An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196

NCT01485952 | Completed Phase 1

• 2 other identifiers: S015-004CTA Number: 2010-021563-32
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of this study is to provide biological samples from patients with Huntington's disease to allow characterisation of the pharmacological mechanism of action of SEN0014196.

Conditions

Huntington Disease

Keywords

Huntington Disease; 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; Cognition Disorders; Mental Disorders

Outcome Measures

Primary Outcomes (1)

• To determine the change from baseline of a series of pharmacodynamic markers in peripheral blood mononuclear cells

  Collection of peripheral blood mononuclear cells for biomarker investigations, specifically acetylation status of mutant huntingtin, levels of circulating huntingtin, innate immune markers and transcriptional profiles

  Baseline, Day 7, Day 14, Follow-Up

Secondary Outcomes (2)

• To determine the safety and tolerability following repeated doses of SEN0014196 over two weeks at two dose levels in patients with Huntington's disease

  Baseline, Day 7, Day 14, Follow-up

• To determine the pharmacokinetics of repeated doses of SEN0014196 at two dose levels when administered over two weeks in patients with Huntington's disease

  Baseline, Day 14

Study Arms (3)

SEN0014196 (Low Dose)

EXPERIMENTAL

10 mg, once daily administration (immediate release capsule)

Drug: SEN0014196 (Low Dose)

SEN0014196 (High dose)

EXPERIMENTAL

100 mg, once daily administration (immediate release capsule)

Drug: SEN0014196 (High Dose)

Placebo

PLACEBO COMPARATOR

Once daily (immediate release capsule)

Drug: Placebo

Interventions

SEN0014196 (Low Dose) DRUG

10 mg once daily administration (immediate release capsule)

SEN0014196 (Low Dose)

SEN0014196 (High Dose) DRUG

100 mg once daily administration (immediate release capsule)

SEN0014196 (High dose)

Placebo DRUG

Once daily administration (immediate release capsule)

Placebo

Eligibility Criteria

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

You may qualify if:

• Patients with early Huntington's Disease (age: 18 to 70 years), i.e. genetically confirmed (CAG repeat length ≥36) HD, motor signs of HD (motor score of the UHDRS \> 5) and a TFC of ≥7.
• All patients will have a body weight greater than 50 kg.
• Female subjects must be surgically sterile or post-menopausal, no spontaneous menstruation for at least one year before the first dose, non-lactating and have a negative urine pregnancy test. Male subjects participating in the trial and their female contraception from the time of taking the first dose of the study drug until three months after taking the last dose. This must include a condom or other barrier method.
• All subjects must be capable of providing written informed consent.
• Subjects must have no clinically significant and relevant history that could affect the conduct of the study and evaluation of the data, as ascertained by the Investigator through detailed medical history and screening assessments.

You may not qualify if:

• Participation in a study of an investigational drug within 30 days of the baseline visit.
• Subjects with presence of psychosis and/or confusional states.
• Subjects with clinically significant laboratory or ECG abnormalities at Screening.
• Subjects with clinically relevant hematological, hepatic, cardiac or renal disease.
• A medical history of infection with human immunodeficiency virus, hepatitis C and/or hepatitis B.
• Any relevant condition, behaviour, laboratory value or concomitant medication which, in the opinion of the Investigator, makes the subject unsuitable for entry into the study.
• Subjects who have previously received histone deacetylase inhibitors e.g. vorinostat or have participated in a clinical trial using compound suspected of interfering with protein acetylation status.
• A history of malignancy of any type within 2 years prior to screening. A history of surgically excised nonmelanoma skin cancers is permitted.
• Subjects with a significant history of drug allergy as determined by the Investigator.
• Subjects who have a significant history of alcoholism or drug/chemical abuse as determined by the Investigator.

Sponsors & Collaborators

• Siena Biotech S.p.A.: lead
• Seventh Framework Programme: collaborator
• European Huntington's Disease Network: collaborator

Study Sites (1)

Universitätsklinik Ulm, Neurologie

Ulm, 89081, Germany

Location

MeSH Terms

Conditions

Huntington Disease; 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; Cognition Disorders; Mental Disorders

Interventions

6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurocognitive Disorders; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Bernhard G Landwehrmeyer, MD, PhD

  European Huntington's Disease Network

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 29, 2011
• First Posted: December 6, 2011
• Study Start: March 1, 2011
• Primary Completion: November 1, 2011
• Study Completion: November 1, 2011
• Last Updated: November 25, 2015

Record last verified: 2015-11

Locations

DE (1)