WITHIN SUBJECT CROSSOVER STUDY OF COGNITIVE EFFECTS OF NEFLAMAPIMOD IN EARLY-STAGE HUNTINGTON DISEASE
EIP Pharma Inc
The main purpose of EIP19-NFD-401 is to determine whether a drug called Neflamapimod can improve cognitive functions (planning, memory etc) in patients with early-stage Huntington’s disease (HD). The drug is taken orally. In the study 16 patients will receive the drug for 10 weeks and then be given placebo for 10 weeks or vice versa. Neither the doctor nor the patient knows in which period they get the active drug or the placebo. The effect will be tested with different cognitive tests. Neflamapimod target an enzyme in the brain called p38 alpha. The aim is to reduce expression of the enzyme in neurons and enable the cells to function better. The drug has proven promising effect in animal tests and it is also currently being tested on patients with Alzheimers Disease.
Study terminated (Due to the long delay because of COVID-19 and results from another study suggesting a higher dose may be more beneficial, EIP Pharma decided on October 15th, 2020, to end the trial prematurely.)
Ages Eligible for Study:
30 Years to 70 Years (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
- Men and women age 30 to 70 years, inclusive.
- Willing and able to provide informed consent.
- Must have genetically confirmed HD and identified cognitive deficits
- Stage 1, as defined by Unified Huntington’s Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) score >10, and,
- CANTAB Paired Associate Learning Total Adjusted Error Score of >16.
- Normal or corrected eye sight and auditory abilities, sufficient to perform all aspects of the cognitive and functional assessments.
- No history of learning difficulties that may interfere with the subject’s ability to complete the cognitive tests.
- A profile of impairment that is not consistent with Huntington’s disease (HD).
- 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.
- 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.
- 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.
- Diagnosis of alcohol or drug abuse within the previous 2 years.
- 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.
- Anemia with a hemoglobin ≤10 g/dL, clinically significant thyroid function abnormality, electrolyte abnormalities.
- 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.
- Known human immunodeficiency virus; or active hepatitis B or hepatitis C virus infection; evidence of active or latent tuberculosis.
- 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.
- History of previous neurosurgery to the brain.
- Female subjects who are pregnant or breast-feeding.
- Male subjects with female partners of child-bearing potential who are unwilling or unable to adhere to contraception requirements specified in the protocol (see Section 5.8).
- Female subjects who have not reached menopause or have not had a hysterectomy or bilateral oophorectomy/salpingo-oophorectomy and are not willing or unable to adhere to contraceptive requirements specified in the protocol (see Section 5.8).
- Requires concomitant use of cytochrome P450 (CYP) 3A4 inhibitors or anti-tumor necrosis factor-alpha therapies during study participation.
- Known allergy to any ingredient of the trial medication or placebo.
John Van Geest Centre for Brain Repair
Address: Cambridge, United Kingdom, CB2 0PY
Tel: 01223 334121