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Displaying 1 - 22 of 22

Research Database for Cell Transplant and Other Cellular Therapies

Condition: Transplant / Stem Cell Transplant
Investigator: Markus Mapara, MD
Status: Currently Recruiting
The Center for International Blood and Marrow Transplant Research (CIBMTR), the research program of the National Marrow Donor Program (NMDP)/Be The Match, invites you to take part in a Research Database. The CIBMTR does research with medical data from patients who have had a transplant or other cellular therapy and donors who…
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Study of R-MVST Cells to Treat Patients with a Viral Infection

Condition: Transplant / Stem Cell Transplant
Investigator: Pawel Muranski, MD
Status: Currently Recruiting
The purpose of this study is to test whether giving an experimental cell product can treat viral infection in patients who have conditions that cause the poor function of their immune system. The patients with infections caused by viruses such as cytomegalovirus (CMV), Epstein-Barr virus (EBV),…
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Long-Term Follow-up Study for Participants of Kite-Sponsored Gene-Modified Therapy Studies

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Currently Recruiting
The purpose of this study is to monitor you for any delayed side effects that may possibly be related to the experimental gene-modified therapy product you received in the Kite-sponsored study for Axicabtagene ciloleucel (axi-cel). The main study does not involve treatment. In the re-treatment portion, you will be provided access to a second course of…
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A study for patients with sickle cell disease receiving stem cell transplants

Condition: Transplant / Stem Cell Transplant
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to see whether or not the medicine, Abatacept, when given after hematopoietic stem cell transplantation (HSCT) is effective in treating GVHD in children and adolescents with sickle cell disease (SCD). We are also planning to gather…
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Study of stem cell transplant effects in patients with sickle cell disease

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle…
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A study for patients undergoing Hematopoietic Stem Cell Transplant using study drug Defibrotide

Condition: Transplant / Stem Cell Transplant
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to test a new medicine to prevent liver venoocclusive disease (VOD) in patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or very high risk for liver VOD following HSCT. Liver VOD can be a complication of bone marrow and/or…
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Study of Itacitinib and Tocilizumab for the Treatment of Steroid Refractory Acute Graft versus Host Disease in Stem Cell Transplant

Condition: Transplant / Stem Cell Transplant
Investigator: Markus Mapara, MD
Status: Closed
Graft versus host disease (GVHD) is a common complication after allogeneic stem cell transplant. It happens when the immune system of the donor is attacking the organs, and usually causes a skin rash, diarrhea, nausea and vomiting, and liver abnormalities. While it…
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Study of GDC-8264 in Stem Cell Transplant Patients with Acute Graft-Versus-Host-Disease (acute GVHD)

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Closed
This study is testing a drug called GDC-8264, which is being developed to treat acute graft-versus-host-disease (acute GVHD). GDC-8264 is an experimental drug, which means health authorities have not approved GDC-8264 in combination with standard medications for the treatment of acute GVHD. The purpose of this study is to evaluate the effects, good or bad,…
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A study for stem cell transplant patients using study drug CSJ148

Condition: Transplant / Stem Cell Transplant
Investigator: Markus Mapara, MD
Status: Closed
The purpose of this study is to see if investigational drug CSJ148 is safe and if it can prevent HCMV infection during the first 98 days after stem cell transplant. Information about any side effect that the study drug can cause will also be collected.Study group will be made up of 6 patients.…
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A study for pediatric patients with acute graft vs host disease using study drug Remestemcel-L

Condition: Transplant / Stem Cell Transplant
Investigator: Prakash Satwani, MD
Status: Closed
The main purpose of the study is to find out about whether the study drug is safe in humans and aneffective treatment in pediatric patients with acute Graft versus Host Disease (GVHD). GVHD mayoccur after a bone marrow or stem cell transplant in which someone receives bone marrow tissue or…
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Study of ALVR105 (Viralym-M) in Patients Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)

Condition: Transplant / Stem Cell Transplant
Investigator: Prakash Satwani, MD
Status: Closed
Are you undergoing a stem cell transplant? You may be at high risk for infections after the transplant. This study is looking at the safety and effectiveness of an investigational medication for those who have undergone a stem cell…
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A Study to Evaluate the Safety of a Dose of Autologous CRISPR-Cas9 Human Stem Cells in Subjects With Severe Sickle Cell Disease

Condition: Blood Disorders
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stem cells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent…
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Study of Drug for the Treatment of Cytomegalovirus (CMV) Infection in Stem Cell Transplant Recipients

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to see which study drug is the better treatment for CMV infection. The study also wants to see how safe the study drugs are. The study drugs are Maribavir and Valganciclovir. Those who participate in the study will not know which drug they are taking. The study team will not know either.
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A study for patients with severe Sickle Cell Disease using study drug

Condition: Blood Disorders
Investigator: Markus Mapara, MD
Status: Closed
We are doing a research study to find out whether it is safe to use a virus to change the genes of bone marrow cells that become red blood cells. We hope that this type of therapy may be helpful in people with sickle cell disease, but it has not been tested before.…
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Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem…
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STAR HCST for Less Severe Sickle Cell Disease (SCD)

Condition: Transplant / Stem Cell Transplant
Investigator: Monica Bhatia, MD
Status: Closed
The primary objectives of this study are to measure the safety and effectiveness of bone marrow transplant (BMT) using specific chemotherapy in children with less severe SCD and to understand the long term effects of bone marrow transplant in children with SCD, by evaluating ovarian reserve, sickle cell related brain problems…
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Off the shelf T-cells therapy for viral infection post HCST

Condition: Transplant / Stem Cell Transplant
Investigator: Prakash Satwani, MD
Status: Closed
In this trial, we will evaluate whether partially-HLA matched allogeneic multivirus-specific VSTs, will be safe and produce anti-viral effects in immunodeficient recipients infected with one of more of the targeted viruses that are persistent despite conventional anti-viral therapy. This will be in patients who have received a hematopoietic…
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A study for patients with chronic Graft Versus Host Disease (cGVHD) using study drug Ibrutinib

Condition: Transplant / Stem Cell Transplant
Investigator: Ran Reshef, MD
Status: Closed
This is a research study of a new drug for the treatment of new onset chronic graft versus host disease (cGVHD). Chronic GVHD is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the…
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Study of CRISPR-CAS9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCS) in Subjects with Beta-Thalassemia

Condition: Blood Disorders
Investigator: Markus Mapara, MD
Status: Closed
This study is being done to learn about the safety and efficacy of CTX001 (the "Study Product") to treat -Thalassemia. CTX001 is "investigational", which means that the Study Product is considered experimental and is not approved by the Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory (government) authority and is…
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Study of Patients Receiving a Stem Cell Transplant for Non-Malignant Disease

Condition: Blood Disorders
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
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Study For Previously Treated Patients Of Light-Chain Amyloidosis Using Study Drug Carfilzomib

Condition: Blood Disorders / Amyloidosis
Investigator: Suzanne Lentzsch, MD
Status: Closed
This is a research study for subjects that have been previously treated for systemic light chain (AL)amyloidosis (a disease in which abnormal protein deposits can damage organs and tissues in your body) and nowrequire further treatment. The main purpose of this study is to evaluate the safety and determine the maximum…
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A study for patients with myelofibrosis using study drug PF-04449913

Condition: Blood Disorders
Investigator: Mark Heaney, MD, PhD
Status: Closed
This is a Phase 2 study for patients with myelofibrosis previously treated with one or more Janus Kinase(JAK) Inhibitor. A JAK inhibitor targets a certain pathway that may be causing cancer to grow, bystopping or inhibiting the cycle of development of the pathway. There is one JAK inhibitor that has beenapproved for the treatment of patients with…
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