# Planning a childhood sJOgren disease Use of Randomized N-of-1 Evaluation of therapY JOURNEY

> **NIH NIH R34** · CASE WESTERN RESERVE UNIVERSITY · 2024 · $297,940

## Abstract

Project Summary/Abstract:
Childhood Sjögren's disease (cSD) is a rare autoimmune disease that primarily affects the
glands, leading to salivary and lacrimal gland inflammation that results in reduced function and
dryness symptoms. Active inflammation of the salivary glands is one of the most common
symptoms in children with cSD. Despite the profound impact on affected children and their
families, there is no pediatric-specific clinical trials to inform therapeutic strategies in cSD.
Although symptomatic episodes of inflammation to the glands are critical to evaluate drug
therapies, how to determine whether a patient has experienced a flare is lacking. There are no
widely accepted cSD outcome measures to tell us whether a treatment is effective, especially
with regards to flares involving the glands. New medications have the potential to be safe and
effective treatments for cSD but they must be evaluated in trials. We propose a novel trial
design known as N-of-1 trials to evaluate cSD therapies. N-of-1 trials is a personalized
treatment strategy based on the individual patient's response. The goal of this grant will be to
successfully finalize a rigorous and impactful clinical N-of-1 trial in cSD. We will accomplish
this through three aims. We will develop patient- and parent-driven outcome measures for the
evaluation of glandular inflammation and its flares, and their impact on quality of life and
mental health for cSD clinical trials. The overall approach for this aim is based off frameworks
successfully used previously in pediatric rheumatology. To reach consensus on cSD flare criteria
and to develop patient-important cSD outcome measures, we will review the literature, conduct
interviews, perform online surveys, and plan a final group meeting, of stakeholders including
patients, health professionals, and methodologists. We will prepare and finalize the details for
N-of-1 trials in cSD using two therapies. To accomplish this aim our team will develop a study
protocol with patient involvement on critical steps of the trials such as the final choice of
medications or outcome measures. We will establish a collaborative network of pediatric
rheumatology centers for participation in the N-of-1 trial that we will test as a model for current
and future studies in cSD.

## Key facts

- **NIH application ID:** 10880455
- **Project number:** 1R34AR084192-01
- **Recipient organization:** CASE WESTERN RESERVE UNIVERSITY
- **Principal Investigator:** NORA SINGER
- **Activity code:** R34 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $297,940
- **Award type:** 1
- **Project period:** 2024-08-15 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10880455

## Citation

> US National Institutes of Health, RePORTER application 10880455, Planning a childhood sJOgren disease Use of Randomized N-of-1 Evaluation of therapY JOURNEY (1R34AR084192-01). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10880455. Licensed CC0.

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