Clinical Readiness to Treat Tuberous Sclerosis–Related Neuropsychiatric Issues
Official title: Clinical Trial Readiness for the Evaluation and Treatment of Neuropsychiatric Manifestations in Tuberous Sclerosis Complex
Funded at Boston Children's Hospital · NIH-11400766 · Data updated 2026-11-12
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.
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 | 2026 |
| Award (this year) | $276,361 |
| NIH project number | 5U54NS092090-12 |
| Project ID | NIH-11400766 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with tuberous sclerosis complex who experience neuropsychiatric or developmental challenges, including autism spectrum features, intellectual disability, sleep or sensory difficulties, may be eligible for related future trials or studies.
✕Less likely to help
Patients without TSC-related neuropsychiatric symptoms or those not involved in future trials may not directly benefit from these readiness activities.
What this research studies
This project focuses on preparing for future therapies, both medicines and non-drug approaches, to help people with TSC who have neuropsychiatric and developmental difficulties. It includes refining a self-administered TSC–associated neuropsychiatric disorder checklist (TAND-SQ) for use in trials, and studying new biomarkers related to autism and intellectual disability within TSC. Researchers will prospectively track psychiatric conditions, mental health symptoms, and psychotropic medication use in people with TSC and TAND, and will explore sleep and sensory processing abnormalities in the TAND phenotype. The work aims to establish trial-ready tools and data to support upcoming clinical studies and potential therapies.
Why it matters
Potential benefit: If successful, the work could accelerate development of effective treatments and support tools for TSC-related neuropsychiatric issues.
How similar studies have gone: Previous efforts have piloted the TAND-SQ and explored biomarkers, showing promise for trial-ready approaches in TSC.
Where this research is happening
-
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.
-
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.
-
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.
-
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.
-
Improving care for autism, learning, sleep, and behavior issues in tuberous sclerosis
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.
-
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.
-
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.