Clinical trials: Genetic Cancer Syndromes
Hereditary Thyroid Gland Medullary Carcinoma, Locally Advanced Thyroid Gland Medullary Carcinoma
Sorafenib Tosylate in Treating Patients With Metastatic, Locally Advanced, or Recurrent Medullary Thyroid Cancer
This phase II trial studies how well sorafenib tosylate works in treating patients with medullary thyroid cancer that has spread to other parts of the body (metastatic), spread to the tissue surrounding the thyroid (locally…
- Conditions
- Hereditary Thyroid Gland Medullary Carcinoma, Locally Advanced Thyroid Gland Medullary Carcinoma, Multiple Endocrine Neoplasia Type 2A, Multiple Endocrine Neoplasia Type 2B
- Trial name
- NCT00390325
- Testing
- Sorafenib Tosylate
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatoses
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
- Trial name
- NCT01682811
- Testing
- Part 1 Levulan injection, Part 1 Levulan surface application, Part 1 Levulan surface application twice
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Harry T Whelan, MD
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
Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma
Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma
This randomized phase II trial studies how well low-dose lenalidomide works compared with high-dose lenalidomide in treating younger patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas that have come back…
- Conditions
- Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Childhood Visual Pathway Glioma
- Trial name
- NCT01553149
- Testing
- Lenalidomide, Pharmacological Study
- Where
- 126 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Cancers
A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair Deficiency
This research study is studying a checkpoint kinase 1 (CHK1) inhibitor as a possible treatment for advanced solid tumors that harbor genetic alterations in the homologous repair (HR) pathway, genetic alterations that indicate…
- Trial name
- NCT02873975
- Testing
- LY2606368
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Dana-Farber Cancer Institute
Vestibular Schwannoma, Neurofibromatosis Type 2
Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)
People who have neurofibromatosis type 2 (NF2) can have tumors that grow on the auditory nerves and cause hearing loss. These tumors are called vestibular schwannomas (VSs), or acoustic neuromas.
- Trial name
- NCT01207687
- Testing
- bevacizumab, laboratory biomarker analysis, quality-of-life assessment
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- National Cancer Institute (NCI)
Hyperparathyroidism, Hypercalcemia
Studies of Elevated Parathyroid Activity
Observational Phase: Patients whose parathyroid activity is elevated above normal are referred to as having hyperparathyroidism.
- Conditions
- Hyperparathyroidism, Hypercalcemia, Parathyroid Neoplasm, Multiple Endocrine Neoplasia
- Trial name
- NCT00001277
- Testing
- 68Ga-Dotatate, 18F-DOPA
- Where
- United States
- Volunteers
- 1,553 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
Acute Myeloid Leukemia, Down Syndrome
Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome
This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome.
- Conditions
- Acute Myeloid Leukemia, Down Syndrome, Myelodysplastic Syndrome, Myeloid Leukemia Associated With Down Syndrome
- Trial name
- NCT02521493
- Testing
- Asparaginase, Asparaginase Erwinia chrysanthemi, Cytarabine
- Where
- 191 sites in United States, Australia and 4 more
- Volunteers
- 280 people
- Sponsor
- Children's Oncology Group
Acute Myeloid Leukemia, Myelodysplastic Syndrome
Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
This randomized phase II/III trial studies how well azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone work in treating older patients with newly diagnosed acute myeloid leukemia or high-risk…
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome/Acute Myeloid Leukemia
- Trial name
- NCT03092674
- Testing
- Azacitidine, Cytarabine, Decitabine
- Where
- 506 sites in United States
- Volunteers
- 76 people
- Sponsor
- National Cancer Institute (NCI)
Familial Adenomatous Polyposis (FAP)
Observational Familial Adenomatous Polyposis Registry Study In Patients Receiving Celecoxib Compared to Control Patients
This is a registry-based observational study assessing clinical outcomes in FAP patients receiving celecoxib compared with historical/concurrent registry patients who have not received celecoxib.
- Trial name
- NCT00151476
- Testing
- Celecoxib, Routine Medical Care
- Where
- 4 sites in United States, Canada and 2 more
- Volunteers
- 68 people
- Sponsor
- Pfizer
Childhood Hepatocellular Carcinoma, Papillary Thyroid Cancer
Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer
This phase II trial studies how well sorafenib tosylate works in treating younger patients with relapsed or refractory rhabdomyosarcoma, Wilms tumor, liver cancer, or thyroid cancer.
- Conditions
- Childhood Hepatocellular Carcinoma, Papillary Thyroid Cancer, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Liver Cancer
- Trial name
- NCT01502410
- Testing
- sorafenib tosylate, pharmacological study, laboratory biomarker analysis
- Where
- 92 sites in United States, Australia and 1 more
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
PTCH1 or SMO Activated Solid and Hematologic Tumors
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
- Trial name
- SIGNATURE (NCT02002689)
- Testing
- LDE225
- Where
- 10 sites in United States
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Familial Adenomatous Polyposis
Use of Curcumin for Treatment of Intestinal Adenomas in Familial Adenomatous Polyposis (FAP)
Familial Adenomatous Polyposis (FAP) is an autosomal dominant disorder characterized by the formation of multiple adenomatous colorectal polyps usually in the teenage years.
- Trial name
- NCT00927485
- Testing
- Calcumin (Curcumin), Risk Factor Questionnaire, Blood samples
- Where
- Puerto Rico
- Volunteers
- 44 people
- Sponsor
- University of Puerto Rico
Peutz-Jeghers Syndrome
Pilot Study of mTOR Inhibitor Therapy in Peutz-Jeghers Syndrome
Pilot study, Open-label, Phase II study of Everolimus. Objective: To determine if Everolimus can diminish large gastrointestinal polyps in patients with Peutz-Jeghers Syndrome.
- Trial name
- NCT00811590
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Utah
Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor
Vincristine, Dactinomycin, and Doxorubicin With or Without Radiation Therapy or Observation Only in Treating Younger Patients Who Are Undergoing Surgery for Newly Diagnosed Stage I, Stage II, or Stage III Wilms' Tumor
This phase III trial is studying vincristine, dactinomycin, and doxorubicin with or without radiation therapy or observation only to see how well they work in treating patients undergoing surgery for newly diagnosed stage I…
- Conditions
- Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor
- Trial name
- NCT00352534
- Testing
- 3-Dimensional Conformal Radiation Therapy, Biospecimen Collection, Chest Radiography
- Where
- 240 sites in United States, Australia and 5 more
- Volunteers
- 808 people
- Sponsor
- Children's Oncology Group
Cutaneous Neurofibroma, Neurofibromatosis 1
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 gel in participants with cNF.
- Trial name
- NCT05005845
- Testing
- NFX-179 gel, Vehicle gel
- Where
- 23 sites in United States
- Volunteers
- 199 people
- Sponsor
- NFlection Therapeutics, Inc.
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors
RATIONALE: Dasatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs in chemotherapy, such as ifosfamide, carboplatin, and etoposide, work in different ways to stop the growth of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00788125
- Testing
- carboplatin, dasatinib, etoposide phosphate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- City of Hope Medical Center
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.
Cancer
Clinical Study of WT2725 in Patients With Advanced Malignancies
This clinical study is designed to evaluate the safety, immunogenicity and antitumor activity of WT2725. WT2725 will be administered to patients with advanced malignancies known to overexpress WT1
- Trial name
- NCT01621542
- Testing
- WT2725
- Where
- 6 sites in United States
- Volunteers
- 64 people
- Sponsor
- Sumitomo Pharma America, Inc.