Clinical trials: Aplasia
Complications of Organ Transplant Stem Cells Umbilical Cord
Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived）
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment.
- Trial name
- MSC-SAA (NCT02218437)
- Testing
- MSC+ATG
- Where
- China
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Chinese Academy of Medical Sciences
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)
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant
This phase I/II trial studies how well cytokine-treated veto cells work in treating patients with hematologic malignancies following stem cell transplant.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Aplastic Anemia, Bone Marrow Failure
- Trial name
- NCT03622788
- Testing
- Anti-Thymocyte Globulin, Cyclophosphamide, Cytokine-treated Veto Cells
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center
Mrk Anomaly, Infertility, Female
Uterus Transplantation LD - Feasibility Study - Sahlgrenska - BMC
Medical know-how concerning uterus transplantation will be transferred to new site by structured process involving theory, animal workshops and a clinical feasibility study on human subjects.
- Trial name
- GOT-BMC-UTx (NCT03590405)
- Testing
- uterus transplantation surgery and IVF
- Where
- 2 sites in Sweden
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Sahlgrenska University Hospital
Corpus Callosum Agenesis
Decision-making Process of Couples Confronted With Prenatal Diagnosis of an Isolated CCA
This research promoted by the AP-HP (DRCI) is financed by the Agence de la biomédecine (ABM) and the Fondation Maladie Rare (FMR) and received a favourable opinion from the CPP on 8/12/2020.
- Trial name
- ACCED (NCT05843110)
- Testing
- Decision-making process
- Where
- France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Abnormality, Congenital
Relationship of Muscular Agenesis: Thin Plantar and Palmar Long as a Biomarker of Injury and Performance Marker in the Lower Limb and Upper Limb Respectively
This study investigates muscle agenesis, specifically the absence of the palmaris longus and plantaris muscles, and its effects on injury risk, strength, and performance.
- Trial name
- MA-TP-PL (NCT06448832)
- Testing
- Ultrasound
- Where
- Spain
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- University of Seville
Aplastic Anemia, Leukemia
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
This is a genetic disease (transmitted through the parents' genes) called Fanconi Anemia. Because of that genetic disease, the bone marrow has changed and now has failed, or has given rise to a preleukemia called myelodysplastic…
- Conditions
- Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00987480
- Testing
- Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
- Where
- 6 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Primary Immune Thrombocytopenic Purpura, Amegakaryocytic Aplasia
Ultrasound to Detect Splenomegally in Patients With Isolated Thrombocytopenia
Investigate prevalence of splenomegally in patients with isolated thrombocytopenia. Asess effect of splenomegally in clinical picture ,Complication, treatment response and disease outcome To explain possible causes of…
- Conditions
- Primary Immune Thrombocytopenic Purpura, Amegakaryocytic Aplasia, Unilineage Myelodysplastic Syndrome ( Megakaryocyte Dysplasia), Lymphoproliferative Disorder With Secondary ITP
- Trial name
- NCT07182760
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut University
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
FANCONI ANEMIA
Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT
While stem cell transplantation has proven an effective means of treating a wide variety of diseases involving hematopoietic stem cells and their progeny, a shortage of donors has proved a major impediment to the widest…
- Trial name
- RAFHAS (NCT00586274)
- Testing
- CD34 selected haploidentical PBSCT, Fludarabine, T cell infusion
- Where
- 2 sites in United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Mayer Rokitansky Kuster Hauser Syndrome
Feasibility Study of Uterine Transplantation From Living Donors in Terms of Efficacy and Safety in Patients With Mayer-Rokitansky-Küster-Hauser Syndrome (MRKH)
In France, one in 4500 women is affected by the MayerRokitantskyKüsterHauser (MRKH) syndrome which is characterized by the absence of uterus at birth.
- Trial name
- NCT03689842
- Testing
- Uterine transplantation
- Where
- France
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Hopital Foch
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
Aplastic Anemia, Drug Effect
A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary TD-NSAA
Aplastic anemia (AA) is a group of clinical syndromes. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but…
- Trial name
- NCT06004791
- Testing
- Herombopag + CsA, rhTPO combined with Herombopag + CsA
- Where
- China
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
Small Penis, Penile Enhancement
Clinical Study to CMDHA0101 as Compared to PowerFill® for Temporary Penile Enhancement
The investigators aimed to confirm the efficacy and safety of transdermal penis enlargement for 24 weeks after CMDHA0101 injection in subjects who wanted penile enlargement in male patients with small-penis syndrome *.
- Trial name
- NCT03153735
- Testing
- CMDHA0101, PowerFill®
- Where
- South Korea
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- CHA MEDITECH Co., Ltd.
Mayer Rokitansky Kuster Hauser Syndrome, Uterine Agenesis
The University of Pennsylvania Uterus Transplant for Uterine Factor Infertility Trial
Options for childbearing are limited for the thousands of women in the United States who suffer from absolute uterine factor infertility. Uterine transplantation is an emerging treatment that provides hope for these individuals.
- Conditions
- Mayer Rokitansky Kuster Hauser Syndrome, Uterine Agenesis, Infertility, Female, of Uterine Origin, Uterine Factor Infertility
- Trial name
- UNTIL (NCT03307356)
- Testing
- Uterine Transplant
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Mayer Rokitansky Kuster Hauser Syndrome
Molecular Genetic Study of Mayer-Rokitansky-Kuster-Hauser Syndrome
In order to understand the molecular mechanisms leading to Mayer-Rokitansky-Kuster-Hauser syndrome (MRKH), the research team has to identify molecular bases of this anomaly.
- Trial name
- MRKH (NCT02967822)
- Testing
- Biological samples for patients, Biological samples for healthy relatives
- Where
- 2 sites in France
- Volunteers
- 410 people, healthy volunteers welcome
- Sponsor
- Imagine Institute
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
AML, ALL
Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders
Allogeneic stem cell transplantation may provide long-term remissions for some patients with hematological malignancies. However, allogeneic transplantation is associated with a significant risk of potentially life threatening…
- Conditions
- AML, ALL, CLL, Myelodysplastic Syndrome
- Trial name
- NCT00533923
- Testing
- Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSF
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center