Clinical trials: Inherited Cancer Syndrome
Neuroblastoma, Rhabdomyosarcoma
Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors
Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma, Peripheral Nerve Sheath Tumors
- Trial name
- NCT06625190
- Testing
- Miltenyi CliniMACS Prodigy ® system, Zoledronic acid
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Florida
Desmoplastic Small Round Cell Tumor, Osteosarcoma
Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
This phase I/II trial studies the effects of trastuzumab deruxtecan (DS-8201a) in treating patients with osteosarcoma, Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) that is newly diagnosed or has come back after…
- Conditions
- Desmoplastic Small Round Cell Tumor, Osteosarcoma, Recurrent Desmoplastic Small Round Cell Tumor, Recurrent Kidney Wilms Tumor
- Trial name
- NCT04616560
- Testing
- Biospecimen Collection, Computed Tomography, Echocardiography Test
- Where
- 32 sites in United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission
Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
RATIONALE: Giving chemotherapy before a donor umbilical cord blood transplant (UCBT) helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT01093586
- Testing
- double-unit umbilical cord blood transplantation, cytogenetic analysis, bone marrow aspiration
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Case Comprehensive Cancer Center
Neurofibromatosis 1, Social Skills
Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1
This is a pilot randomized control trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.
- Trial name
- NCT04860362
- Testing
- PEERS
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Milwaukee
Neurofibroma, Plexiform, Neurofibromatosis Type I
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects…
- Trial name
- NCT00021541
- Testing
- tipifarnib
- Where
- 12 sites in United States, Germany
- Volunteers
- 62 people
- Sponsor
- National Cancer Institute (NCI)
Tuberous Sclerosis Complex (TSC)
Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)
1. To assess treatment efficacy with YCJ-01 by changes in the number of epileptic seizures in patients with refractory epilepsy secondary to Tuberous Sclerosis Complex. 2.
- Trial name
- Spectrum (NCT07403266)
- Testing
- YCJ-01 Full Spectrum cannbis Extract
- Where
- Spain
- Volunteers
- 84 people
- Sponsor
- Oils4Cure
Cancer
Caspofungin Acetate Compared With Amphotericin B Liposomal in Treating Patients With Persistent Fever and Neutropenia Following Cancer Treatment
RATIONALE: Caspofungin acetate or amphotericin B liposomal may be effective in preventing or controlling fever and neutropenia caused by chemotherapy, bone marrow transplantation, or peripheral stem cell transplantation.
- Trial name
- NCT00008359
- Testing
- caspofungin acetate, liposomal amphotericin B
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Graft Versus Host Disease, Leukemia
Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Advanced Hematologic Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of…
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00014469
- Testing
- busulfan, melphalan, methotrexate
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Colorectal Cancer, Lynch Syndrome
Molecular Screening for Lynch Syndrome in Southern Denmark
A clinically applicably strategy for molecular screening for Lynch Syndrome has been implemented in the Region of Southern Denmark.
- Conditions
- Colorectal Cancer, Lynch Syndrome, HNPCC
- Trial name
- NCT01216930
- Testing
- Observation
- Where
- 7 sites in Denmark
- Volunteers
- 2,000 people
- Sponsor
- Vejle Hospital
Neurofibromatosis 1, Neurofibromatosis 2
The NF Registry Sponsored by the Children's Tumor Foundation
The NF Registry is a database of patient-reported symptoms, treatments, and experiences with their neurofibromatosis disease. It is a contact registry to relay clinical trial opportunities to targeted patient subgroups, and to…
- Conditions
- Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis, NF2-related Schwannomatosis
- Trial name
- NCT01885767
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- The Children's Tumor Foundation
Soft Tissue Sarcoma, Lymphoma
Liposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies
The goal of this clinical research study is to learn if treatment with the drug liposomal vincristine can shrink or slow the growth of the patient's cancer. The safety of this drug will also be studied.
- Conditions
- Soft Tissue Sarcoma, Lymphoma, Leukemia, Wilms' Tumor
- Trial name
- NCT00038207
- Testing
- Liposomal Vincristine
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- M.D. Anderson Cancer Center
Neurofibromatosis Type 1
Ophthalmological Screening and Follow-up of Optic Pathway Gliomas in Children With Neurofibromatosis Type 1.
The goal of this project is to get more insight into the (neuro)ophthalmological characteristics of children with neurofibromatosis type 1.
- Trial name
- NCT02078401
- Where
- Belgium
- Volunteers
- 200 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Recurrent Solid Tumor, Recurrent Ewing Sarcoma
Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away).
- Conditions
- Recurrent Solid Tumor, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Malignant Germ Cell Tumor
- Trial name
- NCT04901702
- Testing
- Onivyde, Talazoparib, Temozolomide
- Where
- 10 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Chronic Myeloproliferative Disorders, Leukemia
Reduced-Intensity Busulfan and Fludarabine With or Without Antithymocyte Globulin Followed by Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy, such as busulfan and fludarabine, before a donor stem cell transplant helps stop the growth of cancer and abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00448201
- Testing
- anti-thymocyte globulin, sargramostim, therapeutic allogeneic lymphocytes
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
Huntington Disease, Hereditary Breast and Ovarian Cancer
Genetic Risk: Whether, When, and How to Tell Adolescents
This study is being conducted to learn more about family communication of genetic risk information. Semi-structured interviews lasting up to one hour will be conducted with three populations: parent/child pairs at risk for…
- Conditions
- Huntington Disease, Hereditary Breast and Ovarian Cancer, Hereditary Cancer, Hereditary Non-polyposis Colon Cancer
- Trial name
- NCT03421327
- Where
- United States
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Acute Myeloid Leukemia/Transient Myeloproliferative Disorder, Acute Undifferentiated Leukemia
Fludarabine Phosphate and Total-Body Radiation Followed by Donor Peripheral Blood Stem Cell Transplant and Immunosuppression in Treating Patients With Hematologic Malignancies
This clinical trial studies fludarabine phosphate and total-body radiation followed by donor peripheral blood stem cell transplant and immunosuppression in treating patients with hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia/Transient Myeloproliferative Disorder, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00014235
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- 11 sites in United States, Germany and 1 more
- Volunteers
- 160 people
- Sponsor
- Fred Hutchinson Cancer Center
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Temozolomide and O6-benzylguanine in Treating Children With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00020150
- Testing
- O6-benzylguanine, temozolomide
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
Pituitary Tumor, Endocrine Neoplasia
Epidemiology of Pituitary Tumours: Prevalence of Associated Neoplasia
The study aims to update current knowledge about the epidemiology of pituitary tumours (PiT), based on the wide body of scientific literature on new familial and/or syndromic forms.
- Conditions
- Pituitary Tumor, Endocrine Neoplasia, Hyperparathyroidism, Solid Tumor
- Trial name
- EpidemioPIT (NCT03973450)
- Testing
- Registration of familial forms and associated neoplasia
- Where
- Italy
- Volunteers
- 400 people
- Sponsor
- Neuromed IRCCS
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