Tracking Information

First Submitted Date ^ICMJE

June 7, 2019

First Posted Date ^ICMJE

June 10, 2019

Last Update Posted Date

July 17, 2019

Actual Study Start Date ^ICMJE

July 8, 2019

Estimated Primary Completion Date

June 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

the effects of administration of neflamapimod on hippocampal function, as assessed in [ Time Frame: 20 Weeks ]

Latency during the learning phase of virtual Morris Water Maze (MWM).l MWM (hidden platform training) during the neflamapimod-treatment period compared to that during the placebo-administration period.

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Current Secondary Outcome Measures ^ICMJE

Additional assessment of hippocampal function by MWM [ Time Frame: 20 Weeks ]
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.
To evaluate the effects of neflamapimod on the Cambridge Neuropsychological Test Automated Battery (CANTAB) paired associates learning task. [ Time Frame: 20 Weeks ]
Number of overall errors in the CANTAB paired associates learning task, in addition to larger battery, during the neflamapimod-treatment period compared to that during the placebo-administration period.
To evaluate tolerability and safety of neflamapimod in subjects with HD. [ Time Frame: 20 Weeks ]
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.

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Pre-specified Outcome Measures

Not Provided

Original Other Pre-specified Outcome Measures

Not Provided

Descriptive Information

Brief Title ^ICMJE

Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease

Official Title ^ICMJE

A Double-Blind, Placebo-Controlled Two-Period 10-Week Treatment Within-Subject Crossover Study Of Cognitive Effects Of Neflamapimod in Early-Stage Huntington Disease (HD)

Brief Summary

This is a double-blind, placebo-controlled 2-period 10-week treatment within-subject crossover study of neflamapimod in early-stage Huntington disease (HD). The primary objective is to determine whether neflamapimod can reverse hippocampal dysfunction in patients with early-stage HD, as assessed by the virtual water-maze-test for evaluating spatial learning and selected tests on the Cambridge Neuropsychological Test Automated Battery (CANTAB).

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase ^ICMJE

Phase 2

Study Design ^ICMJE

Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Condition ^ICMJE

Huntington Disease

Intervention ^ICMJE

Drug: neflamapimod
40 mg neflamapimod capsule

Other Name: VX-745
Other: Placebo
matching placebo capsule

Study Arms ^ICMJE

Experimental: neflamapimod
40 mg hard gelatin capsules, taken twice daily with food.

Intervention: Drug: neflamapimod
Placebo Comparator: placebo
hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.

Intervention: Other: Placebo

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

16

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date ^ICMJE

July 2020

Estimated Primary Completion Date

June 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

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:
1. Stage 1, as defined by Unified Huntington's Disease Rating Scale (UHDRS) Total Functional Capacity (TFC) score >10, and,
2. 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.

Exclusion Criteria:

A profile of impairment that is not consistent with 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.

Sex/Gender ^ICMJE

Sexes Eligible for Study:

All

Ages ^ICMJE

30 Years to 70 Years (Adult, Older Adult)

Accepts Healthy Volunteers ^ICMJE

No

Contacts ^ICMJE

Contact: Jennifer Conway

617-945-0794

jconway@eippharma.com

Listed Location Countries ^ICMJE

United Kingdom

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT03980938

Other Study ID Numbers ^ICMJE

EIP19-NFD-401

Has Data Monitoring Committee

No

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product:	Yes
Studies a U.S. FDA-regulated Device Product:	No

IPD Sharing Statement ^ICMJE

Plan to Share IPD:

No

Responsible Party

EIP Pharma Inc

Study Sponsor ^ICMJE

EIP Pharma Inc

Collaborators ^ICMJE

Voisin Consulting, Inc.

Investigators ^ICMJE

Study Director:

John Alam, MD

EIP Pharma

PRS Account

EIP Pharma Inc

Verification Date

July 2019

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease

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