Pitolisant in Refractory Restless Legs Syndrome

William Ondo, MD

Status and phase: Enrolling; Phase 4

Conditions: Restless Legs Syndrome

Treatments: Drug: Pitolisant

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05581576

PRO00033508

Details and patient eligibility

About

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.

Full 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).

Enrollment: 18 estimated patients

Sex: All

Ages: 21 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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18 participants in 1 patient group

Pitolisant (Wakix)

Experimental group

Description:

Pitolisant will be titrated weekly until maximum dosage of 35.6 mg. Titration is dependent on subjects response.

Treatment:

Drug: Pitolisant