Clinical trials: Hereditary Cancer Syndrome
Vestibular Schwannoma, Inner Ear Disease
Vestibular Precision: Physiology & Pathophysiology
This project will investigate the role of noise in the vestibular system, and in particular its effects on the variability (precision) of vestibular-mediated behaviors.
- Conditions
- Vestibular Schwannoma, Inner Ear Disease, Dizziness, Neurofibromatosis 2
- Trial name
- NCT04890132
- Testing
- VOR precision training
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Massachusetts Eye and Ear Infirmary
Fanconi's Anemia, Myelodysplastic Syndromes
Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and radiotherapy in terms of improving survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with…
- Conditions
- Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
- Trial name
- NCT00005892
- Testing
- cyclophosphamide, Allogeneic Bone Marrow Transplantation
- Where
- United States
- Sponsor
- Fairview University Medical Center
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Von Hippel Lindau Disease, Kidney Neoplasms
Von Hippel-Lindau Disease Genetic Epidemiology Study
The Von Hippel-Lindau (VHL) Disease Genetic Epidemiology Study is a family-based case-control study to be conducted by the National Cancer Institute.
- Trial name
- NCT00001803
- Where
- United States
- Volunteers
- 546 people
- Sponsor
- National Cancer Institute (NCI)
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Li-Fraumeni Syndrome, TP53 Gene Mutation
Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress
The purpose of this research study is to learn more about variants in the TP53 gene both associated with Li-Fraumeni Syndrome (LFS), a hereditary cancer risk condition, and TP53 variants found in the blood for other reasons (e.g.
- Conditions
- Li-Fraumeni Syndrome, TP53 Gene Mutation, Hereditary Cancer Syndrome, Clonal Hematopoiesis
- Trial name
- LiFT_UP (NCT04541654)
- Testing
- Data and Specimen Collection
- Where
- 3 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- Dana-Farber Cancer Institute
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Osteosarcoma, Ewing Sarcoma
Neoadjuvant Dual Checkpoint Inhibition and Cryoablation in Relapsed/Refractory Pediatric Solid Tumors
The is a phase II, single arm, open-label, multi-site trial studying the combination of cryoablation therapy and dual checkpoint inhibition with nivolumab (anti-PD-1) and ipilimumab (anti-CTLA-4) given at the recommended phase 2…
- Conditions
- Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Relapsed Pediatric Solid Tumor
- Trial name
- NCT05302921
- Testing
- Cryoablation Therapy, Nivolumab, Ipilimumab
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's National Research Institute
Neurofibromatosis 2, Vestibular Schwannoma
Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
In this research study the researchers want to learn more about the effects (both good and bad) the study drug selumetinib has on participants with neurofibromatosis type II (NF2) related tumor.
- Conditions
- Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma
- Trial name
- SEL-TH-1601 (NCT03095248)
- Testing
- Selumetinib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neurofibromatosis 1, Neurofibroma
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1
This study is being conducted to determine if Whole Body MRI (WBMRI) can be used to identify Atypical Neurofibromas (ANF) in Neurofibromatosis Type 1 (NF1) patients with high tumor burden.
- Conditions
- Neurofibromatosis 1, Neurofibroma, Atypical Neurofibroma, Atypical Neurofibromatosis
- Trial name
- NCT03820778
- Testing
- Whole Body MRI
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's National Research Institute
Neoplastic Syndromes, Hereditary, Urologic Neoplasms
Genetic Analysis of Inherited Urologic Malignant Disorders: Collection of Samples
Investigation of the causes of genetic defects relating to hereditary urologic malignancies will be undertaken. These rare disorders result from inherited or newly arising mutations in genes involved in the development and…
- Trial name
- NCT00001814
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Childhood Acute Lymphoblastic Leukemia, Childhood Rhabdomyosarcoma
Evaluating Dactinomycin and Vincristine in Young Patients With Cancer
This laboratory study is evaluating how well dactinomycin and vincristine work in treating young patients with cancer. Studying samples of blood and urine in the laboratory from patients with cancer may help doctors learn how…
- Conditions
- Childhood Acute Lymphoblastic Leukemia, Childhood Rhabdomyosarcoma, Childhood Soft Tissue Sarcoma, Ewing Sarcoma
- Trial name
- NCT00674193
- Testing
- pharmacological study, laboratory biomarker analysis
- Where
- 38 sites in United States, Australia and 1 more
- Volunteers
- 158 people
- Sponsor
- Children's Oncology Group
Melanoma, Dysplastic Nevus Syndrome
Clinical, Laboratory, and Epidemiologic Characterization of Individuals and Families at High Risk of Melanoma
This study will investigate how genetic and environmental factors contribute to the development of melanoma, a type of skin cancer, and related conditions.
- Trial name
- NCT00040352
- Where
- 2 sites in United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Epilepsy, Focal Cortical Dysplasia
Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery
Refractory epilepsy, meaning epilepsy that no longer responds to medication, is a common neurosurgical indication in children. In such cases, surgery is the treatment of choice.
- Conditions
- Epilepsy, Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly
- Trial name
- NCT04344626
- Testing
- Intra-operative brain tonometry
- Where
- 2 sites in United States, Canada
- Sponsor
- University of California, Los Angeles
Cancer
Itraconazole Compared With Fluconazole to Prevent Infections in Patients Undergoing Peripheral Stem Cell or Bone Marrow Transplantation
RATIONALE: Giving itraconazole or fluconazole may be effective in preventing infections in patients undergoing peripheral stem cell or bone marrow transplantation.
- Trial name
- NCT00003883
- Testing
- fluconazole, itraconazole
- Where
- 2 sites in United States
- Volunteers
- 578 people
- Sponsor
- Fred Hutchinson Cancer Center
Leukemia, Lymphoma
Melphalan, Fludarabine, and Alemtuzumab Followed by Peripheral Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as melphalan and fludarabine, and a monoclonal antibody, such as alemtuzumab, before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer…
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00027560
- Testing
- alemtuzumab, cyclosporine, fludarabine phosphate
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neuroblastoma, Central Nervous System Tumors
Chemotherapy With CD133+ Select Autologous Hematopoietic Stem Cells for Children With Solid Tumors and Lymphomas
Studies have provided evidence that residual microscopic malignant cells in autologous bone marrow or blood stem cell grafts can contribute to posttransplant relapse.
- Conditions
- Neuroblastoma, Central Nervous System Tumors, Lymphomas, Wilms Tumor
- Trial name
- NCT00152126
- Testing
- Stem Cell Transplantation, Busulfan, Melphalan
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- St. Jude Children's Research Hospital
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
TSC
TSC Remote Assessment and Intervention
The investigators propose a study to adapt an evidence-based caregiver training program to improve access in order to better serve the TSC community.
- Trial name
- TRAIN (NCT04698538)
- Testing
- JASPER
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- University of California, Los Angeles
Leukemia
Radiolabeled Monoclonal Antibody, Cyclophosphamide, and Total Body Irradiation Followed By Donor Stem Cell Transplantation in Treating Patients With Advanced Acute Myeloid Leukemia
RATIONALE: Radiolabeled monoclonal antibodies can locate cancer cells and either kill them or deliver radioactive cancer-killing substances to them without harming normal cells.
- Trial name
- NCT00003868
- Testing
- cyclophosphamide, methotrexate, allogeneic bone marrow transplantation
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center