Improving care for autism, learning, sleep, and behavior issues in tuberous sclerosis
Official title: Clinical Trial Readiness for the Evaluation and Treatment of Neuropsychiatric Manifestations in Tuberous Sclerosis Complex
Funded at Boston Children's Hospital · NIH-11173360 · Data updated 2026-11-12
This project develops user-friendly tools, follows patients over time, and searches for sleep, sensory, and brain-activity markers to help people with tuberous sclerosis complex who have autism, intellectual disability, or other neuropsychiatric challenges.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 30, 2014 |
| Project end | July 31, 2030 |
| Funding year | 2025 |
| Award (this year) | $331,018 |
| NIH project number | 2U54NS092090-11 |
| Project ID | NIH-11173360 on NIH RePORTER |
Who could benefit from this research
✓Could help
People of all ages with a clinical diagnosis of tuberous sclerosis complex who experience autism, intellectual disability, sleep or sensory difficulties, or other neuropsychiatric symptoms are the intended participants.
✕Less likely to help
People who do not have tuberous sclerosis complex or who have no neuropsychiatric concerns are unlikely to receive direct benefit from this project.
What this research studies
From a patient's perspective, the team will refine and validate a self-administered TAND checklist you can use to report thinking, behavior, sleep, and sensory concerns. They will follow people with TSC over time to map mental health symptoms and patterns of psychiatric medication use. The researchers will study sleep problems and sensory processing differences and look for electrophysiological brain markers that relate to autism and intellectual disability in TSC. All of this work is meant to make future drug and behavioral treatment trials for TSC-related neuropsychiatric problems possible and better targeted.
Why it matters
Potential benefit: If successful, the work could speed development of treatments tailored to the thinking, behavior, sleep, and sensory issues faced by people with TSC.
How similar studies have gone: Early pilot work on the TAND checklist and some electrophysiological markers has shown promise, but few large studies have yet produced clear treatment successes for TAND.
Where this research is happening
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Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Krueger, Darcy — Boston Children's Hospital
- Study coordinator: Krueger, Darcy
Related research
Other NIH-funded projects on the same topics.
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VIP Interneurons and Brain Circuits in Tuberous Sclerosis
A study to understand how loss of a brain gene (Tsc1) affects VIP interneurons and the frontal brain region in people and models with Tuberous Sclerosis Complex.
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Clinical Readiness to Treat Tuberous Sclerosis–Related Neuropsychiatric Issues
A large NIH grant aims to ready treatments for the neuropsychiatric and developmental challenges that many people with tuberous sclerosis complex (TSC) face, including autism-like symptoms and sleep or sensory problems.
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Understanding why renal tumors in TSC resist therapy to guide new nano-based treatments
Researchers are studying why kidney tumors in TSC patients resist current drugs, with the goal of designing smarter, more effective treatments that could help people with TSC-related AML.
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Immune causes of kidney disease in tuberous sclerosis
The team is looking at whether immune cells and a protein called B7-H3 help kidney cysts and growths in people with tuberous sclerosis complex.
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Investigating the role of specific proteins in kidney disease related to Tuberous Sclerosis Complex.
This study is looking at how certain proteins affect kidney problems in people with Tuberous Sclerosis Complex, with the hope of finding better treatments that could help improve kidney health and reduce the need for long-term medication.
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Understanding early communication in children with tuberous sclerosis complex and its link to autism.
This study is looking at how babies with tuberous sclerosis complex learn to communicate and how those skills might relate to the severity of autism, by watching videos of their vocal sounds and interactions over time.
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.