Evidence map›Paper›PMID 34822113›Full record

Trial reportCNS drugs2021

Time to Onset of Response to Pitolisant for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Patients With Narcolepsy: An Analysis of Randomized, Placebo-Controlled Trials.

Nathaniel F Watson, Craig W Davis, Donna Zarycranski, Ben Vaughn, Jeffrey M Dayno, Yves Dauvilliers, Jean-Charles Schwartz

2 registry-linked trialsOpen access · hybridAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in CNS drugs, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
0.5field-weighted citation impact, top 39% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01067222 phase3completednot on this map

Prospective, Randomized, Double-blind Study, Placebo-controlled, Parallel-group, Multi-center Trial Assessing the Effects of BF2.649 in Treatment of Excessive Daytime Sleepiness in Narcolepsy

TypeinterventionalSponsorBioprojetRan2009 to 2010Enrolled110ConditionsNarcolepsy, Excessive Daytime Sleepiness, Cataplexy, Sleep DisordersArmsBF2.649, Modafinil, Placebo
NCT01800045 phase3completednot on this map

A Randomized, Double Blind Study Comparing Pitolisant (BF2.649) to Placebo in Two Parallel Groups on the Weekly Frequency of Cataplexy Attacks and Excessive Daytime Sleepiness in Narcoleptic Patients With Cataplexy.

TypeinterventionalSponsorBioprojetRan2013 to 2015Enrolled103ConditionsNarcolepsy With Cataplexy, Excessive Daytime SleepinessArmsPitolisant, Placebo
3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. Trial
  2. Histamine HInflammation research : official journal of the European Histamine Research Society ... [et al.] · 2025
    Review
  3. Review
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 3 institutions in 2 countries.

Nathaniel F WatsonDepartment of Neurology, University of Washington School of Medicine, Seattle, WA, USA. nwatson@uw.edu.
Craig W DavisHarmony Biosciences, LLC, Plymouth Meeting, PA, USA.
Donna ZarycranskiHarmony Biosciences, LLC, Plymouth Meeting, PA, USA.
Ben VaughnRho, Durham, NC, USA.
Jeffrey M DaynoHarmony Biosciences, LLC, Plymouth Meeting, PA, USA.
Yves DauvilliersNational Reference Center for Narcolepsy, Montpellier, France.
Jean-Charles SchwartzBioprojet Pharma, Paris, France.
Bioprojet (France) · FRInserm · FRUniversity of Washington · US

Funding

Bioprojet Pharma StudiesHarmony Biosciences LLC Technical editorial and medical writing assistance support
6 · The paper itself

Abstract

backgroundPitolisant is approved in the USA and Europe for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy.

objectiveAnalyses evaluated the time to onset of clinical response during treatment with pitolisant.

methodsData were obtained from two randomized, double-blind, 7-week or 8-week, placebo-controlled studies (HARMONY 1, HARMONY CTP). Study medication was individually titrated to a maximum dose of pitolisant 35.6 mg/day and then remained stable. Efficacy assessments included the Epworth Sleepiness Scale and weekly rate of cataplexy (calculated from patient diaries). Onset of clinical response was defined as the first timepoint at which there was statistical separation between pitolisant and placebo.

resultsThe analysis included 61 patients in HARMONY 1 (pitolisant, n = 31; placebo, n = 30) and 105 patients in HARMONY CTP (pitolisant, n = 54; placebo, n = 51). Onset of clinical response began at week 2 (HARMONY 1) or week 3 (HARMONY CTP) for the mean change in Epworth Sleepiness Scale score, and week 2 (HARMONY CTP) or week 5 (HARMONY 1) for the mean change in weekly rate of cataplexy, with further improvements observed in pitolisant-treated patients through the end of treatment. The percentage of treatment responders was significantly greater with pitolisant vs placebo beginning at week 3 for excessive daytime sleepiness (defined as an Epworth Sleepiness Scale score reduction ≥ 3) and week 2 for cataplexy (defined as a ≥ 50% reduction in weekly rate of cataplexy [HARMONY CTP]).

conclusionsOnset of clinical response for excessive daytime sleepiness and/or cataplexy was generally observed within the first 2-3 weeks of pitolisant treatment in patients with narcolepsy. CLINICALTRIALS. GOV IDENTIFIER: NCT01067222 (February 2010), NCT01800045 (February 2013).

Indexed as

AdolescentAdultAgedAged, 80 and overCataplexyCircadian RhythmDose-Response Relationship, DrugDouble-Blind MethodFemaleHumansMaleMiddle AgedNarcolepsyPiperidinesTime FactorsTreatment OutcomePiperidinespitolisant

Identifiers

PMID34822113
PMCPMC8642365
OpenAlexW3216141547

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.