional Information

Dr. Paula R. Clemens

University of Pittsburgh

Phone: 412-648-9762

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place