Clinical trials: Fanconi Anemia
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
Fallopian Tube, Primary Peritoneal Cancer
Study of ABT-767 in Subjects With Breast Cancer 1 and Breast Cancer 2 (BRCA 1 and BRCA 2) Mutations and Solid Tumors or High Grade Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
This is a Phase 1, dose escalation trial evaluating the tolerability, pharmacokinetics, and pharmacodynamics of ABT-767 in subjects with advanced Breast Cancer 1 or 2 gene (BRCA1 or BRCA2)-mutated solid tumors and high grade…
- Conditions
- Fallopian Tube, Primary Peritoneal Cancer, Solid Tumors (e.g. Breast, Ovarian, Prostate, or Pancreatic) and Ovarian
- Trial name
- NCT01339650
- Testing
- ABT-767
- Where
- 3 sites in Netherlands
- Volunteers
- 93 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Fanconi Anemia
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor…
- Trial name
- NCT04784052
- Testing
- JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Porteus, Matthew, MD
Severe Combined Immunodeficiency, Fanconi Anemia
Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
Allogeneic stem cell transplantation (alloSCT) is utilized for various underlying diseases. AlloSCT is limited by graft versus host disease (GVHD), graft rejection, viral infections, and post-transplant lymphoproliferative…
- Conditions
- Severe Combined Immunodeficiency, Fanconi Anemia, Dyskeratosis Congenita, Sickle Cell Disease
- Trial name
- NCT06731036
- Testing
- CD34+ cell enriched stem cell graft
- Sponsor
- University of Florida
Melanoma, Li-Fraumeni Syndrome
Clinical Genetics Branch Eligibility Screening Survey
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding of cancer and to improve cancer care.
- Conditions
- Melanoma, Li-Fraumeni Syndrome, Pulmonary Blastoma, Chordoma
- Trial name
- NCT07005297
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Cancer Institute (NCI)
Fanconi Anemia
Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia
The main reason for this research study is to determine whether time-restricted eating will reduce inflammation in the bodies of persons with Fanconi anemia (FA) and whether time-restricted eating will improve function in people…
- Trial name
- NCT05598515
- Testing
- Time-Restricted Feeding
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hematologic Malignancies, Nonmalignant Diseases
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609827
- Testing
- Melphalan
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Breast Cancer, Ovarian Cancer
Risk Education and Assessment for Cancer Heredity
Individuals living in geographically underserved areas encounter considerable barriers to access of quality cancer genetic services.
- Conditions
- Breast Cancer, Ovarian Cancer, Genetic Predisposition
- Trial name
- REACH (NCT01346761)
- Testing
- Telephone Genetic Counseling, In-Person Genetic Counseling
- Where
- United States
- Volunteers
- 1,012 people, healthy volunteers welcome
- Sponsor
- University of Utah
Anemia, Aplastic, Fanconi Anemia
Cord Blood Stem Cell Transplantation Study (COBLT)
To evaluate if HLA-mismatched, unrelated-donor umbilical cord blood stem and progenitor cell units (UCBU) offered a clinically acceptable alternative to matched unrelated-donor allogeneic bone marrow for transplantation with…
- Conditions
- Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia
- Trial name
- NCT00000603
- Testing
- stem cell transplantation
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Fanconi Anemia
Bone Marrow Cell Gene Transfer 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 bone marrow cells that produce various types of blood cells.
- Trial name
- NCT00272857
- Testing
- Retrovirus Construct
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Boston Children's Hospital
Head Injuries, Subarachnoid Haemorrhage
Impact of Cranioplasty On Cerebral Perfusion
The purpose of this study is to examine the impact of cranioplasty on cerebral hemodynamic and blood flow as prognostic factor in patients receiving decompressive craniectomy for Head injuries, Subarachnoid haemorrhage…
- Conditions
- Head Injuries, Subarachnoid Haemorrhage, Intra-cerebral Haemorrhage, Cerebral Thrombosis
- Trial name
- CCP (NCT01113645)
- Testing
- Cerebral perfusion evaluation
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Metastatic Breast Cancer, Invasive Breast Cancer
Olaparib In Metastatic Breast Cancer
This research study is for patients with metastatic breast cancer. Metastatic means that the cancer has spread beyond the breast.
- Conditions
- Metastatic Breast Cancer, Invasive Breast Cancer, Somatic Mutation Breast Cancer (BRCA1), Somatic Mutation Breast Cancer (BRCA2)
- Trial name
- NCT03344965
- Testing
- Olaparib
- Where
- 20 sites in United States
- Volunteers
- 114 people
- Sponsor
- Beth Israel Deaconess Medical Center
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Background: Fanconi anemia (FA) is an inherited disorder. People with FA are more likely to get certain cancers, especially squamous cell carcinoma (SCC).
- Trial name
- NCT05687149
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Cancer Institute (NCI)
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, Severe Aplastic Anemia
Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia
The purpose of this study is to discover whether children and adults with Fanconi anemia (FA) can be safely and effectively transplanted with Human Leukocyte Antigen (HLA) mismatched (up to one haplotype), HLA-matched sibling, or…
- Trial name
- Mafia (NCT00590460)
- Testing
- CAMPATH-1H, Anti-CD45, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Baylor College of Medicine
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
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
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)
Fanconi Anemia
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow.
- Trial name
- NCT00630253
- Testing
- Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Masonic Cancer Center, University of Minnesota