# Preparing for preventive clinical trials in Huntington’s disease

> **NIH NIH U01** · UNIVERSITY OF WISCONSIN-MADISON · 2021 · $632,312

## Abstract

Project Summary/Abstract
Our group completed the natural history study of premanifest Huntington disease (HD) entitled Predict-HD
which evaluated over 1400 research volunteers who were healthy but had undergone the predictive test for
the gene that causes HD. Findings revealed that signs and symptoms of HD were evident up to 15 years
before the traditional diagnosis of HD was given in the clinic. From these data we were able to develop
models of prognosis, disease progression and prediction of HD onset. Disease-modifying clinical trials are
currently underway to slow the progression, or delay the onset, of HD. More recently, a collaborative group
published an assay to measure the amount of mutant huntingtin protein in the cerebral spinal fluid of HD
participants. Questions of central importance to the success of this measure for clinical trials require
investigation: (1) how reliable is the measure in the same person when repeated (intra-subject test-retest
reliability); (2) how reliable is the measure in the same person when analyzed by two different labs (inter-lab
reliability); (3) does the measure reflect disease symptoms (content validity); (4) does the measure predict
meaningful disease outcomes (prognostic validity); (5) does the measure track disease progression or
severity; and (6) how many (and what stage of HD) research subjects do we need to know with confidence
that an intervention is working (i.e., delaying onset/slowing progression)? The proposed research study will
address all of these limitations to more effectively test new experimental interventions such as gene therapies
and new drugs. Findings will immediately inform how the field should best design preventive clinical trials for
HD.

## Key facts

- **NIH application ID:** 10213855
- **Project number:** 5U01NS105509-04
- **Recipient organization:** UNIVERSITY OF WISCONSIN-MADISON
- **Principal Investigator:** Jane S Paulsen
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $632,312
- **Award type:** 5
- **Project period:** 2018-03-15 → 2024-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10213855

## Citation

> US National Institutes of Health, RePORTER application 10213855, Preparing for preventive clinical trials in Huntington’s disease (5U01NS105509-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10213855. Licensed CC0.

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