Blood and Marrow Transplantation and Cellular Therapies Clinical Studies

The Division of Blood and Marrow Transplantation and Cellular Therapies at UPMC Children’s Hospital of Pittsburgh maintains an active research program that includes clinical studies in pursuit of new and novel therapies to treat patients with high-risk leukemia or lymphoma, and for those afflicted with other life-threatening inherited conditions that can lead to autoimmune diseases. Select a study below to review a brief description and requirements. Parents or guardians of children who might be eligible are encouraged to inquire about enrollment. Contact information is listed at the end of each study.

Thank you for exploring these opportunities and helping us to find cures and prevent childhood disease.

Clinical Studies

Researchers

Acute Myeloid Leukemia or Myelodysplastic Syndrome Transplant Using Reduced Intensity Conditioning – Phase II

Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

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Blood and Marrow Transplant Research Repository & Database

Protocols for a Research Database and Sample Repository for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

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Chronic Granulomatous Disease Progression and Outcomes Review

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

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Chronic GVHD Prevention Through Naïve T Cell Depletion in BMT Transplants for Leukemia – Phase II

Trial of Naïve T Cell Depletion for Prevention of Chronic Graft-versus-host Disease in Children and Young Adults with Blood Cancers Undergoing Donor Stem Cell Transplant

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CMV Infection Prevention with Letermovir in HSCT Recipients – Phase IIb

Study of Letermovir in Pediatric Participants from Birth to Less than 18 Years of Age at Risk of Developing CMV Infection and/or Disease Following Allogeneic Hematopoietic Stem Cell Transplantation

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Crohn's Disease Treatment using Blood Stem Cells – Phase I/II

Autologous Stem Cell Transplantation with CD34 Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Young Adult Patients with Severe Crohn's Disease

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Immunodeficiency and End-Stage Lung Disease Treatment with BOLT+BMT Procedure

Bilateral Orthotopic Lung Transplant (BOLT) in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant (BMT) From Partially HLA-Matched Cadaveric Donors

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Non-Malignant Marrow Disorder Treatment using Reduced Intensity Conditioning – Phase II

Study of Reduced-Intensity Conditioning In Patients With Non-Malignant Disorders Undergoing Umbilical Cord, Bone Marrow, or Peripheral Blood Stem Cell Transplantation

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Protocol for Transplantation of Cryopreserved CBUs from the National Cord Blood Program

Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

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Protocol for Transplantation of Cryopreserved CBUs Compliant with National Marrow Donor Program Standards

Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications

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Registry & Tissue Banking Study of Cancer, Blood Disorder & Immunodeficiency Patients

Long-term Follow-up Registry and Biorepository for Patients Diagnosed With Primary Immunodeficiencies, Malignancy, and/or Eligible to Undergo Hematopoietic Stem Cell Transplant

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Registry & Tissue Banking Study of Patients Undergoing HCT, SOT, or with Autoimmune Disease

Hematopoietic Graft Engineering: Pre-clinical Studies of Immunocompetence

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Respiratory Tract Infection Identification in HSCT Patients

Pathogen Identification in Pediatric Hematopoietic Stem Cell Transplant Patients with Suspected Lower Respiratory Tract Infection

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Severe Combined Immunodeficiency Disorder Progression Study

A Prospective Natural History Study of Diagnosis, Treatment, and Outcomes of Children with SCID Disorders

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Sickle Cell Disease Treatment with Alternative Donor Bone Marrow Transplant

T-Cell Depleted, Alternative Donor Transplant in Pediatric and Young Adult Patients with Severe Sickle Cell Disease

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Stem Cell Transplant with CliniMACS® Device

Use of Miltenyi Biotec’s CliniMACS CD34 Reagent System as a Humanitarian Use Device for Isolation of Hematopoietic Stem Cells or T-cell Depletion in Multiple Settings

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Systemic Sclerosis Treatment with Autologous Transplant of CD34-Selected Peripheral Blood Stem Cells

Autologous Stem Cell Transplantation with CD34-Selected Peripheral Blood Stem Cells in Patients with Treatment-Resistant Systemic Sclerosis

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Treating Bone Marrow & Lung Failure with Tandem Lung/ Marrow Transplant

Lung Transplant in Tandem with Bone Marrow Transplant for Combined Lung and Bone Marrow Failure

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