Clinical trial NCT02074410: Safety and Efficacy of OMS643762 in Subjects With Huntington's Disease
Official title: Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety and Efficacy of OMS643762 in Subjects With Huntington's Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Huntington's Disease
Lead sponsor: Omeros Corporation
Sponsor class: INDUSTRY
First posted: 2014-02-28
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety, tolerability and pharmacokinetics of OMS643762 (the study drug) in subjects with Huntington's disease (HD).


Eligibility criteria: Inclusion Criteria:

1. Voluntarily provide informed consent, or have a legally authorized representative (LAR) provide informed consent with subject assent, in accordance with local regulations and governing Institution Review Board (IRB) requirements prior to any procedures or evaluations performed specifically for the sole purpose of the study (other than the Montreal Cognitive Assessment (MoCA) to assess capacity to provide informed consent). Capacity to provide informed consent will be determined by the MoCA and investigator judgment according to the following:

   * Subjects with scores of greater than or equal to 21 on the MoCA and, in the judgment of the investigator, have the capacity to provide valid informed consent, can give consent.
   * Subjects with scores of less than 21 but greater than or equal to 18 on the MoCA and, in the judgment of a mental health professional (independent of the investigator) have the capacity to provide valid informed consent, may give consent.
   * Subjects with scores less 21 but greater than or equal to 18 on the MoCA, who lack the capacity to give valid informed consent, in the judgment of a mental health professional (independent of the investigator), will need an LAR to provide informed consent with assent by the subject.
   * Subjects with scores of less than 18 on the MoCA will have an LAR provide informed consent with assent by the subject.
2. Have a clinical diagnosis of HD, confirmed by either CAG repeat number of greater than or equal to 39 or a positive family history (a first degree relative with a clinical diagnosis of HD) if CAG repeat number is not known.
3. Are age greater than or equal to 18 and less than or equal to 65 years at the screening visit (Visit 1).
4. Have a UHDRS Total Functional Capacity greater than or equal to 7 at Visit 1.
5. If currently taking antipsychotic medication(s), have been on a stable regimen for at least 60 days prior to randomization.
6. Are fluent in English.
7. If female, are either a) not of childbearing potential (i.e., surgically sterilized or post-menopausal for more than 1 year) or b) have a negative pregnancy test and if sexually active must agree to use a medically reliable form of contraception throughout the study. Acceptable methods of contraception include a reliable intrauterine device, hormonal contraception or spermicide in combination with a barrier method.
8. If male, are either a) not of reproductive potential or b) if sexually active must agree to use a medically reliable form of contraception throughout the study. Acceptable methods of birth control include spermicide in combination with a barrier method, or subjects' female partner is willing to use medically acceptable methods of birth control.
9. Have normal clinical laboratory test results and ECG, or results with minor deviations, which are not considered to be clinically significant by the investigator.

Exclusion Criteria:

1. Have a history or presence of significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, or neurological disorders other than HD which, in the opinion of the investigator, increases the risk of the study drug or may confound the interpretation of study measures.
2. Have unstable or severe depression, in the opinion of the investigator.
3. Have alcohol or drug abuse or dependence, as defined by the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision.
4. Have received treatment with an investigational drug or device within 60 days prior to Visit 1.
5. Are pregnant or lactating.
6. Have serum alanine transaminase or aspartate transaminase greater than two times upper limit of normal at screening.
7. Have hemoglobin, white blood cell count, absolute neutrophil count, or platelet count outside the normal range at screening.
8. Are an employee of Omeros, an investigator, or study staff member, or their immediate family member.

Primary outcomes:
measure: Assess the Safety of OMS643762
timeFrame: 28 days
measure: Assess the Safety of OMS643762
timeFrame: 28 days
measure: Assess the Safety of OMS643762
timeFrame: 28 days
measure: Assess the Safety of OMS643762
timeFrame: 28 days
measure: Assess the Safety of OMS643762
timeFrame: 28 days
Secondary outcomes:
measure: Motor function
timeFrame: Pre-dose and day 15 and 28 post-dose
measure: Motor function
timeFrame: Pre-dose and day 15 and 28 post-dose
measure: Cognition
timeFrame: Pre-dose and day 28 post-dose
measure: Behavior
timeFrame: Pre-dose and day 28 of dosing
measure: Pharmacokinetics profile
timeFrame: Pre-dose, day 15 and 28 of dosing and up to 14 days post-dose
Other outcomes:
Locations:
city: San Diego
country: United States
state: California
city: Englewood
country: United States
state: Colorado
city: Gainesville
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Baltimore
country: United States
state: Maryland
city: New York
country: United States
state: New York
city: Memphis
country: United States
state: Tennessee
city: Houston
country: United States
state: Texas
city: Kirkland
country: United States
state: Washington
Study officials:
affiliation: Omeros Corporation
name: Steve Whitaker, MD
role: STUDY_DIRECTOR
References: