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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 (71) clinical trials

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. Mutations in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with ...

Phase N/A

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

Thrombosomes in Bleeding Thrombocytopenic Patients

The objectives of this trial, conducted in thrombocytopenic patients with modified World Health Organization (WHO) Grade 1* or 2 bleeding, assessing the safety of infusing increasing doses of allogeneic Thrombosomes in WHO bleeding and measures of coagulation.

Phase

New York Blood Center National Cord Blood Program

Umbilical cord blood donated to the New York Blood Center's National Cord Blood Program is collect, tested, processed, cryoprotected and frozen in liquid nitrogen for possible future transplantation to anyone who needs it. The Program has operated under a FDA IND exemption since 1996 and is licensed as a tissue ...

Phase N/A

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

NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

This is a clinical trial of upfront bone marrow transplantation for patients with SAA who do not have a fully human leukocyte antigen (HLA) matched donor. The trial uses a conditioning regimen which has been successful in the refractory and relapsed setting to maximize engraftment and post transplant therapy to ...

Phase

Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study

PRIMARY OBJECTIVES: I. To assess feasibility and safety of red blood cell transfusions at the time subjects requested based on symptomatic anemia. SECONDARY OBJECTIVES: I. To define a patient-driven red blood cell transfusion (RBCT) threshold and to compare the median hemoglobin (hgb) at the time subjects request RBCT to the ...

Phase N/A

Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

A major challenge in treating pediatric Severe Aplastic Anemia (SAA) is the determination of best primary therapy for patients who lack a fully matched related donor for HSCT. Good survival outcomes have been seen with IST, but initial and late failures, CSA dependence, persistent cytopenias and secondary Myelodysplastic Syndrome (MDS) ...

Phase N/A