Pitolisant in Refractory Restless Legs Syndrome

October 13, 2022 updated by: William Ondo, MD

Open Label Assessment of Pitolisant (Wakix) as Adjunct Therapy for Patients With Refractory Restless Legs Syndrome

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

18

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

17 years to 76 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
International RLS Rating Scale (IRLS)
Time Frame: At week 8
This is a subjective scale to measure the severity of RLS within the past week.
At week 8

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Restless Legs Syndrome - 6 Scale (RLS-6)
Time Frame: Baseline, 4 weeks, 8 weeks, and 16 weeks
This scale measures the severity of daytime versus nighttime RLS symptoms.
Baseline, 4 weeks, 8 weeks, and 16 weeks
Restless Legs Syndrome Quality of Life Questionnaire (RLSQoL)
Time Frame: Baseline, 4 weeks, 8 weeks, and 16 weeks
This scale assess the quality of life in RLS patients.
Baseline, 4 weeks, 8 weeks, and 16 weeks
Fatigue Severity Scale (FSS)
Time Frame: Baseline, 4 weeks, 8 weeks, and 16 weeks
This scale evaluates the impact of fatigue patients are experiencing.
Baseline, 4 weeks, 8 weeks, and 16 weeks
Hamilton Depression Scale (HDS)
Time Frame: Baseline, 4 weeks, 8 weeks, and 16 weeks
This scale assess symptoms of depression.
Baseline, 4 weeks, 8 weeks, and 16 weeks
Montreal Cognitive Assessment (MoCA)
Time Frame: Baseline, 4 weeks, 8 weeks, and 16 weeks
This assessment detects mild cognitive impairment.
Baseline, 4 weeks, 8 weeks, and 16 weeks
Epworth Sleepiness Scale (ESS)
Time Frame: Baseline, 4 weeks, 8 weeks, and 16 weeks
This is a subjective scale that measures a patient's sleepiness.
Baseline, 4 weeks, 8 weeks, and 16 weeks
Clinical Global Impressions - Change
Time Frame: 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.
1 week, 2 weeks, 4 weeks, 8 weeks, 11 weeks, 16 weeks, and 17 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

William Ondo, MD

Collaborators

Harmony Biosciences, LLC

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 20, 2022

Primary Completion (Anticipated)

May 1, 2023

Study Completion (Anticipated)

May 1, 2023

Study Registration Dates

First Submitted

September 1, 2022

First Submitted That Met QC Criteria

October 13, 2022

First Posted (Actual)

October 14, 2022

Study Record Updates

Last Update Posted (Actual)

October 14, 2022

Last Update Submitted That Met QC Criteria

October 13, 2022

Last Verified

October 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PRO00033508

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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