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Pancytopenia clinical trials

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NCT ID: NCT04723706 Completed - Clinical trials for Hematologic Disorders and Bone Marrow Failure, Hematopoietic Stem Cell Transplantation (HSCT) or Cellular Therapy (CART)

Response to SARS-CoV-2 Vaccine in Stem Cell Transplant and Cellular Therapy Patients

Start date: March 19, 2021
Phase:
Study type: Observational

For many patients with hematologic disorders and bone marrow failure, hematopoietic stem cell transplantation (HSCT) or cellular therapy (CART) offers a curative treatment option. Patients after SCT or CART have a variable period of immune deficiency in the post-treatment period. The response to vaccination may affect the outcome of the transplant patients. the immunogenicity of vaccines in this immunosuppressed population is uncertain and variable. HSCT and CAR-T recipients are in a COVID-19 high-risk group and conferring immunity by vaccination at the earliest effective timepoint is desirable. At present, the immunogenicity and efficacy of SARS-CoV-2 vaccines in immune-impaired patients including autologous and allogeneic HSCT recipients is unknown. Furthermore, the impact of GvHD and IST on SARS-CoV-2 vaccine immunogenicity is unknown. the investigators aim to evaluate the vaccination response to COVID vaccines after SCT and CART

NCT ID: NCT04644016 Recruiting - AML Clinical Trials

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Start date: November 20, 2020
Phase: Phase 2
Study type: Interventional

This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.

NCT ID: NCT04558736 Active, not recruiting - Aplastic Anemia Clinical Trials

Haploidentical HCT for Severe Aplastic Anemia

Start date: January 21, 2021
Phase: Phase 2
Study type: Interventional

This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. 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 post-hematopoietic cell transplantation (HCT). Primary Objectives - To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide. - To estimate the overall survival and event free survival at 1-year post transplantation. Secondary Objectives - To calculate the incidence of acute and chronic GVHD after HCT. - To calculate the rate of secondary graft rejection at 1-year post transplantation - To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus). - To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year. Exploratory Objectives - To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA. - To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT. - To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation. - To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI. - Quantify donor derived Treg cells at different time points in patients received HCT. - Determine Treg activation status at different stages after HCT. - Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant? - How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient? - Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?

NCT ID: NCT04528355 Recruiting - Clinical trials for Primary Immunodeficiency (PID)

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

PRO-RIC
Start date: August 20, 2020
Phase:
Study type: Observational

This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.

NCT ID: NCT04478227 Active, not recruiting - Thrombocytopenia Clinical Trials

TPO-Mimetic Use in Children for Hematopoietic Failure

Start date: August 18, 2020
Phase: Early Phase 1
Study type: Interventional

This is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure disorders including acquired and inherited conditions as a first line of therapy along with standard of care.

NCT ID: NCT04356469 Recruiting - Clinical trials for Severe Aplastic Anemia

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Start date: July 22, 2020
Phase: Phase 2
Study type: Interventional

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.

NCT ID: NCT04232085 Recruiting - Clinical trials for Primary Immune Deficiency Disorder

Reduced Intensity BMT for Immune Dysregulatory and Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide

Start date: February 12, 2020
Phase: Phase 2
Study type: Interventional

This is a Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).

NCT ID: NCT04211714 Recruiting - Telomere Shortening Clinical Trials

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Start date: April 8, 2021
Phase: Phase 1
Study type: Interventional

This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.

NCT ID: NCT03773393 Active, not recruiting - Bone Marrow Disease Clinical Trials

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

Start date: May 30, 2019
Phase: Phase 1
Study type: Interventional

The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome. Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.

NCT ID: NCT03622788 Recruiting - Clinical trials for Acute Myeloid Leukemia

Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant

Start date: August 8, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This phase I/II trial studies how well cytokine-treated veto cells work in treating patients with hematologic malignancies following stem cell transplant. Giving chemotherapy and total-body irradiation before a stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Cytokine-treated veto cells may help the transplanted donor cells to develop and grow in recipients without causing graft-versus-host-disease (GVHD - when transplanted donor tissue attacks the tissues of the recipient's body).