Study Exploring Safety, Pharmacokinetic and Pharmacodynamic of BN82451 in Male Huntington's Disease Patients

NCT02231580 | Terminated Phase 2 Results

• 2 other identifiers: 8-55-52966-0052013-002899-41
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of BN82451B versus placebo after oral administration twice daily (bid) for 28 days in patients with Huntington's Disease (HD).

Conditions

Huntington's Disease

Keywords

Neurodegenerative genetic disorder

Outcome Measures

Primary Outcomes (1)

• Numbers of Patients Experiencing Treatment Emergent Adverse Events (TEAEs).

  The safety and tolerability of BN82451B versus placebo was determined after oral administration b.i.d. for 28 days in patients with HD. Numbers of patients experiencing TEAEs, including information on seriousness, intensity, drug relationship and those leading to withdrawal are presented for all doses of BN82451B and placebo.

  From Day 1 to end of study (a period of up to 7 weeks).

Secondary Outcomes (25)

• Area Under the Plasma Concentration Time Curve (AUC)

  0-12 hours on Days 1, 14 and 28

• Peak Plasma Concentration (Cmax)

  Days 1, 14 and 28

• Time to Peak Plasma Concentration (Tmax)

  Days 1, 14 and 28

• Change From Baseline to Day 28 in the Position-index as Determined by Choreomotography

  Baseline (Day-1) to Day 28

• Change From Baseline to Day 28 in the Orientation-index as Determined by Choreomotography

  Baseline (Day -1) to Day 28

Study Arms (2)

BN82451B

EXPERIMENTAL

BN82451B capsule: Up to 3 dose levels (40, 60 or 80 mg) twice daily administered orally.

Drug: BN82451B

Placebo

PLACEBO COMPARATOR

Placebo capsule: Up to 3 dose levels (40, 60 or 80 mg) twice daily administered orally.

Drug: Placebo

Interventions

BN82451B DRUG

BN82451B capsule

BN82451B

Placebo DRUG

Placebo capsule

Placebo

Eligibility Criteria

• Age: 20 Years - 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male subjects 20 to 70 years old (inclusive).
• Provision of written informed consent prior to any study related procedures. In this study consent may be provided by the legal guardian or carer.
• Confirmed symptomatic Huntington's Disease diagnosed based on clinical features (i.e. Diagnostic Confidence Level equal to 4) and presence of at least 36 cytosine adenine guanine (CAG) repeats in the Huntington gene as documented by a copy of a previous genetic test report.
• Unified Huntington's Disease Rated Scale-Total Motor Score (UDHRS-TMS) greater than or equal to 15.
• Ambulatory.
• UDHRS-Total Functional Capacity (TFC) greater than or equal to 3 (i.e. Shoulson \& Fahn Scale stages 1-3 inclusive.
• 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.
• Able to swallow study medication.
• Able to perform Q-Motor tests.
• 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.

You may not qualify if:

• Juvenile forms of Huntington's Disease.
• Any form of chorea other than Huntington's Disease.
• History of seizure, epilepsy or other convulsive disorder, with the exception of febrile seizures in childhood.
• History of conditions susceptible to induce seizures such as severe traumatic brain injury, brain tumours, stroke.
• History of neurosurgical procedure.
• 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.
• History of drug and/or alcohol abuse as per the DSM IV-TR criteria within 12 months prior to Baseline.
• At imminent risk of self harm based on investigator's clinical judgment, with a "yes" answer on item 4 or 5 on the Columbia-Suicide Severity Rating Scale (CSSRS) questionnaire.
• Mini Mental State Exam (MMSE) total score less than or equal to 23.
• Used any investigational drugs within 30 days prior to Screening or 5 half lives, whichever is the longest.
• Known allergy/sensitivity to the study drugs or their excipients.
• A severe or ongoing unstable medical condition (e.g. cardiac, hepatic, renal, metabolic or endocrine).
• Any clinically significant condition which, in the opinion of the investigator, would interfere with the trial evaluations or optimal participation in the trial.
• 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.
• 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).

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Unknown Facility

Mönchengladbach, Germany

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Limitations and Caveats

The study was terminated prematurely due to subject recruitment problems before the completion of cohort 2.

Results Point of Contact

• Title: Vice President Early Development & Clinical Pharmacology
• Organization: Ipsen

clinical.trials@ipsen.com

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 2, 2014
• First Posted: September 4, 2014
• Study Start: September 1, 2014
• Primary Completion: March 31, 2016
• Study Completion: March 31, 2016
• Last Updated: January 15, 2019
• Results First Posted: January 29, 2018

Record last verified: 2019-01

Locations

DE (1)