Essential Tremor
Conditions
Brief summary
To determine the relationship between drug plasma levels and safety, tolerability and efficacy in patients with essential tremors after dosing with Sodium oxybate
Interventions
DRUGSodium Oxybate
Dose 1
OTHERPlacebo
Dose 3
Sponsors
Jazz Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
50 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of moderate or severe classic essential tremor (bilateral, largely symmetric postural or kinetic tremor involving hands and forearms. * Willingness to abstain from ethanol and caffeine intake for at least 48 hours prior to Days 1 and 4 and on inpatient days
Exclusion criteria
* Subjects with a clinically significant unstable medical abnormality, chronic disease or history or presence of significant hepatic, renal, endocrine, cardiovascular, gastrointestinal, pulmonary, psychiatric, or metabolic disease or any other abnormality * Subjects who are on sodium-restricted diets * Subjects with a known history of sleep apnea
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Modified FTM(Fahn-Tolosa-Marin) Essentials Tremor Rating Scale, Sum of All Essential Rating Tremor Scales Including Voice Tremor | Hour 1 | The modified FTM sum of all essential rating tremor scales including voice tremor includes: the tremor rating taken for the left & right hands individually at rest, with posture (arms outstretched), with action (finger to nose). It also includes an evaluation of voice with scores for AAA & EEE sounds, an action evaluation of left & right hands pouring, bringing liquids to mouth, drawing large & small spirals. Scores for indiviuals items range from 0 (no tremor) to 4 (severe tremor). The sum ranges from 0 (no tremor) to 72 points (higher amplitude/more tremors). |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| All Study Participants All treated study participants | 20 |
| Total | 20 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Period 2 (Day 2) | Adverse Event | 1 | 0 |
Baseline characteristics
| Characteristic | All Study Participants |
|---|---|
| Age Continuous | 60.0 years STANDARD_DEVIATION 8.72 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 19 Participants |
| Region of Enrollment United States | 20 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 5 / 20 | 12 / 20 | 1 / 19 |
| serious Total, serious adverse events | 0 / 20 | 0 / 20 | 0 / 19 |
Outcome results
Primary
Modified FTM(Fahn-Tolosa-Marin) Essentials Tremor Rating Scale, Sum of All Essential Rating Tremor Scales Including Voice Tremor
The modified FTM sum of all essential rating tremor scales including voice tremor includes: the tremor rating taken for the left & right hands individually at rest, with posture (arms outstretched), with action (finger to nose). It also includes an evaluation of voice with scores for AAA & EEE sounds, an action evaluation of left & right hands pouring, bringing liquids to mouth, drawing large & small spirals. Scores for indiviuals items range from 0 (no tremor) to 4 (severe tremor). The sum ranges from 0 (no tremor) to 72 points (higher amplitude/more tremors).
Time frame: Hour 1
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Sodium Oxybate 1.5 Grams (A) | Modified FTM(Fahn-Tolosa-Marin) Essentials Tremor Rating Scale, Sum of All Essential Rating Tremor Scales Including Voice Tremor | 13.4 Points |
| Sodium Oxybate 3 Grams (B) | Modified FTM(Fahn-Tolosa-Marin) Essentials Tremor Rating Scale, Sum of All Essential Rating Tremor Scales Including Voice Tremor | 10.8 Points |
| Placebo (C) | Modified FTM(Fahn-Tolosa-Marin) Essentials Tremor Rating Scale, Sum of All Essential Rating Tremor Scales Including Voice Tremor | 13.0 Points |
p-value: 0.013ANOVA
p-value: 0.713ANOVA
p-value: 0.038ANOVA