Clinical trials: Genetic Cancer Syndromes
Gastrin-Producing Neuroendocrine Tumor, Lung Carcinoid Tumor
Pazopanib Hydrochloride in Treating Patients With Advanced Neuroendocrine Cancer
This phase II trial studies how well pazopanib hydrochloride works in treating patients with advanced neuroendocrine cancer. Pazopanib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for…
- Conditions
- Gastrin-Producing Neuroendocrine Tumor, Lung Carcinoid Tumor, Metastatic Digestive System Neuroendocrine Tumor G1, Multiple Endocrine Neoplasia Type 1
- Trial name
- NCT00454363
- Testing
- Laboratory Biomarker Analysis, Pazopanib Hydrochloride, Pharmacological Study
- Where
- 3 sites in United States
- Volunteers
- 52 people
- Sponsor
- National Cancer Institute (NCI)
Basal Cell Nevus Syndrome, Gorlin's Syndrome
Trial Comparing the Effects of Intermittent Vismodegib vs. PDT in Patients With Multiple Basal Cell Carcinomas
The purpose of this study is to evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT).
- Trial name
- Vismodegib (NCT01556009)
- Testing
- Vismodegib, Aminolevulinic acid %20 topical solution
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
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.
Urothelial Cancer
A Phase II Study of Bintrafusp Alfa (M7824) in Checkpoint Inhibitor Naive and Refractory Subjects With Urothelial Carcinoma
Background: Metastatic urothelial carcinoma is lethal and has no cure. Response rates to current treatments are modest. Researchers want to find new strategies to treat the disease.
- Trial name
- NCT04501094
- Testing
- Bintrafusp alfa (M7824)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 2, Meningioma
AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas.
- Trial name
- NCT02831257
- Testing
- AZD2014
- Where
- 2 sites in United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well olaparib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with defects in deoxyribonucleic acid (DNA) damage repair genes that have…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma
- Trial name
- NCT03233204
- Testing
- Olaparib
- Where
- 113 sites in United States, Puerto Rico
- Volunteers
- 6 people
- Sponsor
- National Cancer Institute (NCI)
Complex Partial Seizures
Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients
This is an open-label, phase 4 study to examine the safety and efficacy of vigabatrin (Sabril) in Tuberous Sclerosis patients, a subset of the larger refractory complex partial epilepsy population for which the drug is approved.
- Trial name
- STARS (NCT01266291)
- Testing
- vigabatrin
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Pennsylvania
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
Children with neurofibromatosis type 1 (NF1) commonly suffer from the effects of cognitive, behavioral, and motor impairments. At present there is no specific treatment for this NF1 complication.
- Trial name
- NF1NAC (NCT04481035)
- Testing
- N-acetylcysteine (NAC)
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neuroendocrine Tumors, Von Hippel-Lindau Syndrome
Evaluation of 68Gallium-DOTATATE PET/CT for Detecting Neuroendocrine Tumors
Background: Neuroendocrine tumors (NETs) are rare but have been more common over the past decade. The only treatment for NETs is surgery, but most are found when they are too advanced for surgery.
- Conditions
- Neuroendocrine Tumors, Von Hippel-Lindau Syndrome, Hippel-Lindau Disease
- Trial name
- NCT01967537
- Testing
- 68Gallium DOTATATE, Radio-guided surgery
- Where
- United States
- Volunteers
- 341 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1
Medical Treatment of "High-Risk" Neurofibromas
Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction…
- Trial name
- NCT00846430
- Testing
- Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Corewell Health West
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Hereditary Breast and Ovarian Cancer Syndrome, Osteoporosis
Zoledronate or Observation in Maintaining Bone Mineral Density in Patients Who Are Undergoing Surgery to Remove Both Ovaries
This randomized phase II trial is studying zoledronate to see how well it works compared to observation in maintaining bone mineral density in patients who are undergoing surgery to remove both ovaries.
- Conditions
- Hereditary Breast and Ovarian Cancer Syndrome, Osteoporosis, Ovarian Carcinoma
- Trial name
- NCT00305695
- Testing
- Laboratory Biomarker Analysis, Zoledronic Acid
- Where
- 95 sites in United States
- Volunteers
- 160 people
- Sponsor
- Gynecologic Oncology Group
Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome
Pediatric Reporting of Adult-Onset Genomic Results
The Investigators will conduct a longitudinal, mixed-methods cohort study to assess primary and secondary psychosocial outcomes among MyCode adolescent participants and their parents, and health behaviors of children who received…
- Conditions
- Hereditary Breast and Ovarian Cancer Syndrome, Lynch Syndrome, Familial Hypercholesterolemia
- Trial name
- NCT03832985
- Testing
- Child(ren) receive an adult-onset result, Child(ren) received a pediatric-onset result, Control - Negative Result
- Where
- United States
- Volunteers
- 162 people, healthy volunteers welcome
- Sponsor
- Geisinger Clinic
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).
- Trial name
- ReNeu (NCT03962543)
- Testing
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- Where
- 43 sites in United States
- Volunteers
- 114 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Basal Cell Nevus Syndrome
Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
- Trial name
- NCT04308395
- Testing
- Patidegib Topical Gel, 2%
- Where
- 37 sites in United States, Belgium and 7 more
- Volunteers
- 108 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Liver Cancer, Rhabdomyosarcoma
Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors
Patients may be considered if the cancer has come back, has not gone away after standard treatment or the patient cannot receive standard treatment.
- Conditions
- Liver Cancer, Rhabdomyosarcoma, Malignant Rhabdoid Tumor, Liposarcoma
- Trial name
- NCT04377932
- Testing
- AGAR T cells
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Baylor College of Medicine
Neurofibromatosis, NF1
Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas
This is a pilot study to determine if adults and children with neurofibromatosis type 1 who have plexiform tumors given Sutent® respond to this drug therapy.
- Conditions
- Neurofibromatosis, NF1, Plexiform Neurofibromas
- Trial name
- NCT01402817
- Testing
- Sutent®/Sunitinib
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Indiana University
Pleuropulmonary Blastoma, Recurrent Malignant Peripheral Nerve Sheath Tumor
Lorvotuzumab Mertansine in Treating Younger Patients With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma
This phase II trial studies how well lorvotuzumab mertansine works in treating younger patients with Wilms tumor, rhabdomyosarcoma, neuroblastoma, pleuropulmonary blastoma, malignant peripheral nerve sheath tumor (MPNST), or…
- Conditions
- Pleuropulmonary Blastoma, Recurrent Malignant Peripheral Nerve Sheath Tumor, Recurrent Neuroblastoma, Recurrent Rhabdomyosarcoma
- Trial name
- NCT02452554
- Testing
- Laboratory Biomarker Analysis, Lorvotuzumab Mertansine, Pharmacological Study
- Where
- 69 sites in United States
- Volunteers
- 62 people
- Sponsor
- Children's Oncology Group
Carcinoma, Basal Cell, Recurrent Skin Cancer
Pilot Study of Sonidegib and Buparlisib in Treating Patients With Advanced or Metastatic Basal Cell Carcinoma
This pilot trial studies how well sonidegib and buparlisib work in treating patients with basal cell carcinoma that has spread to other places in the body.
- Conditions
- Carcinoma, Basal Cell, Recurrent Skin Cancer, Skin Neoplasms, Basal Cell Nevus Syndrome
- Trial name
- NCT02303041
- Testing
- Buparlisib, Sonidegib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Anne Chang