A Study of Pitolisant in Patients With Prader-Willi Syndrome

Conditions

• Prader-Willi Syndrome

Eligibility Criteria

Sex

ALL

Age

6 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Pitolisant tablet — DRUG
  Pitolisant tablet
• Placebo tablet — OTHER
  Placebo tablet

Study Details

This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome. The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome. Secondary objectives include assessing the impact of pitolisant on: Irritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech

Key Dates

Start date

May 28, 2024

Status verified

Mar 2026

Primary completion

Jul 31, 2026

Completion

Jul 31, 2027

Study Design

Enrollment

134 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Double-Blind Treatment Period Pitolisant
  Pitolisant tablets administered once daily in the morning upon wakening
• Placebo Comparator: Double-Blind Treatment Period Placebo
  Matching placebo administered tablets once daily in the morning upon wakening
• Other: Open-Label Extension Period Pitolisant
  Pitolisant tablets administered once daily in the morning upon wakening

Primary Outcome Measure

Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score [ Time Frame: Baseline and end of the Double Blind Treatment Period (Day 77) ]

Central Contacts

• Ann Adee
  773-383-6258
  [email protected]
• Linnea Ryan
  [email protected]

Locations (21)

Find similar trials in Los Angeles, CA

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