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Showing 1–24 of 78 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hamartoma Syndrome, Multiple Clear all
Completed No phase listed Observational

Official Record of Patients Diagnosed With Lymphangioleiomyomatosis (LAM)

NCT00001869
View directory record Official record
Conditions
Leiomyomatosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 17, 2026, 9:11 AM EDT
Terminated Phase 3 Interventional Results available

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

NCT04485104
View directory record Official record
Conditions
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Month to 23 Months
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
6
States / cities
Little Rock, Arkansas • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome

NCT02991807
View directory record Official record
Conditions
PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome
Interventions
RAD001, Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
5 Years to 45 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 16, 2025 · Synced Sep 17, 2026, 9:11 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study of Individuals With Autism and Germline Heterozygous PTEN Mutations

NCT02461446
View directory record Official record
Conditions
PTEN, ASD, Autism, Macrocephaly, PTEN Hamartoma Tumor Syndrome
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Months and older
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
5
States / cities
Los Angeles, California • Stanford, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 2 Interventional Results available

Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes

NCT00971789
View directory record Official record
Conditions
Cowden's Disease, Hamartoma Syndrome, Multiple
Interventions
fludeoxyglucose F 18, sirolimus, Clinical Videography
Radiation · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 29, 2015 · Synced Sep 17, 2026, 9:11 AM EDT
Recruiting No phase listed Observational

TSC Biosample Repository and Natural History Database

NCT05676099
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Phlebotomy, Buccal (cheek) swab, Genetic Testing, Tissue donation after routine clinical procedure
Procedure · Genetic · Other
Lead sponsor
National Tuberous Sclerosis Association
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2050
U.S. locations
24
States / cities
Birmingham, Alabama • Loma Linda, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Sep 17, 2026, 9:11 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

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

NCT03836300
View directory record Official record
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 2Phase 3 Interventional Results available

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

NCT03826628
View directory record Official record
Conditions
Facial Angiofibroma, Tuberous Sclerosis
Interventions
rapamycin, placebo
Drug
Lead sponsor
Dermatology Specialties Limited Partnership
Industry
Eligibility
6 Years to 65 Years
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
6
States / cities
Phoenix, Arizona • La Jolla, California • St. Petersburg, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1Phase 2 Interventional

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

NCT00792766
View directory record Official record
Conditions
Tuberous Sclerosis, Angiolipoma
Interventions
everolimus (RAD001)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 17, 2013 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety of Simvastatin in LAM and TSC

NCT02061397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
Simvastatin, Sirolimus Oral Product, Everolimus Oral Product
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 24, 2020 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 2 Interventional Results available

Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)

NCT04285346
View directory record Official record
Conditions
Tuberous Sclerosis
Interventions
Ganaxolone
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2023 · Synced Sep 17, 2026, 9:11 AM EDT
Recruiting No phase listed Observational

Study of the Disease Process of Lymphangioleiomyomatosis

NCT00001465
View directory record Official record
Conditions
Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Toshibia Aquilion One CT
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
16 Years to 100 Years
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

NCT00789828
View directory record Official record
Conditions
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions
Everolimus, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
12
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2016 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 2 Interventional Results available

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

NCT00126672
View directory record Official record
Conditions
Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma
Interventions
Rapamycin
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
3 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
7
States / cities
Loma Linda, California • Hartford, Connecticut • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 2 Interventional

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

NCT05059327
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Basimglurant with crossover to Placebo, Placebo with crossover to Basimglurant
Drug
Lead sponsor
Noema Pharma AG
Industry
Eligibility
5 Years to 30 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
9
States / cities
Los Angeles, California • Baltimore, Maryland • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2025 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 2 Interventional Results available

Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

NCT01289912
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
RAD001, Placebo
Drug
Lead sponsor
Mustafa Sahin
Other
Eligibility
6 Years to 21 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 24, 2018 · Synced Sep 17, 2026, 9:11 AM EDT
Completed No phase listed Observational

Tuberous Sclerosis Complex Natural History Study: Renal Manifestations

NCT00598455
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
7 Years to 65 Years
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated May 3, 2017 · Synced Sep 17, 2026, 9:11 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

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

NCT05467397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
[11C]acetate
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants

NCT04595513
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Epilepsy
Interventions
TAVT-18 (sirolimus)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Day to 6 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated May 15, 2024 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1Phase 2 Interventional

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

NCT05364021
View directory record Official record
Conditions
Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
Interventions
LP352, Placebo
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
12 Years to 65 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
29
States / cities
Tucson, Arizona • Little Rock, Arkansas • Downey, California + 24 more
Source: ClinicalTrials.gov public record
Updated Nov 7, 2024 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

NCT01031901
View directory record Official record
Conditions
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Interventions
Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
13 Years and older
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 26, 2012 · Synced Sep 17, 2026, 9:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

NCT00411619
View directory record Official record
Conditions
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 19, 2014 · Synced Sep 17, 2026, 9:11 AM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Sep 17, 2026, 9:11 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

TANDem-2: Closing the Gap to Interventions for TAND

NCT06879665
View directory record Official record
Conditions
Adult Caregivers of Individuals With TSC, Tuberous Sclerosis Complex (TSC)
Interventions
Well-Beans for Caregivers Program
Behavioral
Lead sponsor
Vrije Universiteit Brussel
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Sep 17, 2026, 9:11 AM EDT