Clinical trials: Hereditary Neoplastic Syndromes
Adenomatous Polyposis Coli
A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis
The purpose of this study is to determine the effect of JNJ-64251330 in participants with Familial Adenomatous Polyposis (FAP) on colorectal polyp burden (sum of the polyp diameters).
- Trial name
- NCT05014360
- Testing
- JNJ-64251330
- Where
- 16 sites in United States, France and 5 more
- Volunteers
- 42 people
- Sponsor
- Janssen Research & Development, LLC
Neurofibromatosis
Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia
Clinical and Demographic Characteristics of Adult Patients with NEurofibromatosis in RUSsia (NEREUS)
- Trial name
- NEREUS (NCT07088991)
- Where
- 16 sites in Russia
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia
Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.
This is a Phase 1, open-label, 3-period study to determine radiprodil's potential to act as a perpetrator of cytochrome P-450 (CYP) metabolic pathways and transporter pathways.
- Conditions
- Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia, Other Neurological Disorders
- Trial name
- NCT06975605
- Testing
- Radiprodil + co-administered drugs
- Where
- Australia
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- GRIN Therapeutics, Inc.
Breast Cancer, Advanced Solid Tumors
A Phase I/II Study of BEZ235 in Patients With Advanced Solid Malignancies Enriched by Patients With Advanced Breast Cancer
This is a first-in-human, phase I/Ib clinical research study with BEZ235, an inhibitor of phosphatidylinositol 3'-kinase (PI3K).
- Conditions
- Breast Cancer, Advanced Solid Tumors, Cowden Syndrome
- Trial name
- NCT00620594
- Testing
- BEZ235
- Where
- 15 sites in United States, Germany and 3 more
- Volunteers
- 183 people
- Sponsor
- Novartis Pharmaceuticals
Healthy
A Pharmacokinetics and Safety of Methylphenidate HCl in Healthy Japanese Adult Male Volunteers
The purpose of the study is to evaluate the pharmacokinetics and safety of 3 single doses of Methylphenidate HCl in healthy Japanese adult male volunteers.
- Trial name
- NCT01202734
- Testing
- Methylphenidate HCl
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Adenomatous Polyposis Coli
A Study of Guselkumab in Participants With Familial Adenomatous Polyposis
The purpose of this study is to determine the effect of treatment with guselkumab in participants with familial adenomatous polyposis (FAP) on rectal/pouch polyp burden.
- Trial name
- NCT03649971
- Testing
- Guselkumab
- Where
- 32 sites in United States, France and 7 more
- Volunteers
- 77 people
- Sponsor
- Janssen Research & Development, LLC
Exostoses, Multiple Hereditary
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple…
- Trial name
- MO-Ped (NCT03442985)
- Testing
- Palovarotene 2.5 mg, Palovarotene 5.0 mg
- Where
- 31 sites in United States, Australia and 10 more
- Volunteers
- 193 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Neurofibromatosis 1, Plexiform Neurofibroma
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT05913037
- Testing
- Test group (Group A): FCN-159 8 mg, orally, once daily;
- Where
- 15 sites in China
- Volunteers
- 167 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Facial Angiofibroma, Tuberous Sclerosis
Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous Sclerosis Complex (TSC).
- Trial name
- NCT03826628
- Testing
- rapamycin
- Where
- 17 sites in United States, Australia and 7 more
- Volunteers
- 107 people
- Sponsor
- Dermatology Specialties Limited Partnership
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
Basal Cell Carcinomas
Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle in patients with…
- Trial name
- BCC (NCT02828111)
- Testing
- Patidegib, Vehicle gel
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- PellePharm, Inc.
Skin Lesion, Melanoma (Skin)
Assessing Tissue Remodeling in the Skin Using SFI (Skin Fluorescence Imaging)
The goal of the SFI is to provide non-invasive information about tissue remodeling occurring during melanocytic transition and atypia development in the skin
- Conditions
- Skin Lesion, Melanoma (Skin), Mole
- Trial name
- NCT03535077
- Testing
- Diagnostic Test
- Where
- 6 sites in United States
- Volunteers
- 186 people
- Sponsor
- Orlucent, Inc
Advanced Breast Cancer
Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR-positive, HER2-negative, Advanced Breast Cancer (aBC) With PIK3CA Mutation, Whose Disease Progressed on or After Endocrine Treatment
The purpose of this study was to determine the safety of alpelisib plus fulvestrant in men and post-menopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, advanced or…
- Trial name
- ALPINIST (NCT05631795)
- Testing
- Alpelisib, Fulvestrant
- Where
- 12 sites in India
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
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
BCCs in Gorlin Syndrome Patients
CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin…
- Trial name
- NCT04893486
- Testing
- PTX-022, Vehicle comparator
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 73 people
- Sponsor
- Palvella Therapeutics, Inc.
Tuberous Sclerosis Complex
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
The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be…
- Trial name
- NCT02962414
- Testing
- everolimus
- Where
- 67 sites in United States, Australia and 16 more
- Volunteers
- 206 people
- Sponsor
- Novartis Pharmaceuticals
Familial Adenomatous Polyposis
Trial of eRapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance
Patients with Familial Adenomatous Polyposis (FAP) who are undergoing endoscopic surveillance will be given Encapsulated Rapamycin (eRapa) at one of three escalating doses/schedules for 12 months with the aim of reducing polyp…
- Trial name
- NCT04230499
- Testing
- Encapsulated Rapamycin (eRapa)
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Rapamycin Holdings Inc.
Low Bone Mass, Low Bone Mineral Density
Study to Compare the Efficacy and Safety of DenosumAb Versus Placebo in Males With Osteoporosis
The purpose of this study is to assess how effective and safe denosumab is in a population of males with low bone mass at risk of fracture.
- Conditions
- Low Bone Mass, Low Bone Mineral Density, Males With Osteoporosis, Osteopenia
- Trial name
- NCT00980174
- Testing
- 60 mg denosumab
- Volunteers
- 242 people
- Sponsor
- Amgen
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How the Study Drug Elinzanetant (BAY 3427080) Affects the Way the Drug Dabigatran Moves Into, Through and Out of the Body in Healthy Male and Female Participants
Researchers are looking for a better way to treat men and women with vasomotor symptoms, a condition of having hot flashes caused by hormonal changes.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05471817
- Testing
- Elinzanetant (BAY3427080), Dabigatran etexilate
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Bayer
Coronary Heart Disease (CHD)
Effect of Evolocumab in Patients at High Cardiovascular Risk Without Prior Myocardial Infarction or Stroke
This study will assess the effect of lowering low-density lipoprotein cholesterol (LDL-C) with evolocumab on major cardiovascular events in adults without a prior myocardial infarction (MI) or stroke who are at high risk of a…
- Trial name
- VESALIUS-CV (NCT03872401)
- Testing
- Evolocumab
- Where
- 852 sites in United States, Argentina and 31 more
- Volunteers
- 12,301 people
- Sponsor
- Amgen