Clinical trials: Inherited Bone Marrow Failure Syndrome
Fanconi Anemia
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia.
- Trial name
- NCT06519786
- Testing
- Metformin
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Ain Shams University
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell…
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT03047746
- Testing
- CliniMACs
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Philadelphia
Cancer
Antithymocyte Globulin, Clofarabine, and Rituximab in Treating Patients After an Unsuccessful Stem Cell Transplant
RATIONALE: Antithymocyte globulin, clofarabine, and rituximab may stop the patient's immune system from rejecting the donor's stem cells when they do not exactly match the patient's blood.
- Trial name
- NCT00617929
- Testing
- anti-thymocyte globulin, rituximab, clofarabine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia
Based on success in other diseases, the Fred Hutchinson Cancer Research Center (FHCRC) has developed a transplant procedure for Fanconi anemia (FA), which does not completely destroy the patient's remaining bone marrow.
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00093743
- Testing
- fludarabine phosphate, cyclosporine, total-body irradiation
- Where
- 5 sites in United States
- Volunteers
- 2 people
- Sponsor
- Fred Hutchinson Cancer Center
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
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
Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia
Background: Fanconi anemia (FA) is a rare, inherited cancer syndrome. FA causes a range of physical issues. Children with FA may have abnormal features; these may include a small head and eyes and issues with their internal…
- Trial name
- NCT06744283
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Leukemia, Lymphoma
Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation
This study tests whether a combination of three treatments - dexamethasone (a steroid), intravenous immunoglobulin (IVIG, a protein that helps the immune system), and an extra dose of donor mononuclear cells - can safely lower…
- Conditions
- Leukemia, Lymphoma, Thalassemia, Aplastic Anemia
- Trial name
- NCT07698080
- Testing
- Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells
- Volunteers
- 60 people
- Sponsor
- Hematology department of the 920th hospital
Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of illness and death.
- Conditions
- Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
- Trial name
- NCT01659606
- Testing
- alemtuzumab, Fludarabine, Cyclosporins
- Where
- 13 sites in United States, Norway and 1 more
- Volunteers
- 40 people
- Sponsor
- Boston Children's Hospital
Telomere Biology Disorders, Dyskeratosis Congenita
Nucleoside Therapy in Patients With Telomere Biology Disorders
The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology disorders.
- Conditions
- Telomere Biology Disorders, Dyskeratosis Congenita, Revesz Syndrome, Hoyeraal Hreidarsson Syndrome
- Trial name
- NCT06817590
- Testing
- deoxycytidine, deoxythymidine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Suneet Agarwal
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Collecting and Storing Blood Samples From Patients With Cancer
RATIONALE: Collecting and storing samples of blood from patients with cancer to test in the laboratory may help the study of cancer in the future.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00900120
- Sponsor
- University of Medicine and Dentistry of New Jersey