Clinical trials: Inherited Cancer Syndrome
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma
Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
This phase II pediatric MATCH treatment trial studies how well selpercatinib works in treating patients with solid tumors that may have spread from where they first started to nearby tissue, lymph nodes, or distant parts of the…
- Conditions
- Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma
- Trial name
- NCT04320888
- Testing
- Computed Tomography, Magnetic Resonance Imaging, Positron Emission Tomography
- Where
- 174 sites in United States, Australia and 1 more
- Volunteers
- 1 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis, Neurofibromas
Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity…
- Trial name
- 0908-09 (NCT01140360)
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Kent Robertson
Childhood Choroid Plexus Tumor, Childhood Medulloblastoma
Study of Fixed vs. Flexible Filgrastim to Accelerate Bone Marrow Recovery After Chemotherapy in Children With Cancer
This randomized phase III trial studies flexible administration of filgrastim after combination chemotherapy to see how well it works compared to fixed administration of filgrastim in decreasing side effects of chemotherapy in…
- Conditions
- Childhood Choroid Plexus Tumor, Childhood Medulloblastoma, Childhood Pineoblastoma, Childhood Soft Tissue Sarcoma
- Trial name
- NCT01987596
- Testing
- filgrastim
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
To determine the clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic neurofibromatosis type 1 (NF1) associated or sporadic MPNST.
- Trial name
- NCT03433183
- Testing
- Selumetinib, Sirolimus
- Where
- 5 sites in United States
- Volunteers
- 21 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
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
Wilms Tumor, Fibrosarcoma
Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.
- Conditions
- Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer
- Trial name
- NCT00141765
- Testing
- Myeloablative Chemotherapy, Stem Cell Rescue
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Michigan Rogel Cancer Center
Epidermal Nevus Syndrome
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
KRN23 is a fully human immunoglobulin monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels.
- Trial name
- ENS (NCT04320316)
- Testing
- Crysvita (burosumab-twza) Treatment
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis 1, Chronic Pain
Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical Trial
To demonstrate the initial feasibility and determine preliminary impact on clinical outcomes of the iCanCope-NF program in a pilot RCT.
- Trial name
- NCT04561765
- Testing
- iCanCope, iCanCope+Contingency Management
- Where
- United States
- Volunteers
- 122 people
- Sponsor
- Yale University
Polycystic Ovary Syndrome
Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome
The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women.
- Trial name
- PCOS (NCT03152591)
- Testing
- LIK066
- Where
- 5 sites in United States, Germany
- Volunteers
- 29 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia, Myelodysplastic Syndromes
Helical Tomotherapy, Fludarabine Phosphate, and Melphalan Followed By Allo-HSCT in Hematological Malignancies
RATIONALE: Giving chemotherapy drugs, such as fludarabine phosphate and melphalan, and HT before a donor stem cell transplant helps stop the growth of cancer cells.
- Trial name
- NCT00544466
- Testing
- fludarabine phosphate, melphalan, allogeneic hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- City of Hope Medical Center
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia
Idarubicin, Cytarabine, and Tipifarnib in Treating Patients With Newly Diagnosed Myelodysplastic Syndromes or Acute Myeloid Leukemia
This phase I/II trial is studying the side effects and best dose of tipifarnib when given with idarubicin and cytarabine and to see how well it works in treating patients with newly diagnosed myelodysplastic syndromes or acute…
- Conditions
- Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT00096122
- Testing
- cytarabine, idarubicin, tipifarnib
- Where
- United States
- Volunteers
- 95 people
- Sponsor
- National Cancer Institute (NCI)
Von Hippel-Lindau Disease
Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. These tumors are more aggressive in some people than in others.
- Trial name
- NCT00062166
- Where
- United States
- Volunteers
- 340 people
- Sponsor
- National Cancer Institute (NCI)
Skin Carcinoma
Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Chest and Back
RATIONALE: Drugs used in chemotherapy, such as tazarotene, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00783965
- Testing
- tazarotene
- Where
- 3 sites in United States
- Volunteers
- 34 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Childhood Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma
Combination Chemotherapy, Radiation Therapy, and/or Surgery in Treating Patients With High-Risk Kidney Tumors
This phase II trial is studying how well combination chemotherapy, radiation therapy, and/or surgery work in treating patients with high-risk kidney tumors.
- Conditions
- Childhood Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma, Clear Cell Sarcoma of the Kidney, Papillary Renal Cell Carcinoma
- Trial name
- NCT00335556
- Testing
- Doxorubicin Hydrochloride, Irinotecan Hydrochloride, Conventional Surgery
- Where
- 187 sites in United States, Australia and 3 more
- Volunteers
- 291 people
- Sponsor
- Children's Oncology Group
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Arsenic Trioxide in Treating Patients With Advanced Neuroblastoma or Other Childhood Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of arsenic trioxide in treating children who have advanced…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00024258
- Testing
- arsenic trioxide
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Fludarabine, Cyclophosphamide, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing a Donor Umbilical Cord Blood Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00255684
- Testing
- graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Rochester
Genetic Testing
Development and Implementation of Electronic Decision Aids for Genetic Testing in Inherited Cancer Syndromes
An electronic decision aid will be used to assist individuals in choosing a multi-gene panel with their medical oncologist instead of a genetic counselor.
- Trial name
- NCT04704193
- Testing
- Survey of Decision Aid
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Massachusetts General Hospital
Systemic Juvenile Idiopathic Arthritis, Hereditary Periodic Fevers
A Study of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 Studies
The objective of this extension protocol is to collect safety data (serious and non-serious adverse events) and to provide continuous canakinumab to patients in France who completed study CACZ885G2301E1(NCT00891046), CACZ885G2306…
- Trial name
- NCT02334748
- Testing
- canakinumab
- Where
- 3 sites in France
- Volunteers
- 31 people
- Sponsor
- Novartis 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)