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A Open-label Food Effect Study With SEN0014196 in Subjects With Huntington Disease

Phase 1
Completed
Conditions
Huntington's Disease
Interventions
Registration Number
NCT01485965
Lead Sponsor
Siena Biotech S.p.A.
Brief Summary

The primary purpose of this study is to assess the effect of food upon the pharmacokinetics (PK) of SEN0014196 in subjects with Huntington disease (HD).

Detailed Description

In addition to the pharmacokinetic endpoints, the study will assess the safety and tolerability of 100 mg once daily (qd) doses of SEN0014196 over 14 days in subjects with HD and explore potential biomarkers for use in subsequent Phase 2/3 studies.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
26
Inclusion Criteria
  • Subjects with early to mid HD, i.e., genetically confirmed HD (cytosine, adenine, guanine [CAG] codon repeat length ≥ 36), motor signs of HD, and a Total Functional Capacity Subscale Score (TFC) ≥ 7
  • Body mass index between 18 and 31 kg/m2 inclusive
  • All subjects must have a body weight greater than 50 kg
  • Female subjects must be surgically sterile, postmenopausal, or willing to practice a highly effective method of contraception. All female participants must be nonlactating and nonpregnant. Male subjects must agree to use a reliable method of birth control during the study and for 3 months after the last dose of study drug.
  • Capable of providing informed consent
  • MMSE ≥24
  • Subjects must have a live-in competent observer
Exclusion Criteria
  • Participation in a study or received an investigational drug within 30 days of the Baseline Visit
  • Any prior or concomitant use of compounds suspected of interfering with protein acetylation
  • Any concomitant use of medications that are known inhibitors of CYP450 enzymes or substrates of CYP1A2 at the time of enrollment
  • Suicide risk, as determined by meeting either of the following criteria: a) a suicide attempt within the past year or suicidal ideation within 60 days of the Screening Visit; b) Significant risk of suicide, as judged by the Investigator
  • Subjects with MMSE < 24
  • Subjects with presence of clinically significant psychosis and/or confusional states, in the opinion of the Investigator
  • Subjects with clinically significant laboratory or ECG abnormalities at Screening or Baseline
  • Subjects with clinically relevant hematologic, hepatic, cardiac, or renal disease
  • Subjects with a current or past (within the last 12 months) history of epilepsy or seizures
  • A medical history of infection with human immunodeficiency virus, hepatitis C, and/or hepatitis B
  • Subjects with a history of substance abuse within the past 12 months
  • Female subjects who are pregnant or breastfeeding
  • Known allergy to any ingredient in the study drug
  • A history of malignancy of any type within 2 years prior to Screening. A history of surgically-excised nonmelanoma skin cancers is permitted.
  • Any relevant condition, behavior, laboratory value, or concomitant medication which, in the opinion of the Investigator, makes the subject unsuitable for entry into the study

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Fasted conditionSEN0014196Subjects in the Fasted group will take study drug after an overnight fast (since at least midnight). Additionally, on PK assessment days, no food will be allowed for at least 4 hours after study drug administration.
Fed conditionSEN0014196Subjects in the Fed group will take study drug within 30 minutes after starting breakfast; these subjects will otherwise maintain their normal eating schedule.
Primary Outcome Measures
NameTimeMethod
To determine the effect of food on the repeated dose pharmacokinetics of SEN0014196 at 100 mg once daily in subjects with Huntington's disease14 Days

The effect of food on the PK of SEN0014196 will be evaluated for the following parameters: 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).

Secondary Outcome Measures
NameTimeMethod
Pharmacodynamics14 Days

The following PD biomarkers will be assessed: soluble huntingtin levels and huntingtin acetylation status (by enzyme-linked immunosorbent assay \[ELISA\] and liquid chromatography-tandem mass spectrometry \[LC-MS/MS\]).

To determine the safety and tolerability of repeated doses of SEN0014196 at 100 mg once daily in subjects with Huntington's disease14 Days

Safety assessments include: AEs, ECGs, vital signs, body weight and height, clinical laboratory evaluations (serum biochemistry and hematology), urinalysis, physical and neurological examinations, C-SSRS, and UHDRS.

Trial Locations

Locations (7)

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

Wake Forest School of Medicine

🇺🇸

Winston-Salem, North Carolina, United States

University of Rochester

🇺🇸

Rocherster, New York, United States

University of California San Diego

🇺🇸

La Jolla, California, United States

University of California Davis Medical Center

🇺🇸

Sacramento, California, United States

Washington University

🇺🇸

St. Louis, Missouri, United States

University of Texas Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

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