Popular Trials
Thrombopoietin Receptor Agonist
Eltrombopag for Aplastic Anemia
This trial is evaluating the safety and effectiveness of eltrombopag, a drug given by mouth, in people with moderate aplastic anemia or bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts.
Hematopoietic Stem Cell Transplantation
Haploidentical Transplant for Severe Aplastic Anemia
This trial is for patients with Severe Aplastic Anemia who will receive a haploidentical transplantation (from a family member that is not fully matched). The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year.
Monoclonal Antibodies
REGN7257 for Severe Aplastic Anemia
This trial is testing a new drug called REGN7257 for patients with severe aplastic anemia who haven't responded to other treatments. The drug aims to help the bone marrow produce more blood cells and reduce the need for transfusions. The study will check if the drug is safe and how well it works.
Immunosuppressant
Cyclosporine + Eltrombopag for Aplastic Anemia
This trial is testing whether a lower dose of cyclosporine with eltrombopag is safe and effective in people with severe aplastic anemia who have not been treated with immunosuppressive therapy and eltrombopag.
T Cell Depletion
Stem Cell Transplant for Blood Cancer
This trial is testing a new combination of chemotherapy and low-dose radiation to see if it is safe and effective in treating bone marrow cancer. The CliniMACS device is approved for one type of T cell depletion but not the type being studied in this trial. This trial will help determine if the CliniMACS device should be approved for the other type of T cell depletion.
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Trials for MDS Patients
CAR T-cell Therapy
Gene Modified T-cells for Blood Disorders
This trial will look at whether T cells from a family member can help a patient's immune system recover after a stem cell transplant, with the added safety measure of a self-destruct switch.
Alkylating agents
Stem Cell Transplant for Severe Aplastic Anemia
This trial is for people with severe aplastic anemia, myelodysplastic syndrome, or paroxysmal nocturnal hemoglobinuria who want to receive a stem cell transplant using peripheral blood stem cells from a family member.
Stem Cell Transplantation
Umbilical Cord Blood Transplant for Aplastic Anemia and Myelodysplastic Syndrome
This trial is testing if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.
Alkylating agents
Cord Blood Transplant for Blood Diseases
This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.
Trials for Myelodysplastic Syndrome Patients
CAR T-cell Therapy
Gene Modified T-cells for Blood Disorders
This trial will look at whether T cells from a family member can help a patient's immune system recover after a stem cell transplant, with the added safety measure of a self-destruct switch.
Stem Cell Transplantation
Umbilical Cord Blood Transplant for Aplastic Anemia and Myelodysplastic Syndrome
This trial is testing if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.
Alkylating agents
Cord Blood Transplant for Blood Diseases
This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.
Trials for HLA Positive Patients
CAR T-cell Therapy
Gene Modified T-cells for Blood Disorders
This trial will look at whether T cells from a family member can help a patient's immune system recover after a stem cell transplant, with the added safety measure of a self-destruct switch.
Alkylating agents
Haploidentical Bone Marrow Transplant for Aplastic Anemia
This trial is testing a new treatment for severe aplastic anemia, which is a rare and serious form of bone marrow failure. The new treatment is using a haploidentical donor for transplantation, which has good response rates and significantly lower rates of acute and chronic GVHD.
Stem Cell Therapy
CD34+ Stem Cell Selection for Bone Marrow Failure Syndromes
This trial uses a special machine to filter out harmful cells from donor blood to make stem cell transplants safer for young patients with non-cancerous diseases. By removing specific cells, it aims to prevent a serious immune reaction.
Trials With No Placebo
CAR T-cell Therapy
Gene Modified T-cells for Blood Disorders
This trial will look at whether T cells from a family member can help a patient's immune system recover after a stem cell transplant, with the added safety measure of a self-destruct switch.
Alkylating agents
Haploidentical Bone Marrow Transplant for Aplastic Anemia
This trial is testing a new treatment for severe aplastic anemia, which is a rare and serious form of bone marrow failure. The new treatment is using a haploidentical donor for transplantation, which has good response rates and significantly lower rates of acute and chronic GVHD.
Immunosuppressant
Bone Marrow Transplant for Dyskeratosis Congenita
This trial tests a new bone marrow transplant method for patients with Dyskeratosis congenita. It uses fludarabine and antibodies to avoid harmful side effects, aiming to improve survival and reduce complications. Fludarabine-based regimens have been shown to be effective and feasible in reducing transplant-related morbidity in patients with Dyskeratosis congenita.
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Frequently Asked Questions
Introduction to aplastic anemia
What are the top hospitals conducting aplastic anemia research?
In the realm of aplastic anemia, several top hospitals are leading the charge in cutting-edge clinical trials. At the National Institutes of Health Clinical Center in Bethesda, a vital hub for medical research, six active trials are currently underway to combat aplastic anemia. With a total of nine completed trials dedicated to this rare condition, their commitment dates back to 2001 when they recorded their very first trial. In Los Angeles, Children's Hospital Los Angeles stands as a beacon of hope with four ongoing aplastic anemia trials and a track record of six past studies since embarking on their inaugural trial in 2006.
Similarly contributing to this crucial field is the renowned Children's Hospital of Philadelphia located in Philadelphia itself; they presently conduct four active clinical tests while having conducted ten previously held trials since starting off with aplastic anemia experimentation back in 2003.
The National Institutes of Health Clinical Center further establishes its prominence by operating at two locations—Bethesda and Rockville Pike—with both centers collectively engaging in four active aplastic anemia trials alongside a commendable history encompassing twenty-six previous investigations that initiated as early as1997.
Rounding out this list is Hackensack University Medical Center situated in Hackensack—a force to reckon with despite being smaller-scale compared to others mentioned herein—where three current aplastic anemia clinical trials align seamlessly with their contribution through six prior investigations; theirs can be traced back only fifteen years ago until 2006.
These esteemed institutions exemplify unwavering dedication towards understanding and combating aplastic anemia—a condition characterized by bone marrow failure—and offer renewed hope for patients worldwide. Their collective efforts push boundaries and bring us closer each day toward innovative treatments that could transform lives affected by this challenging disease.
Which are the best cities for aplastic anemia clinical trials?
When it comes to aplastic anemia clinical trials, several cities are leading the way in research and development. Bethesda, Maryland stands out with 10 active trials focusing on treatments like Eltrombopag and Donor derived G-CSF mobilized PBC. Boston, Massachusetts follows closely behind with 8 ongoing studies examining interventions such as Fludarabine and Matched Unrelated Stem Cell Transplant. Los Angeles, California, Houston, Texas, and Philadelphia, Pennsylvania also contribute significantly to the field with their respective active trials exploring various treatment options for this condition. These cities provide individuals with aplastic anemia access to cutting-edge clinical trials that offer hope for improved outcomes and advancements in care.
Which are the top treatments for aplastic anemia being explored in clinical trials?
Aplastic anemia, a condition characterized by the body's inability to produce enough blood cells, is currently under investigation for several potential treatments in clinical trials. Eltrombopag has emerged as one of the top contenders, with three ongoing trials dedicated to exploring its effectiveness against aplastic anemia. Since its introduction in 2009, it has been featured in a total of 11 trials focused on this particular disease. Another promising treatment being studied is fludarabine, which also boasts three active clinical trials and a significant presence in 24 previous studies since it was first listed as a potential therapy for aplastic anemia back in 2000. These exciting developments offer hope for individuals affected by aplastic anemia and may pave the way for improved treatment options in the future.
What are the most recent clinical trials for aplastic anemia?
Exciting advancements are being made in the field of aplastic anemia treatment through recent clinical trials. One such trial focuses on the effectiveness of exercise interventions, aiming to improve outcomes for patients with this condition. Another promising study explores matched unrelated stem cell transplants as a potential solution for aplastic anemia. Additionally, hematopoietic cell transplantation is being investigated as a viable treatment option in Phase 2 trials. Furthermore, research is underway to evaluate the efficacy of total lymphoid irradiation (TLI) in managing aplastic anemia symptoms. Lastly, REGN7257 shows promise in early and ongoing phases of clinical testing. These groundbreaking trials offer hope and new possibilities for individuals battling aplastic anemia.
What aplastic anemia clinical trials were recently completed?
Recently completed clinical trials in the field of aplastic anemia have focused on exploring innovative treatments. In July 2017, the Medical College of Wisconsin concluded a trial investigating Haplo HSCT as a potential therapy. Similarly, in June 2013, the National Heart, Lung, and Blood Institute wrapped up a trial evaluating Eltrombopag for its efficacy in treating this condition. These studies represent significant efforts to advance our understanding and treatment options for individuals affected by aplastic anemia.