Clinical trial NCT00051324: Neurobiological Predictors of Huntington's Disease (PREDICT-HD)
Official title: Neurobiological Predictors of Huntington's Disease Trial
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Huntington Disease
Keywords: Huntington's disease
Huntington disease
HD
Lead sponsor: University of Iowa
Sponsor class: OTHER
Collaborators: name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2003-01-09
Enrollment: 1700
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: People at risk for HD, who have been tested for the HD gene mutation, and who have not been diagnosed with symptoms of HD.

Brief summary: The purpose of this trial is to study early brain and behavioral changes in people who have the gene expansion for Huntington's disease, but are currently healthy and have no symptoms.

Detailed description: Huntington's Disease (HD) is an inherited disease that causes changes in a person's ability to control movements, thinking, and feelings. The intent of this study is to learn more about the beginning changes in thinking skills, emotional regulation, and brain structure and function as a person begins the transition from health to HD.

Preliminary studies indicate that people with HD may have marked decline before an actual diagnosis. This study will help reveal the earliest indicators of the disease and what factors influence the age at which a person carrying the gene develops the disease. It is necessary to get information on the early stages of HD in order to develop drugs that can slow or postpone the onset of HD. The investigators hope this study will provide essential information for future trials of experimental drugs for HD.

During this study, participants will undergo several detailed tests, including MRI scans of the brain, cognitive assessments, physical exams, bio specimen (blood, urine, cerebral spinal fluid) collection and neurological and psychiatric testing.

Eligibility criteria: Inclusion Criteria:

* men and women at risk for HD, who have been tested for the HD gene mutation, and who have not been diagnosed with symptoms of HD (CAG ≥36 for CAG-expanded group or CAG \<36 for CAG-norm group).

Exclusion Criteria:

* diagnosis of manifest HD (at least 50% confidence by neurologist that symptoms are present);
* clinical evidence of unstable medical or psychiatric illness (including substance abuse);
* history of sever learning disability or mental retardation;
* history of other CNS disease or event (e.g., seizures or head trauma);
* current treatment with antipsychotic medications, including the traditional neuroleptics such as haloperidol as well as the atypical antipsychotics risperidone, clozapine, quetiapine, and olanzapine;
* treatment with phenothiazine-derivative antiemetic medications such as prochlorperazine, metoclopramide, promethazine, and Inapsine on a regular basis (greater than 3 times per month);

Specific exclusion criteria for the lumbar puncture:

* Current use of anti-coagulants
* Current use of anti-platelets
* Unable to provide consent for him/herself

Primary outcomes:
measure: Refine the prediction of disease diagnosis (motor conversion)
timeFrame: One year
measure: Characterize disease progression prior to diagnosis.
timeFrame: One year
measure: Establish possible validity and reliability of disease measures.
timeFrame: One year
Secondary outcomes:
Other outcomes:
measure: Cerebral spinal fluid containing unique biomarker signatures.
timeFrame: One year
measure: Cerebral spinal fluid biomarker changes correlating with HD progression.
timeFrame: One year
Locations:
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
Study officials:
affiliation: University of Iowa
name: Jane S. Paulsen, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Paulsen JS, Lourens S, Kieburtz K, Zhang Y. Sample enrichment for clinical trials to show delay of onset in huntington disease. Mov Disord. 2019 Feb;34(2):274-280. doi: 10.1002/mds.27595. Epub 2019 Jan 14.
citation: Reed ER, Latourelle JC, Bockholt JH, Bregu J, Smock J, Paulsen JS, Myers RH; PREDICT-HD CSF ancillary study investigators. MicroRNAs in CSF as prodromal biomarkers for Huntington disease in the PREDICT-HD study. Neurology. 2018 Jan 23;90(4):e264-e272. doi: 10.1212/WNL.0000000000004844. Epub 2017 Dec 27.
citation: Epping EA, Kim JI, Craufurd D, Brashers-Krug TM, Anderson KE, McCusker E, Luther J, Long JD, Paulsen JS; PREDICT-HD Investigators and Coordinators of the Huntington Study Group. Longitudinal Psychiatric Symptoms in Prodromal Huntington's Disease: A Decade of Data. Am J Psychiatry. 2016 Feb 1;173(2):184-92. doi: 10.1176/appi.ajp.2015.14121551. Epub 2015 Oct 16.