Early-Stage Huntington Disease MedDRA version: 20.0 Level: PT Classification code 10070668 Term: Huntington's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
EIP Pharma, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Men and women age 30 to 70 years, inclusive. 2. Willing and able to provide informed consent. 3. Must have genetically confirmed HD and identified cognitive deficits: a. Stage 1, as defined by Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) score >=9, and, b. CANTAB Paired Associate Learning Total Adjusted Error Score of >16. 4. Normal or corrected eye sight and auditory abilities, sufficient to perform all aspects of the cognitive and functional assessments. 5. No history of learning difficulties that may interfere with the subject’s ability to complete the cognitive tests. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1. A profile of impairment that is not consistent with HD. 2. Diagnosis of any other ongoing central nervous system condition other than HD, including, but not limited to, vascular dementia, dementia with Lewy bodies, and Parkinson’s disease. 3. Suicidality, defined as active suicidal thoughts within 6 months before Screening or at Baseline, defined as answering yes to items 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS), or history of suicide attempt in previous 2 years, or, in the Investigator’s opinion, at serious risk of suicide. 4. Ongoing major and active psychiatric disorder, moderate to severe depressive symptoms, and/or other concurrent medical condition that, in the opinion of the Investigator, might compromise safety and/or compliance with study requirements. 5. Diagnosis of alcohol or drug abuse within the previous 2 years. 6. Poorly controlled clinically significant medical illness, such as hypertension (blood pressure >180 mmHg systolic or 100 mmHg diastolic); myocardial infarction within 6 months; uncompensated congestive heart failure or other significant cardiovascular, pulmonary, renal, liver, infectious disease, immune disorder, or metabolic/endocrine disorders or other disease that would preclude treatment with p38 mitogen activated protein (MAP) kinase inhibitor and/or assessment of drug safety and efficacy. 7. Anemia with a hemoglobin =10 g/dL, clinically significant thyroid function abnormality, electrolyte abnormalities. 8. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >1.5 × the upper limit of normal (ULN), total bilirubin >1.5 × ULN, and/or International Normalized Ratio (INR) >1.5. 9. Known human immunodeficiency virus; or active hepatitis B or hepatitis C virus infection; evidence of active or latent tuberculosis. 10. Subject participated in a study of an investigational drug less than 3 months or 5 half-lives of an investigational drug, whichever is longer, before enrollment in this study. 11. History of previous neurosurgery to the brain. 12. Female subjects who are pregnant or breast-feeding. 13. Male subjects with female partners of child-bearing potential who are unwilling or unable to adhere to contraception requirements specified in the protocol. 14. Female subjects who have not reached menopause or have not had a hysterectomy or bilateral oophorectomy/salpingooophorectomy and are not willing or unable to adhere to contraceptive requirements specified in the protocol 15. Requires concomitant use of cytochrome P450 (CYP) 3A4 inhibitors or anti-tumor necrosis factor-alpha therapies during study participation. 16. Known allergy to any ingredient of the trial medication or placebo
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the effects of administration of neflamapimod on hippocampal function, as assessed in the virtual MWM.;Primary end point(s): Latency during the learning phase of virtual MWM (hidden platform training) during the neflamapimod-treatment period compared to that during the placebo-administration period.;Timepoint(s) of evaluation of this end point: Screening, Baseline, Week 4, Week 10, ET; Secondary Objective: • To evaluate the effects of neflamapimod on the CANTAB paired associates learning task. • To evaluate effects of neflamapimod on a larger battery of parameters in the CANTAB. • To evaluate tolerability and safety of neflamapimod in subjects with HD. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1. and 2. (MWM and CANTAB): Screening, Baseline, Week 4, Week 10, ET 3. (Safety): throughout the trial ; Secondary end point(s): 1. Percent of time spent in the correct quadrant during MWM probe test during the neflamapimod-treatment period compared to that during the placebo-administration period. 2. Number of overall errors in the CANTAB paired associates learning task during the neflamapimod-treatment period compared to that during the placebo-administration period. 3. Safety as determined by the number of related adverse events and general tolerability reported during the neflamapimod treatment period compared to the placebo administration period. In addition, exploratory analyses of the full set of parameters in the CANTAB will be conducted. | — |
Countries
United Kingdom
Contacts
Public ContactClinical Trial Operations
Voisin Consulting
Outcome results
None listed