Pitolisant in Refractory Restless Legs Syndrome

• Sponsor: William Ondo, MD (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT05581576
• Collaborator: Harmony Biosciences, LLC (Industry)
• Enrollment: 18
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 7.3
• Patients Per Site Per Month: 2.5

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Restless Legs Syndrome	Drug: Pitolisant	Phase 4

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).

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 18 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Open Label Assessment of Pitolisant (Wakix) as Adjunct Therapy for Patients With Refractory Restless Legs Syndrome
• Actual Study Start Date: Sep 20, 2022
• Anticipated Primary Completion Date: May 1, 2023
• Anticipated Study Completion Date: May 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Pitolisant (Wakix) Pitolisant will be titrated weekly until maximum dosage of 35.6 mg. Titration is dependent on subjects response.	Drug: Pitolisant; Pitolisant initial dose 8.9 mg, titrated depending on symptomatic response.; Other Names: Wakix

Outcome Measures

Primary Outcome Measures

1. International RLS Rating Scale (IRLS) [At week 8]

   This is a subjective scale to measure the severity of RLS within the past week.

Secondary Outcome Measures

1. 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.

2. 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.

3. Fatigue Severity Scale (FSS) [Baseline, 4 weeks, 8 weeks, and 16 weeks]

   This scale evaluates the impact of fatigue patients are experiencing.

4. Hamilton Depression Scale (HDS) [Baseline, 4 weeks, 8 weeks, and 16 weeks]

   This scale assess symptoms of depression.

5. Montreal Cognitive Assessment (MoCA) [Baseline, 4 weeks, 8 weeks, and 16 weeks]

   This assessment detects mild cognitive impairment.

6. Epworth Sleepiness Scale (ESS) [Baseline, 4 weeks, 8 weeks, and 16 weeks]

   This is a subjective scale that measures a patient's sleepiness.

7. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 21 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Houston Methodist Hospital	Houston	Texas	United States	77030

Sponsors and Collaborators

• William Ondo, MD
• Harmony Biosciences, LLC

Investigators

• Principal Investigator: William Ondo, MD, The Methodist Hospital Research Institute

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: William Ondo, MD, Principal Investigator, The Methodist Hospital Research Institute
• ClinicalTrials.gov Identifier: NCT05581576
• Other Study ID Numbers: PRO00033508
• First Posted: Oct 14, 2022
• Last Update Posted: Oct 14, 2022
• Last Verified: Oct 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes
• Keywords provided by William Ondo, MD, Principal Investigator, The Methodist Hospital Research Institute: Refractory
• Additional relevant MeSH terms: Psychomotor Agitation; Restless Legs Syndrome; Syndrome

Study Results

No Results Posted as of Oct 14, 2022