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Displaying 1 - 25 of 25

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 donate bone marrow or blood…
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Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias (Children and Young Adults)

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
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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), adenovirus (ADV), BK and JC polyomaviruses (PyVs) virus may…
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Early Age-Related Hearing Loss Investigation (EARHLI)

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Justin Golub, MD, MS
Status: Currently Recruiting
Early Age-Related Hearing Loss Investigation (EARHLI) is a study to obtain information on how a hearing aid-based intervention can prevent cognitive decline in those at risk for Alzheimer's Disease and Alzheimer's Disease Related Dementias (AD/ADRD). Age-related hearing loss (ARHL) is the third most common chronic condition in older adults. The…
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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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Speech Perception in Hearing Impaired Listeners

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Nima Mesgarani, PhD
Status: Currently Recruiting
The purpose of this study is to learn more about the mechanisms of hearing loss, specifically to learn how abnormalities in the brain may produce certain experiences that are common in patients with hearing loss. Individuals with hearing loss will be compared to individuals with normal hearing.
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PEPN2113: Study of Uproleselan in Children with AML, MDS, or MPAL

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 study of a drug called uproleselan. We are testing new experimental drugs such as uproleselan in the hopes of finding a treatment that may be effective against acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back or that has not responded to standard therapy. This study looks at how well…
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Study of Quizartinib in Combination with Chemotherapy for Children and Young Adults with Recurrent /Refractory Acute Myeloid Leukemia (AML)

Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
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Study of Nivolumab in Combination with 5-Azacytidine in Acute Myeloid Leukemia (AML) - Children and Adults Age 1 to 30

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
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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 transplant. Recovery after a stem cell transplant can already be challenging without the added worry of life-threatening viral…
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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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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 is often treated with steroids, some patients are considered steroid-refractory, which means…
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MLN4924 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (1 month to 17 years old)

Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in children with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
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A study to Assess Safety and Effectiveness of Study Drug OTO-104 in Patients at Risk of Hearing Loss From Cisplatin Chemotherapy

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Alice Lee, MD
Status: Closed
Cisplatin is a chemotherapy medicine that is used to treat a variety of cancers. At high total (cumulative) doses it can be associated with progressive and irreversible hearing loss. The purpose of this study is see if injections of OTO-104 can protect against hearing loss associated with cisplatin therapy in the treatment of cancer. OTO-104 contains an…
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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 transplant recipient's body. The current way to treat cGVHD is to use prednisone (a type of…
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Decitabine and Vorinostat Followed by Fludarabine, Cytarabine, and G-CSF (FLAG) in Children & Young Adults with Acute Myeloid Leukemia

Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Decitabine (DEC) and Vorinostat (VOR) followed by the standard chemotherapy drugs (Fludarabine, Cytarabine and G-CSF (FLAG)). The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute myeloid leukemia (AML)…
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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 additional information about the safety of treating sickle cell disease with a hematopoietic stem…
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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 stem cell transplant,which may be life threatening. The condition is a…
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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 orcells from a donor (called an allogeneic transplant). The new,…
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A study for Adults with Sudden Sensorineural Hearing Loss Using the drug Am-111 to Evaluate its Effectiveness in Restoring Hearing Loss

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Anil Lalwani, MD
Status: Closed
Acute inner ear hearing loss is the sudden onset of hearing loss, usually in one ear, without any known cause. Acute hearing loss can permanently destroy some of the hair cells (that govern the sense of hearing) and / or hearing nerve in the inner ear.The drug AM-111 has been developed to protect the hearing cells and nerve following sudden inner ear…
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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 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. All patients in this group will receive the study drug, CSJ148.…
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A Study of the Gene Therapy CGF166 in Patients with Severe-to-Profound Hearing Loss

Condition: Ear, Nose and Throat / Hearing Loss
Investigator: Lawrence Lustig, MD
Status: Closed
The purpose of this research is to find out whether it is safe to use a virus to deliver a new gene to the inner ear of people with hearing loss. This is a kind of gene therapy which involves gene transfer. Once the genetic material and vector are surgically administered to you, it is not possible to remove these agents. The study is designed to evaluate…
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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 stem cell…
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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, sickle cell related kidney…
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