Accelerating Clinical Readiness for Shwachman-Diamond Syndrome
Official title: Accelerating Clinical Trial Readiness for Shwachman Diamond Syndrome
Funded at Boston Children's Hospital · NIH-11494516 · Data updated 2026-11-12
A study focused on speeding up how doctors prepare and enroll patients with Shwachman-Diamond Syndrome for clinical trials and treatment advancements.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $599,999 |
| NIH project number | 1R01FD008823-01 |
| Project ID | NIH-11494516 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals diagnosed with Shwachman-Diamond Syndrome who have or are at risk for hematologic complications and who may contribute to or benefit from registry data and related trials.
✕Less likely to help
Patients not connected to SDS or without involvement in hematologic monitoring or registry activities may not directly benefit.
What this research studies
This project builds on the SDS Registry, a long-term effort that collects medical records and biological samples from people with SDS to create high-quality data. It aims to enhance how this information is used to identify when new treatments or trials might help, including evaluation of when to pursue stem cell transplants. By standardizing data, refining protocols, and understanding disease pathways that lead to myeloid malignancy, the work seeks to make future trials safer and more efficient for SDS patients. Participation would involve sharing medical information and samples in a coordinated, patient-centered registry framework.
Why it matters
Potential benefit: If successful, SDS patients could gain faster access to appropriate clinical trials and earlier opportunities for effective treatments.
How similar studies have gone: Precedents like the SDS Registry have demonstrated that comprehensive, standardized data improves understanding of disease progression and trial readiness.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Shimamura, Akiko — Boston Children's Hospital
- Study coordinator: Shimamura, Akiko
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.