A multicentre, multinational prospective observational imaging biomarker study in early stage Huntington’s disease (HD) patients to assess imaging techniques and parameters able to support efficacy studies with SEN0014196 in HD patients during Phase II and III studies

ISRCTN ISRCTN26193242
DOI https://doi.org/10.1186/ISRCTN26193242
EudraCT/CTIS number 2010-021563-32
ClinicalTrials.gov number NCT01485952
Secondary identifying numbers 9944
Submission date
07/06/2011
Registration date
07/06/2011
Last edited
29/03/2017
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data

Plain English summary of protocol

Not provided at time of registration

Study website

Contact information

Prof Sarah Tabrizi
Scientific

University College London
London
WC1E 6BT
United Kingdom

apatel@ion.ucl.ac.uk

Study information

Study designRandomised; Interventional; Design type: Treatment
Primary study designInterventional
Secondary study designRandomised controlled trial
Study setting(s)Hospital
Study typeTreatment
Participant information sheet Not available in web format, please use the contact details to request a patient information sheet
Scientific titleA multicentre, multinational prospective observational imaging biomarker study in early stage Huntington’s disease (HD) patients to assess imaging techniques and parameters able to support efficacy studies with SEN0014196 in HD patients during Phase II and III studies
Study acronymSEN0014196
Study objectivesThe study is an exploratory, multicentre, randomised and double blind placebo controlled, parallel group design at two dose levels with once daily administration over two weeks in clinical stages I-II of Huntington’s disease (HD) patients.

SEN0014196 is an investigational drug previously tested on healthy volunteers and will be tested for the first time on patients with HD. Previous studies in healthy volunteers have shown the drug to be safe and well tolerated. This study is expected to provide further information on how the compound works in humans and help further clinical development of SEN0014196. The study will be conducted at two different dose levels (10mg or 100mg of SEN0014196 given orally once daily) and there will also be a placebo arm. The study will assess what the drug does to the body (Pharmacodynamic effect) and what the body does to the drug (Pharmacokinetics). To do this repeated blood samples will be taken for the duration of the study. Safety assessments will also be conducted including ECGs, Blood Pressure, Pulse, Body Temperature, Laboratory Safety tests and a Physical Exam.

The total duration of the study is 28 days (14 days on treatment plus a 14 day Follow up Period), excluding the Screening period. 60 patients (males & females aged 18 to 70) with early stage HD will be involved in this study which will be conducted across 6 sites in the EU.
Ethics approval(s)10/H0803/169; First MREC approval date 07/02/2011
Health condition(s) or problem(s) studiedTopic: Dementias and Neurodegenerative Diseases Research Network; Subtopic: Huntington’s Disease; Disease: Huntington's disease
InterventionRepeated blood sampling, pharmacokinetic and pharmacodynamic measurements relating to study drug; study drug/placebo, safety and efficacy study of drug versus placebo; Study Entry : Single Randomisation only
Intervention typeOther
Primary outcome measurePharmacokinetic & pharmacodynamic blood sample analysis; Timepoint(s): various depending on study visit
Secondary outcome measuresAcute phenotypical effects; Timepoint(s): cognitive, motor, functional, behavioural and quality of life measures
Overall study start date11/04/2011
Completion date11/11/2011

Eligibility

Participant type(s)Patient
Age groupAdult
Lower age limit18 Years
SexBoth
Target number of participantsPlanned Sample Size: 60; UK Sample Size: 20; Description: Early stage Huntington's disease
Key inclusion criteria1. Patients with early Huntington’s Disease (aged 18 to 70 years), i.e. genetically confirmed (CAG repeat length = 36) HD, motor signs of HD (motor score of the Unified Huntington's Disease Rating Scale [UHDRS] > 5) and a Total Functional Capacity (TFC) of = 7
2. All patients will have a body weight greater than 50kg
3. Female subjects must be surgically sterile or postmenopausal (defined as at least two years post cessation of menses and/or follicular stimulating hormone >18 mIU/mL and serum oestradiol <110 pmol/L), no spontaneous menstruation for at least one year before the first dose, nonlactating and have a negative serum pregnancy test
[Note: For postmenopausal females treated with estrogen replacement therapy, folloicle stimulating hormones (FSH) levels are artificially lowered < 40 IU/L. Estradiol and Luteinizing hormone (LH) measurements need to be performed to confirm reason for low FSH value. Hormone replacement therapy (HRT) is acceptable, provided the above criteria are respected].
4. All subjects must be capable of providing written informed consent
5. 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
6. Male subjects participating in the trial and their female partners must agree to use a highly effective method of 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 (i.e. a diaphragm or cervical/vault cap), with spermicidal cream/gel plus one extra method (i.e. established use of oral, injected or implanted hormonal contraception or IUD or Coil). If the female partner is already pregnant, use of condoms is mandatory during sexual intercourse from the time of taking the first dose of study drug until three months after taking the last dose of study drug.
Target Gender: Male & Female; Upper Age Limit 70 years ; Lower Age Limit 18 years
Key exclusion criteria1. Participation in a study of an investigational drug within 30 days of the baseline visit
2. Subjects with presence of psychosis and/or confusional states
3. Subjects with clinically significant laboratory or electrocardiogram (ECG) abnormalities at screening
4. Subjects with clinically relevant haematological, hepatic, cardiac or renal disease
5. A medical history of infection with Human immunodeficiency virus, Hepatitis C and/or Hepatitis B
6. 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
7. Subjects who have previously received histone deacetylase inhibitors e.g. vorinostat (Zolinza®) or have participated in a clinical trial using a histone deacetylase inhibitor
8. A history of malignancy of any type within 2 years prior to screening. A history of surgically excised nonmelanoma skin cancers is permitted
9. Subjects with a significant history of drug allergy as determined by the Investigator
10. Subjects who consume more than 28 units (males) or more than 21 units (females) of alcohol per week or who have a significant history of alcoholism or drug/ chemical abuse as determined by the Investigator (one unit of alcohol equals 285 mL of beer or lager, one glass [125 mL] of wine, or 25 mL of spirits)
Date of first enrolment11/04/2011
Date of final enrolment11/11/2011

Locations

Countries of recruitment

  • England
  • United Kingdom

Study participating centre

University College London
London
WC1E 6BT
United Kingdom

Sponsor information

Siena Biotech (Italy)
Industry

ddress 35
Strada del Petriccio e Belriguardo
Siena
53100
Italy

ROR logo https://ror.org/02ka1d450

Funders

Funder type

Government

Seventh Framework Programme
Government organisation / National government
Alternative name(s)
EC Seventh Framework Programm, European Commission Seventh Framework Programme, EU Seventh Framework Programme, European Union Seventh Framework Programme, EU 7th Framework Programme, European Union 7th Framework Programme, Siebten Rahmenprogramm, Séptimo Programa Marco, Septième programme-cadre, Settimo programma quadro, 7th Framework Programme, Seventh EU Framework Programme, FP7

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan

Study outputs

Output type Details Date created Date added Peer reviewed? Patient-facing?
Results article results 01/03/2015 Yes No
HRA research summary 28/06/2023 No No

Editorial Notes

29/03/2017: Publication reference added.

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