Clinical trials: Fanconi Anemia Complementation Group A
Fanconi Anemia
TBI Dose De-escalation for Fanconi Anemia
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
- Trial name
- NCT00352976
- Testing
- Cyclophosphamide, Fludarabine, Total Body Irradiation
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fanconi Anemia
Unrelated HSCT in Patients With Fanconi Anemia
The protocol is designed for the compassionate treatment of patients with Fanconi Anemia who do not have an HLA-matched sibling donor.
- Trial name
- NCT02127905
- Testing
- CD34+ selected cells
- Where
- United States
- Sponsor
- Neena Kapoor, M.D.
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
Fanconi Anemia
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy.
- Trial name
- NCT00258427
- Testing
- anti-thymocyte globulin, filgrastim, busulfan
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Leukemia, Myelodysplastic Syndromes
Evaluating Cell Damage in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, or Fanconi Anemia; in Patients Who Were Exposed to Alkylating Agents; and in Healthy Volunteers
RATIONALE: Studying samples of bone marrow from patients with cancer and from healthy volunteers in the laboratory may help doctors learn more about changes that occur in bone marrow stromal (connective tissue) cells.
- Trial name
- NCT00899795
- Testing
- cytogenetic analysis, fluorescence in situ hybridization, flow cytometry
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
Fanconi Anemia Complementation Group A
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the…
- Trial name
- NCT03814408
- Testing
- RP-L102
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Fanconi Anemia, Dyskeratosis Congenita
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects…
- Trial name
- NCT01001598
- Testing
- danazol
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Boston Children's Hospital
Fanconi Anemia
Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
The purpose of this study is to evaluate the safety of the drug oxandrolone (a type of androgen steroid) in patients with Fanconi anemia (FA), and to determine if this drug can help in the treatment of bone marrow failure in…
- Trial name
- NCT00243399
- Testing
- Oxandrolone
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Microcephaly
Microcephaly Genetic Deficiency in Neural Progenitors
The purpose of this study is to: I. Compare neuroradiological phenotype and cognitive functioning of MCPH patients caused by ASPM mutations already characterized and published (Passemard et al.
- Trial name
- MICROFANC (NCT01565005)
- Where
- France
- Volunteers
- 98 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Fanconi Anemia Complementation Group A
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
- Trial name
- NCT04069533
- Testing
- RP-L102
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Hematologic Malignancies, Nonmalignant Diseases
Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation
Fludarabine 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 fludarabine in their bodies and if drug…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- HCT (NCT01316549)
- Testing
- Fludarabine
- Where
- United States
- Volunteers
- 67 people
- Sponsor
- University of California, San Francisco
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
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)