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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Bioprojet
ClinicalTrials.gov Identifier:
NCT01399606
First received: June 21, 2011
Last updated: February 24, 2014
Last verified: February 2014
  Purpose

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.


Condition Intervention Phase
Narcolepsy
Cataplexy
Drug: BF2.649
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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)

Resource links provided by NLM:


Further study details as provided by Bioprojet:

Primary Outcome Measures:
  • Incidence of Treatment Emergent Adverse Event (TEAE) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    Number of Treatment Emergent Adverse Event (TEAE)


Secondary Outcome Measures:
  • efficacy on EDS as measured by ESS ( Epworth Sleepiness Scale) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    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


Estimated Enrollment: 100
Study Start Date: May 2011
Estimated Study Completion Date: May 2015
Estimated Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BF2.649 Drug: BF2.649
5,10,20,or 40 mg per day, in a once daily scheme
Other Name: Pitolisant

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01399606

Locations
France
CHU Montpellier
Montpellier, France, 34000
Sponsors and Collaborators
Bioprojet
Investigators
Study Director: Kati Gutierrez, PharmD Bioprojet
  More Information

No publications provided

Responsible Party: Bioprojet
ClinicalTrials.gov Identifier: NCT01399606     History of Changes
Other Study ID Numbers: P09-10 / BF2.649, 2010-023804-28
Study First Received: June 21, 2011
Last Updated: February 24, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Hungary: National Institute of Pharmacy
Italy: The Italian Medicines Agency

Keywords provided by Bioprojet:
Excessive Daytime Sleepiness (EDS)
Narcolepsy

Additional relevant MeSH terms:
Cataplexy
Narcolepsy
Disorders of Excessive Somnolence
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Disorders
Nervous System Diseases
Mental Disorders

ClinicalTrials.gov processed this record on July 29, 2014