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Long Term Open Label Study in Narcolepsy With BF2.649 (Pitolisant) (HARMONYIII)

Primary Purpose

Narcolepsy, Cataplexy

Status

Completed

Phase

Phase 3

Locations

France

Study Type

Interventional

Intervention

BF2.649

About this trial

This is an interventional treatment trial for Narcolepsy focused on measuring Excessive Daytime Sleepiness (EDS), Narcolepsy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Males or females, aged 18 years old and over.
Patients with a diagnosis of narcolepsy according to the International Classification of Sleep Disorders (ICSD-2) criteria.
Patients should have complained of EDS with an ESS score at least 12 (historical assessment).
Patients having previously participated in and completed a Bioprojet narcolepsy study assessing BF2.649 efficacy (P05-03, P06-06, P07-03 HARMONY I or P07-07 HARMONY II, P09-15 HARMONY I bis) or narcoleptic patients complaining with EDS which in the opinion of the investigator would not be able to participate in a double blind study against placebo but who could benefit from testing a new therapy such as the BF2.649 in an open label study.

or patient receiving BF2.649(Pitolisant) under condition of"ATU nominative" according to the French law (called named temporary authorization of use approved by the Afssaps) for Excessive Daytime Sleepiness associated with narcolepsy.

Exclusion Criteria:

Patients who have discontinued study treatment during the previous studies due to adverse events related to BF2.649.
Patients with an untreated sleep apnoea syndrome or who have any other cause of daytime sleepiness
Patients working in an occupation requiring variable shift work or routine night shifts.
Psychiatric and neurological disorders, other than narcolepsy/cataplexy, or other problem that in the investigator's opinion would preclude the patient's participation and completion of this trial or comprise reliable representation of subjective symptoms.
Current or recent (within one year) history of a substance abuse or dependence disorder including alcohol abuse as defined in DSM-IV.
Other active clinically significant illness, including unstable cardiovascular, or neoplasic pathology which could interfere with the study conduct or counter-indicate the study treatments or place the patient at risk during the trial or compromise the study participation.
Known history of long QTc syndrome, syncope or arrhythmia or any significant serious abnormality of the ECG (e.g. recent myocardial infarction), or QTc interval strictly higher than 450 ms (electrocardiogram Bazett's corrected QT interval
Severe Hepatic Impairment or with Severe Renal Impairment, or with any other significant abnormality in the physical examination or clinical laboratory results.
Known hypersensitivity to the tested treatment including active substance and excipients.
Participation in an other study - in the 30 days prior to the entry in this study

Sites / Locations

CHU Montpellier

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BF2.649

Arm Description

Outcomes

Primary Outcome Measures

Incidence of Treatment Emergent Adverse Event (TEAE)

Number of Treatment Emergent Adverse Event (TEAE)

Secondary Outcome Measures

efficacy on EDS as measured by ESS ( Epworth Sleepiness Scale)

change from baseline of the score of Epworth Sleepiness Scale score (ESS) and calculation of the rate of responders defined as a reduction of 3 points of the ESS score from baseline or the normalization of the ESS score at 10 or below

Full Information

NCT ID

NCT01399606

First Posted

June 21, 2011

Last Updated

January 30, 2017

Sponsor

Bioprojet

1. Study Identification

Unique Protocol Identification Number

NCT01399606

Brief Title

Long Term Open Label Study in Narcolepsy With BF2.649 (Pitolisant)

Acronym

HARMONYIII

Official Title

An Open-label Naturalistic Pragmatic Study to Assess the Long Term Safety of BF2.649 (Pitolisant) in the Treatment of Excessive Daytime Sleepiness (EDS) in Narcolepsy (12 Months Follow-up, Followed by a Prolonged Follow up)

Study Type

Interventional

2. Study Status

Record Verification Date

January 2017

Overall Recruitment Status

Completed

Study Start Date

May 2011 (undefined)

Primary Completion Date

October 2013 (Actual)

Study Completion Date

September 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bioprojet

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a multicentric International Phase III,Long term open label study(12 months)assessing the long-term safety and efficacy of BF2.649 (Pitolisant)in the treatment of Excessive Daytime Sleepiness (EDS) in narcoleptic patients with or without cataplexy.

Detailed Description

In narcoleptic patients, results obtained in the previous studies showed that BF2.649 reduced significantly the diurnal somnolence compared to placebo confirming its wakening effect against EDS and demonstrating its anti-cataplectic effect when administrated on an individual titration scheme established on basis of individual benefit/tolerance ratio.This long-term safety pragmatic study will provide information about long-term treatment of BF2.649, and also allow patients who satisfy of BF2.649 treatment in previous or on-going studies of Bioprojet continuing to benefit this product.This is an open-label, naturalistic pragmatic, prospective longitudinal uncontrolled, multi-centre international trial assessing the safety and effect of long-term therapy of BF2.649 in treatment of excessive daytime sleepiness in narcolepsy, on 12-month follow-up.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Narcolepsy, Cataplexy

Keywords

Excessive Daytime Sleepiness (EDS), Narcolepsy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

102 (Actual)

8. Arms, Groups, and Interventions

Arm Title

BF2.649

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

BF2.649

Other Intervention Name(s)

Pitolisant

Intervention Description

5,10,20,or 40 mg per day, in a once daily scheme

Primary Outcome Measure Information:

Title

Incidence of Treatment Emergent Adverse Event (TEAE)

Description

Number of Treatment Emergent Adverse Event (TEAE)

Time Frame

12 months

Secondary Outcome Measure Information:

Title

efficacy on EDS as measured by ESS ( Epworth Sleepiness Scale)

Description

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Males or females, aged 18 years old and over. Patients with a diagnosis of narcolepsy according to the International Classification of Sleep Disorders (ICSD-2) criteria. Patients should have complained of EDS with an ESS score at least 12 (historical assessment). Patients having previously participated in and completed a Bioprojet narcolepsy study assessing BF2.649 efficacy (P05-03, P06-06, P07-03 HARMONY I or P07-07 HARMONY II, P09-15 HARMONY I bis) or narcoleptic patients complaining with EDS which in the opinion of the investigator would not be able to participate in a double blind study against placebo but who could benefit from testing a new therapy such as the BF2.649 in an open label study. or patient receiving BF2.649(Pitolisant) under condition of"ATU nominative" according to the French law (called named temporary authorization of use approved by the Afssaps) for Excessive Daytime Sleepiness associated with narcolepsy. Exclusion Criteria: Patients who have discontinued study treatment during the previous studies due to adverse events related to BF2.649. Patients with an untreated sleep apnoea syndrome or who have any other cause of daytime sleepiness Patients working in an occupation requiring variable shift work or routine night shifts. Psychiatric and neurological disorders, other than narcolepsy/cataplexy, or other problem that in the investigator's opinion would preclude the patient's participation and completion of this trial or comprise reliable representation of subjective symptoms. Current or recent (within one year) history of a substance abuse or dependence disorder including alcohol abuse as defined in DSM-IV. Other active clinically significant illness, including unstable cardiovascular, or neoplasic pathology which could interfere with the study conduct or counter-indicate the study treatments or place the patient at risk during the trial or compromise the study participation. Known history of long QTc syndrome, syncope or arrhythmia or any significant serious abnormality of the ECG (e.g. recent myocardial infarction), or QTc interval strictly higher than 450 ms (electrocardiogram Bazett's corrected QT interval Severe Hepatic Impairment or with Severe Renal Impairment, or with any other significant abnormality in the physical examination or clinical laboratory results. Known hypersensitivity to the tested treatment including active substance and excipients. Participation in an other study - in the 30 days prior to the entry in this study

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Kati Gutierrez, PharmD

Organizational Affiliation

Bioprojet

Official's Role

Study Director

Facility Information:

Facility Name

CHU Montpellier

City

Montpellier

ZIP/Postal Code

34000

Country

France

12. IPD Sharing Statement

Learn more about this trial

Long Term Open Label Study in Narcolepsy With BF2.649 (Pitolisant)

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