# A Clinical Trial Readiness Study of Patient Reported Outcome Measures in Thrombotic Thrombocytopenic Purpura (TTP)

> **NIH NIH R21** · DUKE UNIVERSITY · 2024 · $456,348

## Abstract

PROJECT SUMMARY/ABSTRACT
 With a prevalence of 10 cases per million, immune thrombotic thrombocytopenic purpura (iTTP) is
classified as a rare disease. Yet, it has significant human and economic impacts – untreated mortality rates of
90%; disproportionate effects on women and minorities; and per-patient costs > $1 million. Adding to these
impacts, patients with iTTP report that their most significant health-related quality of life impact is cognitive
function. Yet, despite promising therapeutic candidates (anfibatide and TAK-755) in early phase clinical trials,
cognitive function impacts cannot be accurately measured because a comprehensive assessment of aspects of
cognitive function influenced by iTTP has not been conducted. Though used in iTTP clinical trials, available
cognitive measures lack sufficient validity evidence. Thus, no validity evidence is insufficient for clinical outcome
assessments (COAs) of cognitive function for the context of use of iTTP clinical trials. Without validity evidence
for COAs, iTTP clinical trials cannot align with FDA recommendations for patient-focused drug development:
They cannot incorporate outcomes that are important to patients. Even worse, patients’ cognitive function
response to new therapies cannot be appropriately assessed. Thus, patients cannot be referred for treatments
that improve cognitive function outcomes. Thus, the absence of validity evidence for COAs of cognitive function
is a critical barrier to iTTP clinical trial readiness. To facilitate clinical trial readiness in outcomes that are
important to patients, there is an urgent need to develop a measurement strategy for iTTP-associated cognitive
impairment.
 The objective of the study is to develop a consensus-driven measurement strategy of cognitive function
for iTTP clinical trials. To this end, the study will both determine iTTP impacts on cognitive function and develop
a cognitive function measurement strategy. To this end, the study will use a sequential mixed methods approach.
First, using qualitative interviews of 24 patient-caregiver dyads, the study will develop an iTTP-induced cognitive
impairment draft conceptual model. Second, using both a literature review and a multistakeholder advisory panel,
a consensus-based approach will be used to select cognitive function COAs that have sufficient evidence for
being fit-for purpose. The study will benefit from robust recruitment at 3 participating sites within the National
Patient-Centered Clinical Research Network – PCORNnet®. The study team has expertise in iTTP, neurogenic
disorders, and COA measurement strategy.
 A boon to iTTP clinical trial readiness, successful completion of this study will support the use of cognitive
function COAs that are psychometrically rigorous. Furthermore, because they will be made available in the public
domain, the conceptual and measurement models from this study will be used to further iTTP clinical trials: They
can be accessed by both industry and regula...

## Key facts

- **NIH application ID:** 10947615
- **Project number:** 1R21TR004694-01A1
- **Recipient organization:** DUKE UNIVERSITY
- **Principal Investigator:** Theresa Coles
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $456,348
- **Award type:** 1
- **Project period:** 2024-08-15 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10947615

## Citation

> US National Institutes of Health, RePORTER application 10947615, A Clinical Trial Readiness Study of Patient Reported Outcome Measures in Thrombotic Thrombocytopenic Purpura (TTP) (1R21TR004694-01A1). Retrieved via AI Analytics 2026-06-12 from https://api.ai-analytics.org/grant/nih/10947615. Licensed CC0.

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