Study to assess the effects of BN82451B and how it is absorbed, distributed and eliminated in the body, when it is given for 4 weeks to men suffering from Hungtington's disease

Phase 1

• Conditions: Huntington's disease; MedDRA version: 18.1Level: PTClassification code 10070668Term: Huntington's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2013-002899-41-DE
• Lead Sponsor: Ipsen Pharma

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-01-24
• Last Update Posted: 22 May 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: Not specified

Inclusion Criteria

(1) Male subjects = 20 to = 70 years old.
(2) Provision of written informed consent prior to any study related procedures.
(3) Confirmed symptomatic HD diagnosed based on clinical features (i.e. Diagnostic Confidence Level = 4) and presence
of = 36 CAG repeats in the Huntington gene as documented by a copy of a previous genetic test report.
(4) UDHRS-TMS = 15.
(5) Ambulatory.
(6) UHDRS-Total Functional Capacity (TFC) =3 (i.e. Shoulson & Fahn Scale stages 1-3 inclusive)
(7) Subjects on antipsychotic, antidepressant, anxiolytic and hypnotic therapy must have been on stable treatment
4 weeks prior to study drug start and during the study period.
(8) Able to swallow study medication.
(9) Able to perform Q-Motor tests.
(10) If his partner is at risk of pregnancy, the subject agrees to use a condom or be abstinent for 14 days after the last intake of study drug.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 28
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

(1) Juvenile forms of HD.
(2) Any form of chorea other than HD.
(3) History of seizure, epilepsy or other convulsive disorder.
(4) History of conditions susceptible to induce seizures such as severe traumatic brain injury, brain tumours, stroke.
(5) History of neurosurgical procedure.
(6) Current evidence or history (within 1 year of Baseline) of psychosis, hallucinations or delusions, including major
depression with psychotic features, as defined in the Diagnostic and Statistical Manual, Fourth Edition, Text
Revision (DSM-IV-TR). Patients currently experiencing mild depression, or moderate depression which is adequately
and appropriately treated in the judgement of the investigator, can participate if depression is not expected to
interfere with study participation.
(7) History of drug and/or alcohol abuse as per the DSM IV-TR criteria within 12 months prior to Baseline.
(8) At imminent risk of self harm based on investigator’s clinical judgment, with a yes” answer on item 4 or 5 on the
CSSRS questionnaire.
(9) Mini Mental State Exam (MMSE) total score = 23.
(10) Used any investigational drugs within 30 days prior to Screening or 5 half lives, whichever is the longest.
(11) Known allergy/sensitivity to the study drugs or their excipients.
(12) A severe or ongoing unstable medical condition (e.g. cardiac, hepatic, renal, metabolic or endocrine).
(13) Any clinically significant condition which, in the opinion of the investigator, would interfere with the trial evaluations or
optimal participation in the trial.
(14) Any significant laboratory results which, in the investigator’s opinion, would not be compatible with study
participation or represent a risk for subjects while in the study.
(15) History of malignant disease within the 5 years prior to Screening (with the exception of basal cell and squamous
cell carcinomas of the skin that have been completely excised, in situ prostate cancer with a normal prostate
specific antigen).
(16) An estimated Creatinine Clearance (CrCl) of <60 mL/minute (using the Cockcroft-Gault formula).
(17) ALT or AST values =2x the Upper Limit of Normal range (ULN) or both or both GGT and ALP >3x ULN
(18) Known history of hepatitis B or C or Human Immunodeficiency Virus (HIV) or positive serology at
Screening.
(19) Corrected QT interval using Bazett's correction (QTcB) >450 ms or other clinically significant ECG findings.
(20) Receiving tetrabenazine within 4 weeks prior to Baseline
(21) Receiving drugs at doses known to induce seizures with an incidence of more than 1% as per the reference list
(22) Taking the following prohibited medications/substances: Strong Cytochrome (CYP) 3A4 inhibitors, strong CYP3A4 inducers and CYP2B6 substrates (Wash out prior to Baseline 30 days or 5 half lives,whichever is the longest). CYP1A2 substrates, CYP3A4 substrates and CYP2C19 substrates may be allowed and will be assessed on a case by case basis according to the dose received.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified