Clinical trials: Gene Mutation-Related Cancer
Epstein-Barr Virus (EBV) Infections, Lymphoproliferative Disorders
Expanded Access Protocol for Tabelecleucel for Patients With Epstein-Barr Virus-Associated Viremia or Malignancies
The primary objective of this protocol is to provide expanded access to tabelecleucel to participants with Epstein-Barr virus-associated diseases and malignancies for whom there are no other appropriate therapeutic options, and…
- Conditions
- Epstein-Barr Virus (EBV) Infections, Lymphoproliferative Disorders, EBV+ Associated Lymphoma, EBV+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
- Trial name
- NCT02822495
- Testing
- tabelecleucel
- Sponsor
- Atara Biotherapeutics
ALL, Childhood, Pediatric Cancer
Pharmacogenomic Association Study in Indian Children With Acute Lymphoblastic Leukemia
A five-year prospective observational cohort study. The study is focused on observing the relation between static germline variants and therapeutic response in Indian children with acute lymphoblastic leukemia (ALL).
- Conditions
- ALL, Childhood, Pediatric Cancer, Toxicity, Drug, Adverse Drug Event
- Trial name
- MPGx-INDALL (NCT05512169)
- Where
- 2 sites in India
- Volunteers
- 556 people
- Sponsor
- University of Geneva, Switzerland
Pleuropulmonary Blastoma, Cystic Nephroma
DICER1-related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study
Background: Pleuropulmonary blastoma (PPB) is a rare fast-growing lung tumor that is associated with other, rare tumor types. Most cases of PPB appear in children younger than 6 years of age.
- Conditions
- Pleuropulmonary Blastoma, Cystic Nephroma, Ovarian Sertoli-Leydig Cell Tumors, Ocular Medulloepithelioma
- Trial name
- NCT01247597
- Where
- 2 sites in United States
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
NF2 Deficiency
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors.
- Trial name
- NCT06834438
- Testing
- ST002
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Core Binding Factor Acute Myeloid Leukemia, KIT Mutation-Related Tumors
Avapritinib in CBF-AML With KIT Mutations
AML with t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as CBF-AML. KIT mutations are common in CBF-AML, which have a worse prognosis.This study is aimed to evaluate the efficacy of Avapritinib, an highly…
- Trial name
- NCT05821738
- Testing
- Avapritinib
- Where
- China
- Volunteers
- 50 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Tuberous Sclerosis Complex
Dermatologic Patterns of Tuberous Sclerosis Patients and Somatic Mutation Relationship
Tuberous Sclerosis is a rare genetic disorder that affects about one in 15,000 individuals. It is part of the phacomatoses: a germline mutation of the gene Tuberous Sclerosis Complex 1 (TSC1) or TSC2 causes a protein dysfunction…
- Trial name
- NCT04112537
- Where
- France
- Volunteers
- 90 people
- Sponsor
- University Hospital, Montpellier
Acute Myeloid Leukemia
FLT3-ITD Gene Mutation and CD135 Expression in Acute Myeloid Leukemia.
1. To evaluate expression levels of CD135 2. To assess the frequency of FLT3 gene mutations (ITD) 3. association between FLT3-ITD mutation and CD135 expression and their correlation with hematological, immunophenotypic,and…
- Trial name
- NCT05383014
- Volunteers
- 82 people
- Sponsor
- Assiut University
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia
Personalized NK Cell Therapy in CBT
This phase II clinical trial studies how well personalized natural killer (NK) cell therapy works after chemotherapy and umbilical cord blood transplant in treating patients with myelodysplastic syndrome, leukemia, lymphoma or…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia, Acute Lymphoblastic Leukemia, Acute Lymphoblastic Leukemia in Remission
- Trial name
- NCT02727803
- Testing
- Allogeneic Natural Killer Cell Line NK-92, Anti-Thymocyte Globulin, Busulfan
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- M.D. Anderson Cancer Center
16P11.2 Deletion Syndrome, 16p11.2 Duplications
Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight
Simons Searchlight is an observational, online, international research program for families with rare genetic variants that cause neurodevelopmental disorders and may be associated with autism.
- Conditions
- 16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder)
- Trial name
- NCT01238250
- Where
- 2 sites in United States
- Volunteers
- 100,000 people
- Sponsor
- Simons Searchlight
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
RAS Mutation, Neurofibromatosis 1
RASopathy Biorepository
The RASopathies are a group of developmental disorders caused by genetic changes in the genes that compose the Ras/mitogen activated protein kinase (MAPK) pathway. New RASopathies are being diagnosed frequently.
- Conditions
- RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines
- Trial name
- NCT04395495
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations
Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies
This phase II trial studies reduced-intensity conditioning before donor stem cell transplant in treating patients with high-risk hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Acute Myeloid Leukemia With Inv(3) (q21.3;q26.2); GATA2, MECOM, Acute Myeloid Leukemia With t(6;9) (p23;q34.1); DEK-NUP214
- Trial name
- NCT01760655
- Testing
- Fludarabine phosphate, Busulfan, Total-Body Irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Acute Biphenotypic Leukemia, Acute Erythroid Leukemia in Remission
Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well donor peripheral blood stem cell (PBSC) transplant works in treating patients with hematologic malignancies.
- Conditions
- Acute Biphenotypic Leukemia, Acute Erythroid Leukemia in Remission, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia in Remission
- Trial name
- NCT01028716
- Testing
- Cyclophosphamide, Filgrastim, Fludarabine Phosphate
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Fred Hutchinson Cancer Center
BRCA Mutation, Ovarian Cancer
Prediction of Germline BRCA 1/2 Genes From Healthy Ovaries
The project aims at enhancing performance metrics and prospectively validating a radiogenomics model based on ovarian US images for predicting germline breast cancer susceptibility gene 1 and/or 2 (BRCA) status in women with…
- Conditions
- BRCA Mutation, Ovarian Cancer, Ultrasound Therapy; Complications
- Trial name
- PROBE-II (NCT05769517)
- Testing
- Germinal BRCA
- Where
- Italy
- Volunteers
- 6,465 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
PIK3CA-Related Overgrowth Spectrum (PROS), Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation)
Roll-Over Study of Alpelisib (BYL719) for Continued Access and Long-Term Safety.
The purpose of this study is to provide post-trial access to alpelisib and to assess its long-term safety when administered as a single agent or in combination with other drugs.
- Trial name
- EPIK-RO (NCT07679269)
- Testing
- Alpelisib, Fulvestrant, Trastuzumab
- Volunteers
- 51 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia, Lymphoma
Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies
This is a single-arm study evaluating allogeneic hematopoietic stem cell transplantation (HSCT) using mobilized peripheral blood stem cells from unrelated or partially matched related donors that are ex vivo depleted of α/β T…
- Conditions
- Leukemia, Lymphoma, Myelodysplasia
- Trial name
- AB-CliniMACs (NCT02323867)
- Testing
- CliniMACS α/β T-Cell and CD19+ B-Cell Depleted Peripheral Blood Stem Cell Transplant
- Where
- United States
- Volunteers
- 98 people
- Sponsor
- Children's Hospital of Philadelphia
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Natural Killer Cells Before and After Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia
This phase I/II studies the side effects and best dose of natural killer cells before and after donor stem cell transplant and to see how well they work in treating patients with acute myeloid leukemia, myelodysplastic syndrome…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Gene Mutations, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Trial name
- NCT01904136
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Bone Marrow Transplantation, Cyclophosphamide
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- M.D. Anderson Cancer Center
Prostatic Neoplasms
Prevalence of HRR-related Genes Mutations and Prognosis in Metastatic Castration Resistant Prostate Cancer (mCRPC) Patients in Real World Setting
The purpose of this study is to investigate the prevalence of tissue homologous recombination repair (HRR)-related gene mutations (positive/negative/Variant of uncertain significance (VUS)), clinical outcome such as…
- Trial name
- ZENSHIN (NCT04425200)
- Where
- 26 sites in Japan
- Volunteers
- 205 people
- Sponsor
- AstraZeneca
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia
Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)
The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not…
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Non-hodgkin Lymphoma
- Trial name
- BMT CTN 1702 (NCT03904134)
- Testing
- Donor Search Prognosis Score
- Where
- 52 sites in United States
- Volunteers
- 1,753 people
- Sponsor
- Center for International Blood and Marrow Transplant Research
Ewing Sarcoma, PEComa
Nivolumab (Opdivo®) Plus ABI-009 (Nab-rapamycin) for Advanced Sarcoma and Certain Cancers
This study investigates the safety/toxicity and potential anti-tumor activity of sequential administration of nivolumab and escalating doses of the mammalian target of rapamycin (mTOR) inhibitor nab-rapamycin (ABI-009) in…
- Conditions
- Ewing Sarcoma, PEComa, Epithelioid Sarcoma, Desmoid Tumor
- Trial name
- NCT03190174
- Testing
- Nab-Rapamycin, Nivolumab
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Sarcoma Oncology Research Center, LLC