Clinical trials: Fanconi Anemia Complementation Group A
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
Advanced Cancers (Breast, Ovarian, mCRPC, Pancreatic Ductal Adenocarcinoma (PDAC), Brain Mets) With Specific Mutations (Homologous Recombination Deficiency)
Study to Assess Safety, Tolerability and Activity of DSB2455 in Participants With Advanced Malignancies
Open label, multi-centre, Phase Ia/b adaptive design study with an initial 2-stage Dose Escalation Phase followed by a Dose Expansion Phase.
- Trial name
- NCT06458712
- Testing
- DSB2455
- Where
- 21 sites in United States, France and 3 more
- Volunteers
- 180 people
- Sponsor
- Duke Street Bio Ltd
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
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
Fanconi's Anemia, Hematologic Disease
Medical Treatment for Diamond Blackfan Anemia
Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it.
- Trial name
- NCT00001749
- Testing
- Antithymocyte globulin, Cyclosporine
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease
- Trial name
- NCT00290628
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fanconi Anemia, Squamous Cell Carcinoma
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous…
- Trial name
- NCT03476330
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Dyskeratosis Congenita, Fanconi Anemia
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.
- Conditions
- Dyskeratosis Congenita, Fanconi Anemia, Myelodysplastic Syndromes, Pearson Marrow-pancreas Syndrome
- Trial name
- NCT00499070
- Testing
- polymerase chain reaction, flow cytometry, immunologic technique
- Where
- 10 sites in Austria, Belgium and 8 more
- Volunteers
- 119 people
- Sponsor
- University Hospital Freiburg
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
Fanconi Anemia
Prognostic Significance of Mutation Type and Chromosome Fragility in Fanconi Anemia
The goal of this observational study is to analyze the data included in the Spanish Registry of Patients with Fanconi anemia to better understand the natural history of the disease, identify genetic risk and prognostic factors…
- Trial name
- NCT06490510
- Where
- Spain
- Volunteers
- 227 people
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Fanconi Anemia, Autosomal or Sex Linked Recessive Genetic Disease
Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents
Fanconi anemia is a rare autosomal or sex linked recessive genetic disease. The disease is characterized by bone marrow hematopoiesis failure, multiple congenital abnormalities, and susceptibility to neoplastic diseases.
- Conditions
- Fanconi Anemia, Autosomal or Sex Linked Recessive Genetic Disease, Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases., Hematopoiesis Maintainance.
- Trial name
- NCT01995305
- Testing
- human whole exome, whole genomic
- Where
- China
- Sponsor
- Xiaofan Zhu
Ovarian Neoplasm
Study to Assess the Efficacy and Safety of a PARP Inhibitor for the Treatment of BRCA-positive Advanced Ovarian Cancer
The purpose of the study is to see if the drug KU 0059436 is effective and well tolerated in treating patients with measurable BRCA1- or BRCA2-positive advanced ovarian cancer and for whom no curative therapeutic option exists.
- Trial name
- ICEBERG 2 (NCT00494442)
- Testing
- KU-0059436 (AZD2281)(PARP inhibitor)
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 58 people
- Sponsor
- AstraZeneca
Fanconi Anemia, Severe Aplastic Anemia
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine…
- Conditions
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion
- Trial name
- NCT03579875
- Testing
- Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi's Anemia, Myelodysplastic Syndromes
Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and radiotherapy in terms of improving survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with…
- Conditions
- Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
- Trial name
- NCT00005892
- Testing
- cyclophosphamide, Allogeneic Bone Marrow Transplantation
- Where
- United States
- Sponsor
- Fairview University 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)
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
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
Ovarian Neoplasms
Dose-finding Study Comparing Efficacy and Safety of a PARP Inhibitor Against Doxil in BRCA+ve Advanced Ovarian Cancer
The purpose of the study is to compare the efficacy and safety of 2 doses of drug AZD2281 against liposomal doxorubicin to see which is effective and well tolerated in treating patients with measurable BRCA1- or BRCA2-positive…
- Trial name
- ICEBERG 3 (NCT00628251)
- Testing
- AZD2281, Liposomal Doxorubicin
- Where
- 24 sites in United States, Australia and 7 more
- Volunteers
- 97 people
- Sponsor
- AstraZeneca
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum