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 Tuberous sclerosis (TS) is a rare, multi-system genetic disease that causes benign tumors to grow in the brain and on other vital organs such as the kidneys, heart, eyes, lungs, and skin. It commonly affects the central nervous system and results in a combination of symptoms including seizures, developmental delays, behavioral problems, skin abnormalities, and kidney disease.
Tuberous sclerosis affects as many as 25,000 to 40,000 individuals in the United States and about 1 to 2 million individuals worldwide, with an estimated prevalence of one in 6,000 newborns affected by the disease each year. TS occurs in all races and ethnic groups, and in both genders. (1)
 Developing new treatments to treat both the disease and symptoms of tuberous sclerosis is a long and complex process, but an essential one in finding new and innovative treatments. Without new clinical research studies, new medicines would not be made available to patients. (2)
In our quest to find novel and effective treatments for TS , Novartis will be conducting several new clinical trials for patients diagnosed with tuberous sclerosis. Find out more about the Novartis tuberous sclerosis clinical trials below:
EXIST-2
Tuberous Sclerosis Clinical Trial CRAD001M2302
The goal of this phase 3 clinical research study is to learn if a Novartis investigational drug can shrink or slow the growth of kidney lesions, known as angiomyolipoma in patients with tuberous sclerosis (TS) or lymphangioleiomyomatosis (LAM). An “investigational drug” is a drug that is being tested and is not approved for sale in the United States by the U.S. Food and Drug Administration (FDA) for the indication being studied. This investigational drug may not become commercially available.
Angiomyolipomas are benign growths consisting of fatty tissue and muscle cells. These growths are seen in the majority of tuberous sclerosis patients. In most patients, these growths display no outward systems, however, pain in hip or side, enlarged kidneys, blood in the urine, and loss of kidney function can all occur.
Participants enrolling in this tuberous sclerosis research study will be required to visit the study center one time every 2 weeks during the first 2 months, then one time every 4-6 weeks until week 24. After week 24, visits will occur once every 12 weeks until the study is completed.
Participants may be eligible to enroll in the EXIST-2 clinical trial if they:
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Are 18 years of age or older
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Have been diagnosed with tuberous sclerosis or lymphangioleiomyomatosis (LAM)
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Have evidence of at least one angiomyolipoma ≥ 3cm in its longest diameter
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Have not had organ transplant surgery
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Do not suffer from severe kidney, liver, or lung problems
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Do not suffer from uncontrolled diabetes or high cholesterol
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Are not currently pregnant or breast feeding
Travel assistance may be available.
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For U.S. residents only.
Non-U.S. residents
