Pitolisant to Assess Weekly Frequency of Cataplexy Attacks and EDS in Narcoleptic Patients (HARMONY CTP)

• ClinicalTrials.gov Identifier: NCT01800045
• Recruitment Status: Completed
• First Posted: February 27, 2013
• Last Update Posted: August 31, 2016
• Sponsor: Bioprojet
• Information provided by (Responsible Party): Bioprojet

Tracking Information

• First Submitted Date: February 13, 2013
• First Posted Date: February 27, 2013
• Last Update Posted Date: August 31, 2016
• Study Start Date: April 2013
• Actual Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 30, 2016)

Measure of anticataplectic efficacy [ Time Frame: At week 7 ]

Measure of anticataplectic efficacy assessed by the change in the average number of cataplexy attacks per week.

Original Primary Outcome Measures (submitted: February 25, 2013)

Measure of anticataplectic efficacy [ Time Frame: At week 49 ]

Measure of anticataplectic efficacy assessed by the change in the average number of cataplexy attacks per week.

Current Secondary Outcome Measures (submitted: February 25, 2013)

• Excessive Daytime Sleepiness assessment [ Time Frame: At week 7 ]
  Excessive Daytime Sleepiness assessment by evaluation of ESS score, and other questionnaires.
• Safety assessment [ Time Frame: 11 weeks ]
  Safety assessment thanks to AE recording, vitals signs assessment, ECG...

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Pitolisant to Assess Weekly Frequency of Cataplexy Attacks and EDS in Narcoleptic Patients (HARMONY CTP)
• Official Title: 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.

Brief Summary

Double blind, randomized, parallel groups study of Pitolisant versus placebo, in narcoleptic patients experiencing EDS, and cataplexy (minimum of 3 complete or partial cataplexy attacks per week).

The patients will be treated during 7 weeks with Pitolisant or placebo.

Detailed Description

The treatment period (7 weeks double blind) will aim at demonstrating the efficacy of Pitolisant on the number of cataplexy attacks and the Excessive Daytime Sleepiness by verifying whether the results of Pitolisant are superior to those of placebo.

The safety will also be assessed.

• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Narcolepsy With Cataplexy
• Excessive Daytime Sleepiness

Intervention

• Drug: Pitolisant
  1 capsule in the morning before breakfast
  Other Name: BF2.649
• Drug: Placebo
  1 capsule in the morning before breakfast
  Other Name: Capsules of placebo containing lactose

Study Arms

• Experimental: Pitolisant
  Pitolisant at 5, 10, 20 or 40mg
  Intervention: Drug: Pitolisant
• Placebo Comparator: Placebo
  Capsules of placebo containing lactose
  Intervention: Drug: Placebo

Publications *

• Meskill GJ, Davis CW, Zarycranski D, Doliba M, Schwartz JC, Dayno JM. Clinical Impact of Pitolisant on Excessive Daytime Sleepiness and Cataplexy in Adults With Narcolepsy: An Analysis of Randomized Placebo-Controlled Trials. CNS Drugs. 2022 Jan;36(1):61-69. doi: 10.1007/s40263-021-00886-x. Epub 2021 Dec 21.
• Watson NF, Davis CW, Zarycranski D, Vaughn B, Dayno JM, Dauvilliers Y, Schwartz JC. 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. CNS Drugs. 2021 Dec;35(12):1303-1315. doi: 10.1007/s40263-021-00866-1. Epub 2021 Nov 25.
• Szakacs Z, Dauvilliers Y, Mikhaylov V, Poverennova I, Krylov S, Jankovic S, Sonka K, Lehert P, Lecomte I, Lecomte JM, Schwartz JC; HARMONY-CTP study group. Safety and efficacy of pitolisant on cataplexy in patients with narcolepsy: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017 Mar;16(3):200-207. doi: 10.1016/S1474-4422(16)30333-7. Epub 2017 Jan 25.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: January 20, 2015): 103
• Original Estimated Enrollment (submitted: February 25, 2013): 120
• Actual Study Completion Date: January 2015
• Actual Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)

Eligibility Criteria

Main Inclusion Criteria:

• Narcoleptic with cataplexy : at least 3 weekly cataplexy attacks
• ESS > or = 12
• ICF signed and dated

Main Exclusion Criteria:

• Other conditions that could generate EDS
• Psychological and neurological disorders
• Acute or chronic severe disease
• Treatment by prohibited medication

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Hungary

Administrative Information

• NCT Number: NCT01800045
• Other Study ID Numbers: P11-05 / Pitolisant; 2012-003076-39 ( EudraCT Number )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Bioprojet
• Original Responsible Party: Same as current
• Current Study Sponsor: Bioprojet
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Evelyne De Paillette, Dr; Bioprojet

• PRS Account: Bioprojet
• Verification Date: July 2015