Clinical Trials Browser

Creatine deficiency, X-linked, Telomeric 22q13 Monosomy Syndrome, Chromosome 15q, partial deletion, Fragile X Syndrome, Hamartoma Syndrome, Multiple, Tuberous Sclerosis, Rett Syndrome, Angelman Syndrome

NCT05104983 2026-01-22

TSC-STEPS

Phase 2 Recruiting

64 enrolled

Epilepsy, Tuberous Sclerosis

NCT05467397 2026-01-21

Feasibility of [11C]Acetate-PET in LAM and TSC

Phase 1/2 Active not recruiting

7 enrolled 6 charts

Tuberous Sclerosis, Lymphangioleiomyomatosis

NCT07287202 2025-12-23

Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)

Phase 1/2 Not yet recruiting

15 enrolled

Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly

NCT05626634 2025-12-03

Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

Phase 2 Completed

41 enrolled

CDKL5 deficiency disorder, Epilepsy, Epilepsies, Myoclonic, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT03363763 2025-12-01

Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex

Phase 2 Terminated

24 enrolled 11 charts

Erythema, Exanthema, Fibroma, Tuberous Sclerosis, Facies

NCT06965881 2025-11-21

Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults

Phase 1 Terminated

30 enrolled

Focal Cortical Dysplasia, Tuberous Sclerosis

NCT06975605 2025-11-18

Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.

Phase 1 Completed

18 enrolled

Focal Cortical Dysplasia, Tuberous Sclerosis

NCT04485104 2025-10-31

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Phase 3 Terminated

3 enrolled 28 charts

Tuberous Sclerosis 1, Tuberous Sclerosis 2, Epilepsy, Epilepsies, Myoclonic, Seizures, Spasms, Infantile, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT05604170 2025-10-01

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Phase 3 Terminated

117 enrolled 11 charts

Tuberous Sclerosis

NCT06879665 2025-09-22

TANDem-2

Phase NA Enrolling by invitation

30 enrolled

Tuberous Sclerosis

NCT03836300 2025-07-31

Parent and Infant Inter(X)Action Intervention (PIXI)

Phase NA Enrolling by invitation

120 enrolled

Telomeric 22q13 Monosomy Syndrome, Developmental Disabilities, DiGeorge Syndrome, Down Syndrome, Fragile X Syndrome, Klinefelter Syndrome, Prader-Willi Syndrome, Tuberous Sclerosis, Turner Syndrome, Rett Syndrome, Angelman Syndrome, Williams Syndrome, Muscular Dystrophy, Duchenne, Smith-Magenis Syndrome

NCT06081348 2025-07-16

CALM

Phase 2 Recruiting

130 enrolled

Autism Spectrum Disorder, Anxiety Disorders, Attention Deficit Disorder with Hyperactivity, Autistic Disorder, DiGeorge Syndrome, Fragile X Syndrome, Tourette Syndrome, Tic Disorders, Tuberous Sclerosis, 22q11 Deletion Syndrome, Neurodevelopmental Disorders

NCT05323734 2025-07-11

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

Phase 3 Completed

129 enrolled 13 charts

Tuberous Sclerosis

NCT05059327 2025-06-04

Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC

Phase 2 Completed

61 enrolled

Seizures, Tuberous Sclerosis

NCT05495425 2024-11-22

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC

Phase 3 Completed

43 enrolled

Tuberous Sclerosis

NCT05364021 2024-11-08

PACIFIC

Phase 1/2 Completed

52 enrolled

CDKL5 deficiency disorder, Epilepsy, Epilepsies, Myoclonic, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT05286632 2024-11-06

KidneYou - Innovative Digital Therapy

Phase NA Completed

210 enrolled

Genitourinary

Nephronophthisis, familial juvenile, Treatment Adherence and Compliance, Albuminuria, Diabetes Mellitus, Hypertension, Polycystic Kidney Diseases, Tuberous Sclerosis, Renal Insufficiency, Chronic, Kidney Diseases, Cystic

NCT04698538 2024-10-30

TRAIN

Phase NA Completed

54 enrolled

Tuberous Sclerosis

NCT02849457 2024-08-19

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

Phase 2 Completed

84 enrolled 16 charts

Tuberous Sclerosis

NCT04595513 2024-05-16

STOP2

Phase 1/2 Completed

5 enrolled 15 charts

Epilepsy, Tuberous Sclerosis

NCT01906866 2024-04-23

Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities

Phase 3 Completed

125 enrolled 22 charts

Autism Spectrum Disorder, Sleep Wake Disorders, Tuberous Sclerosis, Angelman Syndrome, Parasomnias, Smith-Magenis Syndrome

NCT05867576 2024-02-14

ACT in TSC

Phase NA Unknown

15 enrolled

Psychological Well-Being, Tuberous Sclerosis

NCT04987463 2024-02-08

ViRap

Phase 2/3 Unknown

60 enrolled

Epilepsy, Neoplasms, Tuberous Sclerosis

NCT04344626 2023-10-10

Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery

Phase NA Withdrawn

CNS

Focal Cortical Dysplasia, Brain Neoplasms, Encephalitis, Epilepsy, Gliosis, Sturge-Weber Syndrome, Tuberous Sclerosis, Stroke, Hemimegalencephaly, Polymicrogyria

NCT03826628 2023-09-08

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Phase 2/3 Completed

107 enrolled 24 charts

Tuberous Sclerosis

NCT05534672 2023-08-08

RaRE-TS

Phase 3 Recruiting

200 enrolled

Drug Resistant Epilepsy, Epilepsy, Tuberous Sclerosis

NCT04285346 2023-04-04

TSC

Phase 2 Completed

23 enrolled 9 charts

Tuberous Sclerosis

NCT02544763 2022-09-28

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Phase 3 Completed

224 enrolled 18 charts

Seizures, Tuberous Sclerosis

NCT02544750 2022-07-14

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Phase 3 Completed

199 enrolled 14 charts

Seizures, Tuberous Sclerosis

NCT00126672 2022-04-25

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

Phase 2 Completed

36 enrolled 8 charts

Neoplasms, Tuberous Sclerosis, Angiomyolipoma

NCT03525834 2021-09-09

Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.

Phase 4 Completed

40 enrolled 10 charts

Tuberous Sclerosis, Lymphangioleiomyomatosis, Angiomyolipoma

NCT02451696 2021-09-01

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

Phase 2 Completed

15 enrolled 9 charts

Focal Cortical Dysplasia, Epilepsy, Tuberous Sclerosis

NCT02860494 2021-05-03

EVEROST

Phase 2/3 Withdrawn

Tuberous Sclerosis

NCT01929642 2021-03-29

RAPT

Phase 2 Completed

3 enrolled 10 charts

Autistic Disorder, Tuberous Sclerosis, Self-Injurious Behavior

NCT03650452 2021-02-18

ELEKTRA

Phase 2 Completed

141 enrolled 24 charts

CNS

CDKL5 deficiency disorder, Brain Diseases, Central Nervous System Diseases, Epilepsy, Epilepsies, Myoclonic, Nervous System Diseases, Seizures, Spasms, Infantile, Tuberous Sclerosis, Hypoxia-Ischemia, Brain, Malformations of Cortical Development, Lennox Gastaut Syndrome

NCT03422367 2020-11-06

JETS

Phase NA Unknown

64 enrolled

Autistic Disorder, Child Development Disorders, Pervasive, Epilepsy, Tuberous Sclerosis

NCT01526356 2020-10-26

Treatment

Phase 2 Completed

179 enrolled 13 charts

Tuberous Sclerosis, Angiofibroma

NCT02201212 2020-09-16

Everolimus for Cancer With TSC1 or TSC2 Mutation

Phase 2 Completed

30 enrolled 12 charts

Tuberous Sclerosis

NCT02061397 2020-08-25

SOS

Phase 1/2 Completed

10 enrolled 6 charts

Tuberous Sclerosis, Lymphangioleiomyomatosis

Clinical Trials

STBETA

A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) Guidelines

EpiCom

Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)

Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

RAINBOW

Spectrum

Sapu003 in Advanced mTOR-sensitive Solid Tumors

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

TSC-STEPS

Feasibility of [11C]Acetate-PET in LAM and TSC

Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)

Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex

Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults

Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

TANDem-2

Parent and Infant Inter(X)Action Intervention (PIXI)

CALM

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC

PACIFIC

KidneYou - Innovative Digital Therapy

TRAIN

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

STOP2

Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities

ACT in TSC

ViRap

Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

RaRE-TS

TSC

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

EVEROST

RAPT

ELEKTRA

JETS

Treatment

Everolimus for Cancer With TSC1 or TSC2 Mutation

SOS