# Advancing the Diagnosis and Treatment of Rare Primary Immune Disorders

> **NIH NIH U54** · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · 2020 · $22,787

## Abstract

PIDTC PILOT/FEASIBILITY CORE
SUMMARY/ABSTRACT
The Primary Immune Deficiency Treatment Consortium (PIDTC) was established in 2009 as a collaboration
among North American centers with expertise in treating infants and children with rare, but serious primary
immunodeficiencies (PIDs). In this application, we propose a clinical research project examining a group of
newly described disorders, the Primary Immune Regulatory Disorders (PIRD, Project 6906), which extends the
scope of PIDTC to encompass genetic disorders affecting immune regulation. The Pilot/Feasibility Core has
funded 7 projects in its lifetime, 4 in the past cycle. These annual $50,000 awards are granted after rigorous
review for scientific merit, prioritizing projects that have the potential to impact the field, leverage data and
infrastructure of the RDCRN and PIDTC, and have a high likelihood of translation to clinical trials. Investigators
wishing to receive a 2nd year of funding are reviewed in a competitive renewal process that puts emphasis on
demonstration of meeting milestones stated in the application. In this application, we plan to continue our
successful and productive Pilot program, transitioning the leadership to Dr. Sung-Yun Pai, to fulfill Specific Aim
1: To select and fund timely innovative research in PID and its treatment which in turn results in tangible
outcomes that advance the goals of PIDTC and fuel clinical trial readiness. Notwithstanding our successes to
date, we have recognized that implementation of funded Pilot studies using data and samples acquired in the
context of the clinical research projects has been slow. For future Pilot projects, we will pursue Specific Aim 2:
To use a PIDTC-specific, consortium-wide, biological sampling and sharing protocol to facilitate rapid
implementation of Pilot/Feasibility Core supported studies and other studies relying on samples from patients.
Through these specific aims, we hope to broaden the success we have had leveraging clinical research
protocol and Pilot project data in SCID to develop and launch an evidence-based clinical trial in SCID
(Conditioning SCID Infants Diagnosed Early, CSIDE study, NCT03619551) to other PID and PIRD.

## Key facts

- **NIH application ID:** 10018654
- **Project number:** 5U54AI082973-12
- **Recipient organization:** UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- **Principal Investigator:** SUNG-YUN PAI
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $22,787
- **Award type:** 5
- **Project period:** 2009-09-12 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10018654

## Citation

> US National Institutes of Health, RePORTER application 10018654, Advancing the Diagnosis and Treatment of Rare Primary Immune Disorders (5U54AI082973-12). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10018654. Licensed CC0.

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