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Aplastic Anemia Clinical Trials

A listing of Aplastic Anemia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (66) clinical trials

Pilot Study of Metformin for Patients With Fanconi Anemia

This is a single institution, open-label, single arm pilot study of Metformin in patients with Fanconi Anemia (FA) and cytopenias with the primary endpoint of hematologic response. This study will also assess safety, tolerability, and the biologic effects of Metformin in patients with FA.

Phase

Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients

With compromised bone marrow function, patients with aplastic anemia (AA) and/and hypoplastic myelodysplastic syndrome (hMDS) are at an increased risk of invasive fungal infection. Moreover, the use of antithyocyte globulin (ATG), a part of standard first line treatment for AA/hMDS, increases the risk of fungal infection due to its antilymophocytic ...

Phase

Gene Therapy for Fanconi Anemia

PRIMARY OBJECTIVES: I. To determine the safety of lentiviral gene transfer for patients with Fanconi anemia complementation group A. SECONDARY OBJECTIVES: I. To determine the feasibility and efficacy of filgrastim (G-CSF) and plerixafor mobilization in FA patients. II. To determine the feasibility and efficacy of lineage depletion of bone marrow ...

Phase

Chemotherapy Total Body Irradiation and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

PRIMARY OBJECTIVES: I. To determine the cumulative incidence of extensive chronic graft versus host disease (GVHD) at 1 year after transplantation utilizing the novel conditioning/GVHD prophylactic regimen for patients undergoing allogeneic hematopoietic cell transplantation, in patients who do not progress before day 100. SECONDARY OBJECTIVES: I. To evaluate clinical response, ...

Phase

Umbilical Cord Blood Transplantation From Unrelated Donors

This study is a single-center treatment protocol with four possible preparative regimens, designed to validate the process of umbilical cord blood stem cell transplantation at our institution. Enrolled patients will receive chemotherapy +/-total body radiation as a pre-transplant conditioning regimen. Patients will then receive cord blood stem cells followed by ...

Phase

Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA)

After enrollment (see detailed inclusion and exclusion criteria below) the patients are randomized either to the Placebo or Eltrombopag arm. The randomization is double blinded. Randomization will take in account patient's age and disease severity by stratifying into 4 block combinations to ensure homogeneity between treatment arms. All patients receive ...

Phase

Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA

This trial will evaluate safety and efficacy of combination eltrombopag with standard hATG/CSA as first line therapy in patients with SAA. The primary endpoint is going to be estimating the rate of complete hematologic response at the point in four months after the end of the treatment. Secondary endpoints are ...

Phase

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

The main objective of this open-label Phase I / II clinical trial is to evaluate the safety and therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi's Anemia Subtype A. The drug to be ...

Phase

Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia

Aplastic anemia (AA) is a disorder thought to be caused by an immune-mediated bone marrow failure. Not all people with AA are eligible for today's standard treatments. One new treatment approach uses umbilical cord derived mesenchymal stem cellsspecialized cells capable of developing into other types of cellsto provide the basis ...

Phase

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG) Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

This will be an open-label, single arm, phase IIa study in subjects with AA or Hypoplastic MDS. Eligible subjects will receive subcutaneous (SC) injections of BL-8040 monotherapy over 10 days. From Day 11 through Day 14, subjects will receive hATG, Methylprednisolone and Cyclosporine. From Day 15 until Day 30 (of ...

Phase