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Tuberous Sclerosis clinical trials

The registry lists 115 studies for Tuberous Sclerosis across more than 10 countries. 27 are recruiting or about to start. 74 of them are mainly about Tuberous Sclerosis (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.

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Status

Completed
56
Recruiting
21
Active, not recruiting
11
Status unknown
9
Terminated
8
Withdrawn
4

Phase

Phase 2
35
Phase 3
20
Phase 1
16
Phase 4
6

Where

United States
63
Australia
18
United Kingdom
16
France
14
Canada
13
Italy
13

Who runs them

Novartis Pharmaceuticals (7) · Children's Hospital Medical Center, Cincinnati (7) · Jazz Pharmaceuticals (6) · University of California, Los Angeles (5) · National Heart, Lung, and Blood Institute (NHLBI) (5) · Marinus Pharmaceuticals (3) · Nobelpharma (3) · GRIN Therapeutics, Inc. (3)

What is being tested here

Drugs under test in Tuberous Sclerosis studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Sirolimus1312
Vigabatrin113
GDC-0084101
Sertraline101
Everolimus0516
Cannabidiol035
Ganaxolone023
Radiprodil002
Bexicaserin002
Topical Sirolimus022

Drug classes tested here mTOR inhibitors 1 open

Coming up

4phase 3 studies under way
13studies finish main data collection within a year1 of them phase 3

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

The 10 nearest, phase 3 first

Who these studies take

Of the 21 Tuberous Sclerosis studies recruiting now, 10 (48%) set no upper age limit and 8 (38%) stop at 75 or younger. 15 (71%) take children under 18. 1 (5%) are for women only and 0 (0%) for men only; 2 (10%) take healthy volunteers.

Recruiting studies that take each age, of 21
AgeWomenMen
1012 (57%)11 (52%)
1818 (86%)17 (81%)
3017 (81%)16 (76%)
4016 (76%)15 (71%)
5016 (76%)15 (71%)
6515 (71%)14 (67%)
7513 (62%)12 (57%)
8513 (62%)12 (57%)

Across all 115 studies, recruiting or not: 58% stop at 75 or younger and 57% at 65; 77% take children; 5% are for women only and 1% for men only; 10% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 50 of 115. Browse all 115 → or search them.