Trial record 1 of 4 for:
Huntington's disease, Siena Biotech
An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196 (PADDINGTON)
This study has been completed.
Sponsor:
Siena Biotech S.p.A.
Collaborators:
European Commission 7th Framework Programme
European Huntington's Disease Network
Information provided by (Responsible Party):
Siena Biotech S.p.A.
ClinicalTrials.gov Identifier:
NCT01485952
First received: November 29, 2011
Last updated: March 13, 2012
Last verified: March 2012
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Huntington's Disease |
Drug: SEN0014196 (Low Dose) Drug: SEN0014196 (High Dose) Drug: Placebo |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Pharmacokinetics/Dynamics Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients to Assess Pharmacokinetics, Candidate Pharmacodynamic Measures of Target Engagement and Disease Modulation as Well as Acute Phenotypical Effects Following Multiple Oral Doses of SEN0014196. |
Resource links provided by NLM:
Genetics Home Reference related topics:
chorea-acanthocytosis
Huntington disease
McLeod neuroacanthocytosis syndrome
MedlinePlus related topics:
Brain Diseases
Degenerative Nerve Diseases
Dementia
Huntington's Disease
Mental Disorders
Movement Disorders
Neurologic Diseases
U.S. FDA Resources
Further study details as provided by Siena Biotech S.p.A.:
Primary Outcome Measures:
- To determine the change from baseline of a series of pharmacodynamic markers in peripheral blood mononuclear cells [ Time Frame: Baseline, Day 7, Day 14, Follow-Up ] [ Designated as safety issue: No ]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
Secondary Outcome Measures:
- To determine the safety and tolerability following repeated doses of SEN0014196 over two weeks at two dose levels in patients with Huntington's disease [ Time Frame: Baseline, Day 7, Day 14, Follow-up ] [ Designated as safety issue: Yes ]Safety assessments will include ECG, vital signs, laboratory safety tests, and physical and neurological examination. Tolerability will include type and frequency of adverse events.
- To determine the pharmacokinetics of repeated doses of SEN0014196 at two dose levels when administered over two weeks in patients with Huntington's disease [ Time Frame: Baseline, Day 14 ] [ Designated as safety issue: No ]The following parameters will be assessed: maximum observed plasma concentration (Cmax), time of maximum observed plasma concentration(tmax), AUC from time zero to the length of the dosing interval (tau) (AUC0-τ), AUC from time zero to the last quantifiable concentration (AUC0-last), AUC from time zero to infinity (AUC0-∞), terminal elimination half-life (t1/2), and terminal elimination rate constant (λz). Gender differences. Dose proportionality.
| Enrollment: | 55 |
| Study Start Date: | March 2011 |
| Study Completion Date: | November 2011 |
| Primary Completion Date: | November 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: SEN0014196 (Low Dose)
10 mg, once daily administration (immediate release capsule)
|
Drug: SEN0014196 (Low Dose)
10 mg once daily administration (immediate release capsule)
|
|
Experimental: SEN0014196 (High dose)
100 mg, once daily administration (immediate release capsule)
|
Drug: SEN0014196 (High Dose)
100 mg once daily administration (immediate release capsule)
|
|
Placebo Comparator: Placebo
Once daily (immediate release capsule)
|
Drug: Placebo
Once daily administration (immediate release capsule)
|
Detailed Description:
This study will establish the acute phenotypical and biological effects of repeated dose application of SEN0014196 in patients with Huntington's disease, providing biomaterials for biomarker studies (levels of circulating huntingtin, acetylation status of mutant huntingtin, innate immune markers, transcriptional profiles). Evaluation of phenotypic effects will include UHDRS scores, total functional capacity. Safety assessments will include ECG, vital signs, laboratory safety tests and physical examination.
Eligibility| Ages Eligible for Study: | 18 Years to 70 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01485952
Locations
| Germany | |
| Universitätsklinik Ulm, Neurologie | |
| Ulm, Germany, 89081 | |
Sponsors and Collaborators
Siena Biotech S.p.A.
European Commission 7th Framework Programme
European Huntington's Disease Network
Investigators
| Principal Investigator: | Bernhard G Landwehrmeyer, MD, PhD | European Huntington's Disease Network |
More Information
Additional Information:
No publications provided
| Responsible Party: | Siena Biotech S.p.A. |
| ClinicalTrials.gov Identifier: | NCT01485952 History of Changes |
| Other Study ID Numbers: | S015-004, CTA Number: 2010-021563-32 |
| Study First Received: | November 29, 2011 |
| Last Updated: | March 13, 2012 |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency Germany: Federal Institute for Drugs and Medical Devices Poland: Ministry of Health |
Keywords provided by Siena Biotech S.p.A.:
|
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Movement Disorders Heredodegenerative Disorders, Nervous System |
Neurodegenerative Diseases Genetic Diseases, Inborn Cognition Disorders Mental Disorders Dementia Chorea Dyskinesias |
Additional relevant MeSH terms:
|
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
Genetic Diseases, Inborn Cognition Disorders Delirium, Dementia, Amnestic, Cognitive Disorders Mental Disorders Dementia Chorea Dyskinesias |
ClinicalTrials.gov processed this record on June 18, 2013