NCT00051324

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
1,700

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Aug 2002

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2002

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

January 8, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 9, 2003

Completed
22.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2025

Completed
Last Updated

January 14, 2025

Status Verified

January 1, 2025

Enrollment Period

22.9 years

First QC Date

January 8, 2003

Last Update Submit

January 10, 2025

Conditions

Huntington Disease

Keywords

Huntington's diseaseHuntington diseaseHD

Outcome Measures

Primary Outcomes (3)

  • Refine the prediction of disease diagnosis (motor conversion)

    HD diagnosis will be better predicted by adding longitudinal change to the baseline measures of striatal and white matter volumes, tone-paced and speeded tapping score, stroop interference and motor score.

    One year

  • Characterize disease progression prior to diagnosis.

    Document change scores for each marker using its slope. Comparisons of change rates across time will suggest measures best suited to clinical trials by large effect sizes and low variability.

    One year

  • Establish possible validity and reliability of disease measures.

    This will require that we continuously analyze recently collected data, remove items that are insensitive, and add new items to be tested throughout the course of the study. The power and sensitivity of future multi-site trials and studies depend on accurate measures of marker validity. HD diagnosis will be better predicted by UHDRS total motor score following new standardized reliability training and by the tapping task under modified more challenging, conditions. Psychiatric and functional ratings will be improved with item response analyses and dynamic piloting of item edits to establish the most psychometrically sound items for clinical trials.

    One year

Other Outcomes (2)

  • Cerebral spinal fluid containing unique biomarker signatures.

    One year

  • Cerebral spinal fluid biomarker changes correlating with HD progression.

    One year

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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).

You may not qualify if:

  • 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);
  • Current use of anti-coagulants
  • Current use of anti-platelets
  • Unable to provide consent for him/herself

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Related Publications (4)

  • Chelsky D, Joyce C, Bockholt HJ, Rudnick PA, Adams WH, McAllister F, Smock JW, Newton MA, Paulsen JS. Discovery and Targeted Proteomic Studies Reveal Striatal Markers Validated for Huntington's Disease. Ann Clin Transl Neurol. 2025 Dec 9. doi: 10.1002/acn3.70272. Online ahead of print.

    PMID: 41363816DERIVED

  • 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.

    PMID: 30644132DERIVED

  • 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.

    PMID: 29282329DERIVED

  • 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.

    PMID: 26472629DERIVED

Related Links

Biospecimen

Retention: SAMPLES WITH DNA

Cerebral spinal fluid acquired and retained since 2012. Urine, plasma and cell lines to be acquired and retained since study initiation 2002.

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Study Officials

  • Jane S. Paulsen, Ph.D.

    University of Iowa

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

January 8, 2003

First Posted

January 9, 2003

Study Start

August 1, 2002

Primary Completion

June 30, 2025

Study Completion

June 30, 2025

Last Updated

January 14, 2025

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will share

All data have been de-identified and anonymized with the best possible methods to allow and facilitate data sharing with other investigators. Data has been shared to CHDI and dbGaP.

Shared Documents
STUDY PROTOCOL, CSR
Time Frame
Data will be on dbGaP and at CHDI for data sharing with no anticipated endpoint.
Access Criteria
NINDS monitors dbGaP and CHDI scientific staff monitors their data copy.

Locations

US(1)