Clinical trials: Genetic Cancer Syndromes
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Neurofibromatosis Type 1, Reading Disabilities
Neurobiology and Treatment of Reading Disability in NF-1
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with…
- Trial name
- NCT00624234
- Testing
- Tutoring Program I, Tutoring Program II
- Where
- United States
- Volunteers
- 184 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
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
Stomach Neoplasms, Stomach Cancer
Hereditary Gastric Cancer Syndromes: An Integrated Genomic and Clinicopathologic Study of the Predisposition to Gastric Cancer
Background: Gastric cancers are cancers of the stomach. Hereditary ones are passed from parent to child. Researchers want to gather data about hereditary gastric cancers.
- Conditions
- Stomach Neoplasms, Stomach Cancer, Gastric Adenocarcinoma and Proximal Polyposis of the Stomach (GAPPS), Hereditary Diffuse Gastric Cancer (HDGC)
- Trial name
- NCT03030404
- Where
- United States
- Volunteers
- 733 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
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
Li-Fraumeni Syndrome, Hereditary Cancer Syndromes
Biomarker Monitoring in TP53 Mutation Carriers
Purpose This study is an 'N-of-one' observational study focusing on individuals with a hereditary predisposition to cancer due to a genetic mutation in the TP53 gene.
- Conditions
- Li-Fraumeni Syndrome, Hereditary Cancer Syndromes, TP53 Gene Germline Mutation Carrier
- Trial name
- NCT02289326
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Scripps Health
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma
- Trial name
- NCT03220035
- Testing
- Laboratory Biomarker Analysis, Vemurafenib
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 4 people
- Sponsor
- National Cancer Institute (NCI)
Tuberous Sclerosis
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy through the end of the 12 week…
- Trial name
- TSC (NCT04285346)
- Testing
- Ganaxolone
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Marinus Pharmaceuticals
Multiple Endocrine Neoplasia Syndromes
Registry for Multiple Endocrine Neoplasia Syndromes: MEN1/MEN2
Objectives: To contribute new and prospective data to our existing database library for patients with MEN1 and MEN2 at The University of Texas M.D. Anderson Cancer Center.
- Trial name
- NCT03048279
- Testing
- Questionnaires
- Where
- United States
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center
Wilms Tumor
Treatment of Newly Diagnosed Patient's With Wilm's Tumor Requiring Abdominal Radiation Delivered With Proton Beam Irradiation
Participants are being asked to take part in this clinical trial, a type of research study, because the participants have a Wilms Tumor cancer.
- Trial name
- NCT04968990
- Testing
- Proton Beam Radiation (PBRT), DD-4A Chemotherapy Regimen, Complete Surgical Resection
- Where
- United States
- Volunteers
- 260 people
- Sponsor
- St. Jude Children's Research 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
Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission
Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
This randomized phase III trial is studying how well Caphosol rinse works in preventing mucositis in young patients undergoing autologous or donor stem cell transplant.
- Conditions
- Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes
- Trial name
- NCT01305200
- Testing
- supersaturated calcium phosphate rinse, questionnaire administration, quality-of-life assessment
- Where
- 35 sites in United States, Australia and 1 more
- Volunteers
- 226 people
- Sponsor
- Children's Oncology Group
Ataxia-Telangiectasia
Study for Treatment of Cancer in Children With Ataxia-telangiectasia
This is a pilot/feasibility study designed to investigate the feasibility of treating children with Ataxia-Telangiectasia (A-T) and cancer with regimens nearly as intense as non-A-T patients with cancer would receive.
- Trial name
- NCT00187057
- Testing
- vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- St. Jude Children's Research Hospital
Tuberous Sclerosis Complex
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).
- Trial name
- ViRap (NCT04987463)
- Testing
- Vigabatrin, Rapamycin
- Where
- 2 sites in Poland
- Volunteers
- 60 people
- Sponsor
- Katarzyna Kotulska
Chronic Myeloproliferative Disorders, Kidney Cancer
Light-Emitting Diode Therapy in Preventing Mucositis in Children Receiving Chemotherapy With or Without Radiation Therapy Before Bone Marrow Transplantation
RATIONALE: Light-emitting diode (LED) therapy may be able to prevent mucositis of the mouth. PURPOSE: Randomized phase II trial to determine the effectiveness of LED therapy in preventing mucositis of the mouth in children who…
- Conditions
- Chronic Myeloproliferative Disorders, Kidney Cancer, Leukemia, Lymphoma
- Trial name
- NCT00036712
- Testing
- management of therapy complications, pain therapy
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Medical College of Wisconsin
Gait, Drop Foot, Chemotherapy-induced Peripheral Neuropathy
Impact of an Orthotic Intervention in Children With Peripheral Neuropathy
This research study will investigate the effect of two orthotic (brace) devices for the ankle and foot on walking and ankle flexibility in children with cancer not involving the brain or spinal cord.
- Conditions
- Gait, Drop Foot, Chemotherapy-induced Peripheral Neuropathy, Acute Lymphocytic Leukemia, Pediatric, Rhabdomyosarcoma
- Trial name
- IOPN (NCT03655587)
- Testing
- Solid ankle foot orthotic, Resting night splint
- Where
- 2 sites in United States
- Volunteers
- 27 people
- Sponsor
- Children's Hospitals and Clinics of Minnesota
Malformations, Multiple Abnormalies
Phenotype and Etiology of Pallister-Hall Syndrome
We aim to delineate the range of severity, natural history, molecular etiology, and pathophysiology of Pallister-Hall syndrome (PHS), Greig cephalopolysyndactyly syndrome (GCPS), McKusick-Kaufman syndrome (MKS), Bardet-Biedl…
- Conditions
- Malformations, Multiple Abnormalies, Polydactyly
- Trial name
- NCT00001404
- Where
- 4 sites in United States, Turkey (Türkiye)
- Volunteers
- 1,170 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Neurofibromatosis Type 1
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN.
- Trial name
- INSPIRE-NF1 (NCT06541847)
- Testing
- HLX-1502
- Where
- 14 sites in United States
- Volunteers
- 25 people
- Sponsor
- Healx Limited
Cancer
Methotrexate and Glucocorticoids in Treating Patients With Newly Diagnosed Acute Graft-Versus-Host Disease After Donor Stem Cell Transplant
RATIONALE: Methotrexate and glucocorticoid therapy, such as prednisone or methylprednisolone, may be an effective treatment for acute graft-versus-host disease caused by a donor stem cell transplant.
- Trial name
- NCT00357084
- Testing
- methotrexate, methylprednisolone, prednisone
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Fred Hutchinson Cancer Center
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma
- Trial name
- NCT00084695
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Milton S. Hershey Medical Center