Condition

Tuberous Sclerosis clinical trials and research

• 10Clinical trials
• 12NIH research projects
• 5Countries

Newest Tuberous Sclerosis clinical trials

All recruiting trials (10)

• Understanding genetic causes of neuroendocrine tumors in Mexican patients

  This study is trying to find out the genetic changes that cause neuroendocrine tumors in Mexican patients to help develop better treatments.

  Recruiting at 3 sites Universidad Nacional Autonoma de Mexico · Mexico City, Mexico City and 2 other locations · NCT06523582

• Understanding reproductive counseling for patients with skin genetic disorders

  This study is trying to understand how people with skin genetic disorders and their partners make decisions about having children, while also looking at what doctors know and think about these choices.

  Recruiting Maastricht University Medical Center · Maastricht, Limburg · NCT06330350

• Comparing Sertraline and Placebo for Treating Anxiety in Children with Neurodevelopmental Disorders

  This study is testing if the medication sertraline can help reduce anxiety in children and teens aged 8-17 with neurodevelopmental disorders like autism and Fragile X syndrome compared to a placebo.

  Recruiting at 6 sites Phase 2 Holland Bloorview Kids Rehabilitation Hospital · Calgary, Alberta and 6 other locations · NCT06081348

• National Registry for Rare Kidney Diseases

  This study is collecting health information from people with rare kidney diseases to help improve treatments and create better care guidelines.

  Recruiting UK Kidney Association · Bristol, South West · NCT06065852

• Collection of biosamples and clinical data for tuberous sclerosis and lymphangioleiomyomatosis

  This study is collecting blood and tissue samples from people with tuberous sclerosis and lymphangioleiomyomatosis to help researchers learn more about these conditions and how they affect the body over time.

  Recruiting at 25 sites National Tuberous Sclerosis Association · Birmingham, Alabama and 25 other locations · NCT05676099

• Observing the health management of patients with rare genetic syndromes

  This study looks at how patients with rare genetic syndromes like Prader-Willi and Klinefelter manage their health as they move from children's care to adult care to see what support they need.

  Recruiting Erasmus Medical Center · Rotterdam, South Holland · NCT04463316

• Investigating genetic factors in childhood cancer

  This study is trying to find out if certain genes passed down in families can increase the risk of childhood cancer by looking at samples from kids who have had cancer and their relatives.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT03050268

• Investigating skin tumors in patients with tuberous sclerosis

  This study is testing how skin tumors in adults with tuberous sclerosis develop by looking at their medical history and examining their skin to understand what causes these tumors.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT00001975

• Exploring genetic factors in lung disease development

  This study is trying to find out how certain genes might play a role in lung diseases like cystic fibrosis and asthma by looking at lung cells from both patients and healthy people.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland and 1 other locations · NCT00001532

• Understanding the disease process of lymphangioleiomyomatosis

  This study looks at how lymphangioleiomyomatosis affects the lungs in women and tries to find out what causes the disease to help develop better treatments.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT00001465

NIH-funded research (12)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• 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.

  NIH research Henry Ford Health + Michigan State University Health Sciences · East Lansing, United States · NIH-11461450

• 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.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11400766

• 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.

  NIH research Brigham and Women's Hospital · Boston, United States · NIH-11345874

• 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.

  NIH research Brigham and Women's Hospital · Boston, United States · NIH-11324582

• 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.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11173360

• 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.

  NIH research Brigham and Women's Hospital · Boston, United States · NIH-11118807

• 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.

  NIH research University of Tennessee Health Sci Ctr · Memphis, United States · NIH-11105881

• Understanding how immune cells affect kidney disease in Tuberous Sclerosis Complex

  This study is looking at how certain immune cells might be affecting kidney problems in people with Tuberous Sclerosis Complex, and it aims to find new ways to help improve kidney health for those living with this condition.

  NIH research Brigham and Women's Hospital · Boston, United States · NIH-11101408

• Understanding how kidney tumors in Tuberous Sclerosis Complex resist treatment to create new therapies

  This study is looking at how certain kidney tumors in people with Tuberous Sclerosis Complex resist treatment, so researchers can learn more about them and find better ways to help patients.

  NIH research Brigham and Women's Hospital · Boston, United States · NIH-11093992

• Understanding how neuron cilia dysfunction affects Tuberous Sclerosis Complex

  This study is looking at how tiny hair-like structures in brain cells, called primary cilia, might be linked to the brain problems caused by Tuberous Sclerosis Complex, which can lead to issues like epilepsy and autism, with the hope of finding new ways to help improve treatment for those affected.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11071164

• Investigating a new approach to treat epilepsy in patients with tuberous sclerosis complex

  This study is looking at how a specific protein in the brain affects seizure control in people with tuberous sclerosis complex (TSC), with the hope of finding new treatments to help manage seizures better.

  NIH research Washington University · Saint Louis, United States · NIH-11051253

• New treatment approach for Pulmonary Lymphangioleiomyomatosis and Tuberous Sclerosis Complex

  This study is testing a new combination treatment for people with Pulmonary LAM and Tuberous Sclerosis Complex to see if it can help manage their symptoms and improve their quality of life while being safer than current options.

  NIH research Biosputnik LLC · New York, United States · NIH-10697901

Related topics

Quick answers

How many Tuberous Sclerosis clinical trials are recruiting right now?

10, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 5 countries. The most sites are in United States (5 trials), Canada (2 trials), Netherlands (2 trials), United Kingdom (1 trial), Mexico (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

Describe your situation →