Duchenne Muscular Dystrophy
Conditions
Brief summary
This is an interventional study on Duchenne muscular dystrophy patients who will be receiving sedation for a muscle biopsy as part of another study.
Interventions
DRUGKetamine
1 mg/kg IV, additional doses of 0.5 mg/kg as needed
DRUGDexmedetomidine
0.5 mcg/kg/hr IV
Sponsors
Nationwide Children's Hospital
Study design
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
* Patients undergoing a muscle biopsy for IRB11-00203.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to Sedation Score of 3-4 | Immediately prior to incision | The depth of sedation was judged using the University of Michigan Sedation Scale (UMSS). The score ranges from zero, awake and alert, to four, unarousable. A score of three, deeply sedated, or more was considered to be an appropriate level of sedation for the procedure. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Heart Rate Change After Dexmedetomidine Loading Dose | Baseline to immediately post dexmedetomidine infusion. | Difference in heart rate from baseline to immediately following infusion of dexmedetomidine loading dose. |
| Mean Arterial Pressure (MAP) Change After Dexmedetomidine Loading Dose | Baseline to immediately post dexmedetomidine infusion. | Change in MAP from baseline measurement to immediately post dexmedetomidine infusion measured via blood pressure cuff. |
| Oxygen Saturation Change After Dexmedetomidine Loading Dose | Baseline to immediately post dexmedetomidine infusion. | Change in oxygen saturation from baseline measurement to immediately post dexmedetomidine infusion. |
| EtCO2 Change After Dexmedetomidine Loading Dose | Baseline to immediately post dexmedetomidine infusion. | Change in end-tidal carbon dioxide from baseline measurement to immediately post dexmedetomidine infusion. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Dexmedetomidine 1 mcg/kg Patients receive a loading dose of dexmedetomidine 1 mcg/kg followed by a continuous infusion of 1 mcg/kg/hr.
Ketamine: 1 mg/kg IV, additional doses of 0.5 mg/kg as needed
Dexmedetomidine: 0.5 mcg/kg/hr IV | 24 |
| Dexmedetomidine 0.5 mcg/kg Patients receive a loading dose of dexmedetomidine 0.5 mcg/kg followed by a continuous infusion of 0.5 mcg/kg/hr.
Ketamine: 1 mg/kg IV, additional doses of 0.5 mg/kg as needed
Dexmedetomidine: 0.5 mcg/kg/hr IV | 29 |
| Total | 53 |
Baseline characteristics
| Characteristic | Dexmedetomidine 1 mcg/kg | Dexmedetomidine 0.5 mcg/kg | Total |
|---|---|---|---|
| Age, Continuous | 9.7 years STANDARD_DEVIATION 1.4 | 8.9 years STANDARD_DEVIATION 1.9 | 9.4 years STANDARD_DEVIATION 1.7 |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 24 Participants | 29 Participants | 53 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 24 | 0 / 29 |
| serious Total, serious adverse events | 0 / 24 | 0 / 29 |
Outcome results
Primary
Time to Sedation Score of 3-4
The depth of sedation was judged using the University of Michigan Sedation Scale (UMSS). The score ranges from zero, awake and alert, to four, unarousable. A score of three, deeply sedated, or more was considered to be an appropriate level of sedation for the procedure.
Time frame: Immediately prior to incision
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dexmedetomidine 1 mcg/kg | Time to Sedation Score of 3-4 | 3.7 minutes | Standard Deviation 2.3 |
| Dexmedetomidine 0.5 mcg/kg | Time to Sedation Score of 3-4 | 2.8 minutes | Standard Deviation 1.6 |
Secondary
EtCO2 Change After Dexmedetomidine Loading Dose
Change in end-tidal carbon dioxide from baseline measurement to immediately post dexmedetomidine infusion.
Time frame: Baseline to immediately post dexmedetomidine infusion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dexmedetomidine 1 mcg/kg | EtCO2 Change After Dexmedetomidine Loading Dose | 1 mmHg | Standard Deviation 5 |
| Dexmedetomidine 0.5 mcg/kg | EtCO2 Change After Dexmedetomidine Loading Dose | 2 mmHg | Standard Deviation 6 |
Secondary
Heart Rate Change After Dexmedetomidine Loading Dose
Difference in heart rate from baseline to immediately following infusion of dexmedetomidine loading dose.
Time frame: Baseline to immediately post dexmedetomidine infusion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dexmedetomidine 1 mcg/kg | Heart Rate Change After Dexmedetomidine Loading Dose | -13 BPM | Standard Deviation 13 |
| Dexmedetomidine 0.5 mcg/kg | Heart Rate Change After Dexmedetomidine Loading Dose | -5 BPM | Standard Deviation 10 |
Secondary
Mean Arterial Pressure (MAP) Change After Dexmedetomidine Loading Dose
Change in MAP from baseline measurement to immediately post dexmedetomidine infusion measured via blood pressure cuff.
Time frame: Baseline to immediately post dexmedetomidine infusion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dexmedetomidine 1 mcg/kg | Mean Arterial Pressure (MAP) Change After Dexmedetomidine Loading Dose | 9 mmHg | Standard Deviation 10 |
| Dexmedetomidine 0.5 mcg/kg | Mean Arterial Pressure (MAP) Change After Dexmedetomidine Loading Dose | 5 mmHg | Standard Deviation 8 |
Secondary
Oxygen Saturation Change After Dexmedetomidine Loading Dose
Change in oxygen saturation from baseline measurement to immediately post dexmedetomidine infusion.
Time frame: Baseline to immediately post dexmedetomidine infusion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Dexmedetomidine 1 mcg/kg | Oxygen Saturation Change After Dexmedetomidine Loading Dose | -0.3 percentage of oxygen | Standard Deviation 1.2 |
| Dexmedetomidine 0.5 mcg/kg | Oxygen Saturation Change After Dexmedetomidine Loading Dose | 0.4 percentage of oxygen | Standard Deviation 0.7 |