Huntington DiseasePhase 1PADDINGTONNCT01485952
An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196
A Phase 1 study testing SEN0014196 (Low Dose) and SEN0014196 (High Dose) in people with Huntington Disease, run by Siena Biotech S.p.A. at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- SEN0014196 (Low Dose) and SEN0014196 (High Dose)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 55 participants
- Where
- 1 siteGermany
- Study dates
- Mar 2011to Nov 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
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.
Read the 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.
- Conditions
- Huntington Disease
- Being tested
- SEN0014196 (Low Dose), SEN0014196 (High Dose), Placebo
- 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
- Medical subject headings
- Huntington Disease, Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Dementia, Chorea, Dyskinesias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SEN0014196 (Low Dose)Drug
10 mg once daily administration (immediate release capsule)
SEN0014196 (High Dose)Drug
100 mg once daily administration (immediate release capsule)
PlaceboDrug
Once daily administration (immediate release capsule)
| Study group | Role | What they receive |
|---|---|---|
| SEN0014196 (Low Dose) | Experimental | 10 mg, once daily administration (immediate release capsule) Receives: Drug: SEN0014196 (Low Dose) |
| SEN0014196 (High dose) | Experimental | 100 mg, once daily administration (immediate release capsule) Receives: Drug: SEN0014196 (High Dose) |
| Placebo | Placebo comparator | Once daily (immediate release capsule) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Safety assessments will include ECG, vital signs, laboratory safety tests, and physical and neurological examination. Tolerability will include type and frequency of adverse events.
Measured: 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
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.
Measured: Baseline, Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2011 (estimated)
- Study startMar 2011
- Primary completionNov 2011
- Study completionNov 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- Siena Biotech S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Seventh Framework Programme, European Huntington's Disease Network
- Responsible party
- Sponsor
- Investigators
- Bernhard G Landwehrmeyer, MD, PhD, Principal investigator, European Huntington's Disease Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01485952
- Sponsor's protocol ID
- S015-004
- Other IDs
- CTA Number: 2010-021563-32
- Acronym
- PADDINGTON
- Status last verified
- Nov 2015
- First submitted
- 29 Nov 2011
- Last updated
- 25 Nov 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT01485952
- Acronym / sponsor ID
- PADDINGTON / S015-004
- Conditions
- Huntington Disease
- Keywords
- Huntington Disease, Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Dementia, Chorea, Dyskinesias
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 55 participants (actual)
- Lead sponsor
- Siena Biotech S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- Seventh Framework Programme, European Huntington's Disease Network
- Study start
- Mar 2011
- Primary completion
- Nov 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 6 Dec 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SEN0014196 (Low Dose) | Experimental | Drug: SEN0014196 (Low Dose)10 mg, once daily administration (immediate release capsule) |
| SEN0014196 (High dose) | Experimental | Drug: SEN0014196 (High Dose)100 mg, once daily administration (immediate release capsule) |
| Placebo | Placebo comparator | Drug: PlaceboOnce daily (immediate release capsule) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SEN0014196 (Low Dose)10 mg once daily administration (immediate release capsule) | Drug | SEN0014196 (Low Dose) |
| SEN0014196 (High Dose)100 mg once daily administration (immediate release capsule) | Drug | SEN0014196 (High dose) |
| PlaceboOnce daily administration (immediate release capsule) | Drug | Placebo |
Primary outcome
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
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
2 secondary outcomes
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bernhard G Landwehrmeyer, MD, PhD principal investigator
European Huntington's Disease Network
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SEN0014196 (Low Dose) and SEN0014196 (High Dose) in people with Huntington Disease, run by Siena Biotech S.p.A. at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Nov 2015.