Clinical trials: Neoplastic Syndromes, Hereditary
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
Solid Tumors, Breast Cancer
A Phase I/II Study of BGT226 in Adult Patients With Advanced Solid Malignancies Including Patients With Advanced Breast Cancer
This is a phase I/II clinical research study with BGT226, an inhibitor of phosphatidylinositol 3'-kinase (PI3K). The study consists of a Phase I dose escalation part followed by a safety expansion part and a Phase II expansion…
- Conditions
- Solid Tumors, Breast Cancer, Cowden Syndrome
- Trial name
- NCT00600275
- Testing
- BGT226
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Neurofibromatosis 1, Cutaneous Neurofibroma
NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
This study will enroll and treat subjects with cutaneous neurofibromas with NFX-179, a topical study drug. Eligible subjects will receive treatment for 28 days and be observed by a study doctor for approximately 56 days.
- Trial name
- NCT04435665
- Testing
- NFX-179 Gel, Vehicle Gel
- Where
- 6 sites in United States
- Volunteers
- 48 people
- Sponsor
- NFlection Therapeutics, Inc.
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn if Elinzanetant Affects the Ability to Drive and Brain Function in Healthy Women
Researchers are looking for a better way to treat vasomotor symptoms (VMS), also known as hot flashes. Hot flashes are intense and sudden feelings of heat along with sweating and reddening of the skin.
- Trial name
- NCT06219902
- Testing
- Elinzanetant (BAY3427080), Zopiclone
- Where
- Canada
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Bayer
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- NCT05626634
- Testing
- LP352, bexicaserin
- Where
- 28 sites in United States, Australia
- Volunteers
- 41 people
- Sponsor
- Longboard Pharmaceuticals
Neurofibromatosis 1, Plexiform Neurofibroma
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT04954001
- Testing
- FCN-159
- Where
- 14 sites in United States, China and 1 more
- Volunteers
- 160 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Tuberous Sclerosis Complex
Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy.
- Trial name
- NCT05323734
- Testing
- Ganaxolone
- Where
- 63 sites in United States, Australia and 8 more
- Volunteers
- 129 people
- Sponsor
- Marinus Pharmaceuticals
Familial Adenomatous Polyposis
Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this randomized, double-blind, Phase III trial is to determine if the combination of eflornithine plus sulindac is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of…
- Trial name
- NCT01483144
- Testing
- Eflornithine, Sulindac 150 MG
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 171 people
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Age-Related Macular Degeneration
Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections
This clinical study was designed to compare the safety and efficacy of brolucizumab 6 mg dosed every 4 weeks to aflibercept 2 mg dosed every 4 weeks in those neovascular age-related macular degeneration (nAMD) patients with…
- Trial name
- MERLIN (NCT03710564)
- Testing
- Brolucizumab, Aflibercept
- Where
- 61 sites in United States, Puerto Rico
- Volunteers
- 535 people
- Sponsor
- Novartis Pharmaceuticals
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How a Proton Pump Inhibitor Affects the Way Elinzanetant (BAY 3427080) Moves Into, Through and Out of the Body, and How Much of it Gets Absorbed by the Body When Taken as a Single and Small Radioactive Dose in Healthy Adults
Researchers are looking for a better way to treat people who have vasomotor symptoms (VMS). VMS such as hot flashes are caused by hormonal changes occurring during menopausal transition when women may have also changes in their…
- Trial name
- NCT05061563
- Testing
- Elinzanetant (BAY3427080), Esomeprazole
- Where
- Netherlands
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Bayer
Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients
A Trial to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of LDE225 on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients
Part I was a double-blind, randomized, vehicle-controlled Proof of Concept (PoC) study to evaluate the safety, local tolerability, pharmacokinetics and pharmacodynamics of multiple topical administrations of LDE225 (a specific…
- Trial name
- NCT00961896
- Testing
- Vehicle, LDE225 0.25%, LDE225 0.75%
- Where
- 3 sites in Austria, Switzerland
- Volunteers
- 18 people
- Sponsor
- Novartis Pharmaceuticals
Recurrent Basal Cell Carcinoma
A Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas in Patients With Non-Gorlin High Frequency BCC
This is a multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with non-Gorlin HF-BCC…
- Trial name
- NCT04155190
- Testing
- Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
- Where
- 14 sites in United States
- Volunteers
- 50 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Tuberous Sclerosis Complex
Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
- Trial name
- NCT05059327
- Where
- 27 sites in United States, India and 6 more
- Volunteers
- 61 people
- Sponsor
- Noema Pharma AG
Tuberous Sclerosis Complex Associated Neuropsychiatric Disease
A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex
The purpose of this study is to investigate behavioral and other co-occurring outcomes with EPID(I/Y)OLEX as an add-on therapy in participants aged 1 to 65 years with tuberous sclerosis complex (TSC) who experience seizures.
- Trial name
- EpiCom (NCT05864846)
- Testing
- Cannabidiol Oral Solution [Epidiolex]
- Where
- 19 sites in United States, Canada and 2 more
- Volunteers
- 79 people
- Sponsor
- Jazz Pharmaceuticals
Nevi, Dysplastic
INTER-PATHOLOGIST READER STUDY
This is a post study histology analysis from previously obtained pathology slides.
- Trial name
- NCT06246903
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Orlucent, Inc
Neuroblastoma, Rhabdomyosarcoma
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
This study is a Phase 1, open-label, dose escalation and cohort expansion trial designed to characterize the safety, tolerability, PK, PD, immunogenicity and preliminary antitumor activity of enoblituzumab administered IV on a…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT02982941
- Testing
- Enoblituzumab
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- MacroGenics
Dravet Syndrome, Lennox Gastaut Syndrome
Intermediate-Size Expanded Access Protocol (EAP) for LP352
This is an intermediate-size expanded access program (EAP) study. The purpose of this EAP is to provide continued access to LP352, an investigational drug product being investigated in participants with DEEs.
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Developmental and Epileptic Encephalopathies
- Trial name
- NCT06149663
- Testing
- LP352
- Where
- 22 sites in United States, Australia
- Sponsor
- Longboard Pharmaceuticals
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn How Different Levels of Decreased Liver Function Influence Blood Levels of Elinzanetant Compared to Normal Liver Function in Male and Female Participants
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, like those that happen in women during menopause. These symptoms can include vasomotor symptoms.
- Trial name
- NCT04903821
- Testing
- BAY3427080
- Where
- 2 sites in Germany
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Bayer
Tuberous Sclerosis Complex
Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001
- Trial name
- NCT05604170
- Testing
- Ganaxolone
- Where
- 54 sites in United States, Australia and 8 more
- Volunteers
- 117 people
- Sponsor
- Marinus Pharmaceuticals
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)
This study evaluated the efficacy and safety of Everolimus in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.
- Trial name
- EXIST-1 (NCT00789828)
- Testing
- Everolimus
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 117 people
- Sponsor
- Novartis Pharmaceuticals