Restless Legs Syndrome
Conditions
Keywords
Refractory
Brief summary
This is an open-label trial in Restless Legs Syndrome (RLS) patients inadequately treated with standard therapy, defined by an IRLS score greater than 15. Investigators hypothesize that the study drug, Pitolisant (Wakix), may improve RLS symptoms.
Detailed description
This is an open-label trial in RLS patients who have been inadequately treated with standard therapies, defined by an IRLS \> 15. This trial consists of four study center visits (weeks 0, 4, 8, and 16) and four telephone visits (weeks 1, 3, 11, and a post safety phone call at week 17). Subjects will titrate pitolisant for three weeks, starting with a dose of 8.9 mg at week 1, then increase to 17.8 mg at week 2, and a final increase to maximum dosage of 35.6 mg at week 3. If subjects are unable to tolerate the highest dose, they may reduce their dose to 17.8 mg. Titration of the drug will be monitored via telephone visits. During weeks 0-8, subjects will continue to take their current RLS medications. From week 8-16, subjects may either reduce or stop their other RLS medications as mutually agreed upon with investigator. Several questionnaire assessments will be conducted throughout the study to monitor the subjects. Subjects will complete the International RLS Rating Scale (IRLS), Restless Legs Syndrome - 6 Scale (RLS-6), RLS Quality of Life Questionnaire (RLSQoL), Fatigue Severity Scale (FSS), Hamilton Depression Scale (HDS), Montreal Cognitive Assessment (MoCA), Epworth Sleepiness Scale (ESS), Clinical Global Impressions (CGI), and Clinical Global Impressions-Change (CGI-C).
Interventions
DRUGPitolisant
Pitolisant initial dose 8.9 mg, titrated depending on symptomatic response.
Sponsors
Harmony Biosciences Management, Inc.
William Ondo, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* RLS diagnosed by standard criteria with an IRLS \> 15 while taking at least 1 RLS medication * Stable RLS medications for at least 2 weeks prior to study entry
Exclusion criteria
* MoCA \< 24 * Concurrent untreated sleep disorders, not felt to be stable * Subjects with any significant, unstable cardiovascular, liver, lung, renal, psychiatric, or neurological diseases (not including RLS) * Intravenous iron within 4 weeks of study entry * Breast feeding or pregnancy determined by urine pregnancy test in subjects where pregnancy is possible (pre-menopausal, sexually active women) * Subjects with previous allergic reaction or lack of tolerability to Pitolisant
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| International RLS Rating Scale (IRLS) | At week 8 | This is a subjective scale to measure the severity of RLS within the past week. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Restless Legs Syndrome Quality of Life Questionnaire (RLSQoL) | Baseline, 4 weeks, 8 weeks, and 16 weeks | This scale assess the quality of life in RLS patients. |
| Fatigue Severity Scale (FSS) | Baseline, 4 weeks, 8 weeks, and 16 weeks | This scale evaluates the impact of fatigue patients are experiencing. |
| Hamilton Depression Scale (HDS) | Baseline, 4 weeks, 8 weeks, and 16 weeks | This scale assess symptoms of depression. |
| The Restless Legs Syndrome - 6 Scale (RLS-6) | Baseline, 4 weeks, 8 weeks, and 16 weeks | This scale measures the severity of daytime versus nighttime RLS symptoms. |
| Epworth Sleepiness Scale (ESS) | Baseline, 4 weeks, 8 weeks, and 16 weeks | This is a subjective scale that measures a patient's sleepiness. |
| Clinical Global Impressions - Change | 1 week, 2 weeks, 4 weeks, 8 weeks, 11 weeks, 16 weeks, and 17 weeks | This scale assess how much a patients illness has improved or worsened when compared to a baseline state from the beginning of the intervention. |
| Montreal Cognitive Assessment (MoCA) | Baseline, 4 weeks, 8 weeks, and 16 weeks | This assessment detects mild cognitive impairment. |
Countries
United States
Contacts
Primary ContactWilliam Ondo, MD
Backup ContactShivani M Desai, BS
Outcome results
None listed