Clinical trial NCT05581576: Pitolisant in Refractory Restless Legs Syndrome
Official title: Open Label Assessment of Pitolisant (Wakix) as Adjunct Therapy for Patients With Refractory Restless Legs Syndrome
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Restless Legs Syndrome
Keywords: Refractory
Lead sponsor: The Methodist Hospital Research Institute
Sponsor class: OTHER
Collaborators: name: Harmony Biosciences Management, Inc.
First posted: 2022-10-14
Enrollment: 18
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

Eligibility criteria: 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

Primary outcomes:
measure: International RLS Rating Scale (IRLS)
timeFrame: At week 8
Secondary outcomes:
measure: The Restless Legs Syndrome - 6 Scale (RLS-6)
timeFrame: Baseline, 4 weeks, 8 weeks, and 16 weeks
measure: Restless Legs Syndrome Quality of Life Questionnaire (RLSQoL)
timeFrame: Baseline, 4 weeks, 8 weeks, and 16 weeks
measure: Fatigue Severity Scale (FSS)
timeFrame: Baseline, 4 weeks, 8 weeks, and 16 weeks
measure: Hamilton Depression Scale (HDS)
timeFrame: Baseline, 4 weeks, 8 weeks, and 16 weeks
measure: Montreal Cognitive Assessment (MoCA)
timeFrame: Baseline, 4 weeks, 8 weeks, and 16 weeks
measure: Epworth Sleepiness Scale (ESS)
timeFrame: Baseline, 4 weeks, 8 weeks, and 16 weeks
measure: Clinical Global Impressions - Change
timeFrame: 1 week, 2 weeks, 4 weeks, 8 weeks, 11 weeks, 16 weeks, and 17 weeks
Other outcomes:
Locations:
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
Study officials:
affiliation: The Methodist Hospital Research Institute
name: William Ondo, MD
role: PRINCIPAL_INVESTIGATOR
References: