Clinical trials: Fanconi Anemia
Bone Marrow Failure Syndrome
French National Registry of Bone Marrow Failures
This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France (multicenter registry), from diagnosis and…
- Trial name
- RIME (NCT04781790)
- Testing
- Bone Marrow Failure
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Fanconi Anemia
Corrective Exercise and Rehabilitation in Fanconi Anemia: A Case Study
Introduction: Fanconi anemia (FA) is a rare autosomal recessive DNA repair disorder characterized by congenital malformations, progressive bone marrow failure, and reduced quality of life.
- Trial name
- NCT06958380
- Testing
- Corrective exercise and rehabilitation
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Pardis Specialized Wellness Institute
Fanconi Anemia
Stem Cell Transplantation for Fanconi Anemia
The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched…
- Trial name
- NCT00167206
- Testing
- Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Breast Cancer
High Risk Breast Cancer Screening Program
Objectives: To screen population with high risk for breast cancer To promote the concept and importance of surveillance mammography To find out the incidence of breast cancer in population who are in high risk for breast cancer…
- Trial name
- NCT00509509
- Where
- Hong Kong
- Sponsor
- Organisation for Oncology and Translational Research
BRCA1 Gene, BRCA2 Gene
A Qualitative Exploration of the Impact of Positive BRCA1/2 Mutation Status on the Lives of Young Women
Background: Mutations detected in the BRCA1 or BRCA2 genes have been show to produce very high risks of breast and ovarian cancer (as high as 90% and 60%, respectively), as well as accompanying risks for several other types of…
- Trial name
- NCT00858078
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- National Cancer Institute (NCI)
Breast Cancer, Ovarian Cancer
Methods in Education for Breast Cancer Genetics
In 1997, the Genetics Department of the NCI Medicine Branch helped establish a breast cancer genetics program at the National Naval Medical Center s Breast Care Center.
- Trial name
- NCT00001806
- Testing
- Genetic Education and Counseling
- Where
- United States
- Volunteers
- 170 people
- Sponsor
- National Cancer Institute (NCI)
Fanconi Anemia, Anemia, Hypoplastic, Congenital
Prenatal Transplantation for Fetuses With Fanconi Anemia
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
- Conditions
- Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders
- Trial name
- NCT07408583
- Testing
- IUHSCT for FA-affected fetuses
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Agnieszka Czechowicz
Fanconi Anemia
Hematopoietic Stem Cell Transplant for Fanconi Anemia
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan…
- Trial name
- FA (NCT01071239)
- Testing
- CliniMACs device, Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Medical College of Wisconsin
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Pancreatic Cancer Metastatic
Vilastobart+Retifanlimab in BRCA or PALB2 Deficient PC
This is a single arm, open-label, non-randomized multi-institution phase II trial of a Fc enhanced CTLA4 antibody, vilastobart, in combination with PD-1 antibody, retifanlimab, in patients with BRCA1, BRCA2 or PALB2 deficient…
- Trial name
- EKLAVYA-HRD (NCT07765836)
- Testing
- vilastobart (XTX101), Retifanlimab
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Massachusetts General Hospital
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Fanconi's Anemia
Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
OBJECTIVES: I. Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia. II. Determine the efficacy of this treatment regimen in this patient population. III.
- Trial name
- NCT00006127
- Testing
- amifostine
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Dana-Farber Cancer Institute
Fanconi Anemia
Blood Cell Collection for Future Use in Individuals With Fanconi Anemia
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells.
- Trial name
- NCT00271089
- Testing
- PBSC Collection, Bone Marrow Harvest, CliniMacs Cell Selection System
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia
Gene Therapy for Fanconi Anemia
This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA).
- Trial name
- NCT01331018
- Testing
- Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Fred Hutchinson Cancer Center
Fanconi Anemia
Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi…
- Trial name
- FANCOLEN-1 (NCT03157804)
- Testing
- IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs), Genetically Engineered Hematopoietic Stem/Progenitor Cells, Laboratory Biomarker Analysis
- Where
- 2 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Fanconi Anemia
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated…
- Trial name
- NCT05910853
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sanguine Biosciences
Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I
Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease
The goal of this study is to determine if the Lactate Plus meter is accurate compared to lab lactate levels, and to determine if the Accu chek guide glucometer is accurate compared to lab serum glucose levels in patients with…
- Conditions
- Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB, Glycogen Storage Disease Xi
- Trial name
- NCT07459582
- Testing
- Lactate Meter, Accu Chek
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Connecticut Children's Medical Center
Chronic Myeloproliferative Disorders, Fanconi Anemia
Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of…
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00900055
- Testing
- microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction
- Where
- United States
- Volunteers
- 213 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
Triple Negative Breast Neoplasms, Triple Negative Breast Cancer
Novel Neoadjuvant and Adjuvant Strategy for Germline BRCA 1/2 Mutated Triple Negative Breast Cancer
This is a Phase II, single-arm, open label study to evaluate Olaparib plus Pembrolizumab following platinum-based chemotherapy plus Pembrolizumab as neoadjuvant therapy for germline BRCA (gBRCA) 1/2 mutated triple negative breast…
- Conditions
- Triple Negative Breast Neoplasms, Triple Negative Breast Cancer, Breast Neoplasms, Breast Cancer
- Trial name
- NCT05485766
- Testing
- Pembrolizumab, Paclitaxel, Carboplatin
- Where
- 3 sites in Japan
- Volunteers
- 23 people
- Sponsor
- Okayama University