Clinical trials: Bone Marrow Failure
Severe Aplastic Anemia, Telomere Biology Disorders
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking.
- Conditions
- Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT05012111
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood…
- Trial name
- NCT06090669
- Testing
- TruSight Oncology, imatinib
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol.
- Conditions
- Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder
- Trial name
- 2023-12377081 (NCT07877727)
- Where
- 4 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Istituto Giannina Gaslini
Bone Marrow Failure Disorders, VEXAS Syndrome
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
STUDY DESCRIPTION: Bone marrow failure can result from immune mediated attack on stem cells or from inherited genetic defects such as telomere biology disorders or Fanconi anemia.
- Conditions
- Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes
- Trial name
- NCT07102849
- Where
- United States
- Volunteers
- 1,400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Bone Marrow Failure Syndrome
Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders
The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to…
- Trial name
- NCT07585136
- Testing
- Filgrastim, Plerixafor, Leukapheresis
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- St. Jude Children's Research Hospital
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)
Aplastic Anemia, Bone Marrow Failure Syndrome
Haploidentical HCT for Severe Aplastic Anemia
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation.
- Trial name
- NCT04558736
- Testing
- Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- St. Jude Children's Research Hospital
Aplastic Anemias, Hemoglobinopathies
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood…
- Conditions
- Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
- Trial name
- HOPE Resource (NCT07227155)
- Where
- 14 sites in United States
- Volunteers
- 375 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Aplastic Anemia
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment
This research study is testing a medicine called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow does not produce enough blood cells.
- Trial name
- NCT07786922
- Testing
- Avatrombopag 20 mg Oral Tablet
- Volunteers
- 26 people
- Sponsor
- Swedish Orphan Biovitrum
Acute Myeloid Leukemia, Myelodysplastic Syndromes
A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies
The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid…
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloid Malignancies, MDS
- Trial name
- MORE (NCT05796570)
- Testing
- Decitabine, Filgrastim
- Where
- 2 sites in United States
- Volunteers
- 29 people
- Sponsor
- Franziska Wachter
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
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Hematopoietic Cell Transplant
The MedMemos Study: Promoting Adherence to Medications in Pediatric Bone Marrow Transplant Patients
The goal of this clinical trial is to test the MedMemos Intervention with parents and children who have received a bone marrow transplant.
- Trial name
- MedMemos (NCT07718204)
- Testing
- MedMemos
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nationwide Children's Hospital
Leukemia, Bone Marrow Failure Syndrome
Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC
The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT).
- Conditions
- Leukemia, Bone Marrow Failure Syndrome, Immunodeficiencies
- Trial name
- NCT03145545
- Testing
- Apha/beta T and CD19+ cell depletion using CliniMACS device
- Where
- United States
- Sponsor
- Children's Hospital of Philadelphia
Hemoglobinopathy (Disorder), Severe Aplastic Anemia
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition.
- Conditions
- Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
- Trial name
- NCT04356469
- Testing
- Haploidentical Hematopoietic Cell Transplantation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins All Children's Hospital
Steroid-refractory Diamond-Blackfan Anemia (DBA)
Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia
Background: Diamond-Blackfan anemia (DBA) is an inherited disease that affects the bone marrow. People with DBA have chronic anemia that can be severe. Many must have frequent transfusions of red blood cells.
- Trial name
- NCT05828108
- Testing
- Bitopertin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)