Clinical trials: Bone Marrow Failure, open to volunteers
Aplastic Anemia
Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)
- Trial name
- NCT07572084
- Testing
- Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital
Idiopathic Pulmonary Fibrosis, Emphysema or COPD
Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore…
- Trial name
- NCT03500731
- Testing
- CD3/CD19 negative hematopoietic stem cells, Rituximab, Alemtuzumab
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Paul Szabolcs
Lymphoblastic Leukemia, Cancer Survivors
Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors
This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle quality and VO2max in AYA HCT survivors.
- Conditions
- Lymphoblastic Leukemia, Cancer Survivors, Aplastic Anemia, Myelodysplastic Syndromes
- Trial name
- IAMFIT (NCT05194397)
- Testing
- Nicotinamide Riboside, Exercise Intervention
- Where
- 3 sites in United States
- Volunteers
- 80 people
- Sponsor
- Children's Hospital of Philadelphia
Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
This clinical trial tests next generation sequencing (NGS) for the detection of precursor features of pre-myeloid cancers and bone marrow failure syndromes.
- Conditions
- Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion, Cytopenia
- Trial name
- NCT02958462
- Testing
- Biospecimen Collection, Bone Marrow Biopsy, Punch Biopsy
- Where
- 3 sites in United States
- Volunteers
- 2,000 people
- Sponsor
- Mayo Clinic
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
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
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) +eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe…
- Trial name
- NCT07558434
- Testing
- Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital
Fanconi Anemia, Anemia, Hypoplastic, Congenital
Prenatal Transplantation for Fetuses With Fanconi Anemia
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
- Conditions
- Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders
- Trial name
- NCT07408583
- Testing
- IUHSCT for FA-affected fetuses
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Agnieszka Czechowicz
Alcohol-Related Carcinoma, Fanconi Anemia
Role of Acetaldehyde in the Development of Oral Cancer
This is a minimal risk intervention study where healthy volunteers and individuals with Fanconi anemia will consume a single dose of alcohol and provide primarily non-invasive biological samples at various time points.
- Conditions
- Alcohol-Related Carcinoma, Fanconi Anemia, Oral Cavity Carcinoma
- Trial name
- NCT05973656
- Testing
- Alcohol, Biospecimen Collection, Breath Test
- Where
- United States
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Masonic Cancer Center, University of Minnesota
Aplastic Anaemia
A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia
Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells.
- Trial name
- NCT07499284
- Testing
- Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells, Haploidentical hematopoietic cell transplantation
- Where
- 4 sites in China
- Volunteers
- 224 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Severe Aplastic Anemia
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells.
- Trial name
- NCT04304820
- Testing
- Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hematopoietic Stem Cell Transplantation (HSCT), Acute Myeloid Leukemia (AML)
High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)
The High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) study is the first prospective real-world cohort of hematologic diseases and transplantation in the Qinghai-Tibet Plateau.
- Conditions
- Hematopoietic Stem Cell Transplantation (HSCT), Acute Myeloid Leukemia (AML), Leukemias, Acute Myeloid, Myeloid Leukemias, Acute
- Trial name
- HALO-SCT (NCT07205523)
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Yigeng Cao,MD,PhD
Chronic Myeloid Leukemia (CML)
Second STOP After Pioglitazone Priming in CML Patients
Single-center study, prospective, phase II trial. The study objectives are : To assess safety and pharmacokinetics of the combination of PIO and TKI in CML subjects who experience a loss of MMR following a first TKI…
- Trial name
- PIO2STOP (NCT02889003)
- Testing
- Pioglitazone + TKI
- Where
- 6 sites in France
- Volunteers
- 26 people
- Sponsor
- Versailles Hospital
Aplastic Anemia, Myelodysplastic Syndromes
Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRS
Anemia is a global public health concern and is closely linked to dysfunction of brain cognitive neural networks, a key mechanism underlying cognitive impairment.
- Conditions
- Aplastic Anemia, Myelodysplastic Syndromes, Autoimmune Hemolytic Anemia, Functional Near-Infrared Spectroscopy
- Trial name
- AN-CNA (NCT07578675)
- Where
- China
- Volunteers
- 323 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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)
Myelodysplastic Syndromes
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults.
- Trial name
- NCT06303193
- Testing
- pacritinib
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Cancer Institute (NCI)
Recurrent Severe Aplastic Anemia, Refractory Severe Aplastic Anemia
A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia
This phase I trial evaluates the safety and feasibility of using a reduced-intensity regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin prior to a CD4+ T-cell depleted haploidentical hematopoietic cell…
- Trial name
- NCT05757310
- Testing
- Anti-Thymocyte Globulin, Biospecimen Collection, Bone Marrow Aspirate
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
Pancytopenia
Clinical, Hematological and Pathological Profile of Pancytopenia
Describe clinical characteristic, peripheral blood parameters and bone marrow examination in pancytopenia patients and Analyze etiology of pancytopenia and study the outcomes regarding duration of hospital stay.
- Trial name
- NCT07785726
- Volunteers
- 140 people
- Sponsor
- Assiut University
Primary Immune Deficiency Disorder, Immune Deficiency Disease
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune…
- Conditions
- Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length
- Trial name
- NCT04232085
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins