263 Clinical Trials for Stem Cell Transplant
The purpose of this study is to find the smallest amount of the 131 I-apamistamab needed for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant. This is the first time 131 I-apamistamab is being used for advanced Sickle Cell Disease (SCD) in the setting of allogeneic stem cell transplant. 131 I-apamistamab is an investigational product. This means that 131 I-apamistamab has not been approved by the Food and Drug Administration (FDA) for medical use in patients. The study treatment that is given before the transplant is called the conditioning regimen. In this study, the investigators are adding a drug called 131 I-apamistamab instead of the conditioning regimen typically given before a stem cell transplant.
The purpose of this study is to find out whether adding belumosudil to a usual approach for reducing the risk of graft-versus-host disease (GVHD) may be an effective GVHD prevention approach for people with blood cancer who have a stem cell transplant. The investigators will also look at the safety of the study approach.
This is an open-label, multi-site, Phase II randomized trial with response-adaptive design for newly diagnosed multiple myeloma (NDMM) participants who have had prior induction therapy. The primary objective of this study is to compare the rates of achieving undetectable measurable residual disease (MRD) in the bone marrow with elranatamab and daratumumab employed as post-induction consolidation and maintenance treatment (Arm A) versus autologous stem cell transplant (ASCT) followed by lenalidomide and daratumumab treatment (Arm B).
The goal of this clinical trial is to learn if using an intervention website (Mosaic) improves selected patient-reported outcomes in adult blood cancer patients undergoing allogeneic or autologous stem cell transplant, compared to using an educational website (control group). Patients will be recruited prior to their scheduled transplant, then randomized to use one of these two study websites throughout the study. They will complete five assessments during the study: one before transplant (baseline) and four after transplant (2, 4, 6, and 8 month follow-ups). The main questions this trial aims to answer are: 1. Compared to patients using the control group website, do patients using the intervention website report greater improvements in general psychological distress, cancer treatment-related distress, physical symptoms, and health-related quality of life? 2. Are these benefits at least partially explained by improvements in perceived preparedness, self-efficacy, and approach coping and/or reductions in avoidant coping and perceived stress? 3. Do some patients benefit more from using the intervention website than others? Specifically, we will examine whether patients' primary language (English/Spanish) and their initial psychological distress are related to the benefit they get from using the intervention website. We will also explore effects of sex, race, ethnicity, and transplant type.
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options.
Infections are a major cause of morbidity and mortality in patients undergoing hematopoietic stem cell transplant (HSCT). The purpose of this study is to evaluate if metagenomic next-generation sequencing (mNGS) can detect microbial signatures in people undergoing HSCT, and if microbial identification can be correlated with clinical features of infection (e.g., fever). Participants undergoing HSCT as part of other studies at the NIH Clinical Center (CC) will provide blood before the transplant and through 6 months after. Total nucleic acid will be extracted from plasma and subjected to mNGS. The primary objective of this study is to investigate if by using plasma and an mNGS approach, we can detect bacterial, fungal, protozoan, or viral DNA/RNA over time, in immunocompromised patients undergoing transplantation. Secondary objectives are to: (1) To correlate microbial identification with episodes of fever or clinical suspicion of infection; and to (2) correlate change in microbial signatures in patients with suspected immune reconstitution inflammatory syndrome. The study is conducted at the NIH Clinical Center. Participants, aged 3 years and older, on other research studies at the NIH CC who are undergoing HSCT are invited to take part of this study. Expected participation is up to six months.
The researchers are doing this study to find out if an allogeneic hematopoietic stem cell transplant (HSCT) or maintenance therapy with azacitidine and venetoclax is more effective at keeping AML from coming back (relapsing).
The goal of this clinical research study is to learn if intermediate-intensity conditioning therapy followed by a cord blood transplant can help to control high-risk hematological malignancies in patients who need a second allogeneic stem cell transplantation.
This clinical trial compares the use of virtual reality to standard care for improving symptom management in patients undergoing hematopoietic stem cell transplantation (HSCT). Significant symptoms experienced by hospitalized HSCT patients include, but are not limited to, depression, tiredness, anxiety, drowsiness, lack of appetite, pain, and overall decreased quality of life and well-being. Virtual reality (VR) as an intervention can provide these patients with a much-needed escape from their reality and has proven results in clinical settings as a distraction therapy. VR technology targets the patient's auditory, visual, and physical contact/touch senses, and has been evidenced to improve depression, fatigue, anxiety, appetite, and pain. Virtual reality may improve symptom management in patients undergoing HSCT.
The goal of this clinical trial is to test BSB-1001 which is a new type of cellular therapy to treat blood cancers (AML, ALL and MDS). It will evaluate the safety of BSB-1001 and also determine whether it works to prevent relapse of your cancer.
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).
A serious consequence of systemic diffuse large B-cell lymphoma (DLBCL) is secondary central nervous system (CNS) relapse, which occurs in approximately 5% of all patients. Many CNS relapses occur within the first year after completion of frontline treatment and are associated with significantly increased mortality; thus, it is important to tailor frontline treatment to provide prophylaxis against CNS relapse in those patients who are determined to be high-risk. Autologous stem cell transplantation (ASCT) is standard of care for patients with DLBCL who relapse one year or more after first remission, and it has been shown to improve progression-free survival for patients with primary CNS lymphoma. The four-drug BEAM regimen (carmustine, etoposide, cytarabine, and melphalan) is the preferred conditioning regimen for DLBCL patients undergoing ASCT; however, patients with primary CNS lymphoma receive thiotepa plus carmustine as their conditioning regimen due to its better CNS penetration. This study tests the hypothesis that consolidation thiotepa/carmustine ASCT in first complete remission will reduce the risk of CNS relapse in transplant-eligible patients with DLBCL with no prior CNS disease at high risk of secondary CNS recurrence.
The purpose of this study is to see if siltuximab plus population pharmacokinetic (PK)-dosed melphalan works as well as the usual approach (body surface area \[BSA\]-dosed melphalan) in people with multiple myeloma (MM) who are receiving an autologous stem cell transplant (ASCT) as part of their standard treatment. The researchers will also see if siltuximab in combination with population PK-dosed melphalan works to decrease symptoms after an ASCT, and will study the safety of siltuximab. For the run-in, 15 patients will receive siltuximab, 11 mg/kg, seven days before and 14 days after autologous hematopoietic stem cell infusion (+/-2 day).
The goal of this trial is to develop an Independent Music Listening tool for stem cell transplant patients that have prolonged hospitalizations. The main questions it aims to answer are: \[primary hypothesis or outcome measure 1\]? \[primary hypothesis or outcome measure 2\]? Participants will use a self-guided music listening intervention of 1-hour daily music listening during their inpatient hsopitalization and delivered via web-based platform.
This study evaluates mucositis and other oral symptoms in patients undergoing photobiomodulation in patients undergoing hematopoietic stem cell transplantation.
The purpose of this study is to assess Tacrolimus/Methotrexate/Ruxolitinib versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation
This phase I trial tests the safety, side effects, best dose and effectiveness of revumenib in treating patients with acute leukemia after allogeneic stem cell transplant. Revumenib is in a class of medications called menin inhibitors. Revumenib targets and binds to the protein menin, thereby preventing the interaction between menin and the mixed lineage leukemia protein. Disrupting this interaction prevents the activation of specific genes that fuel the development of leukemia cells and inhibits the survival, growth, and production of certain kinds of leukemia cells. Giving revumenib may be safe, tolerable, and/or effective in treating patients with acute leukemia after allogeneic stem cell transplant.
This research study is evaluating the efficacy of a novel self-administered digital application for improving sexual health outcomes, quality of life, and psychological distress in hematopoietic stem cell transplant survivors.
This phase 2 study aims to confirm the efficacy seen in the prior phase 1 trial, and further contribute to this effort through the collection of leukemia cells pre- and post- in vivo IFN-γ therapy. As in the previously conducted phase 1 trial, this trial will test whether leukemia blasts were responsive to IFN-γ in vitro and in vivo, with single-cell RNA sequencing (scRNAseq) conducted to understand the transcriptomic changes induced by IFN-γ in leukemia cell subsets, including those with stem cell characteristics.
This is a feasibility study of a Work Support (WorkS) intervention designed to ameliorate employment challenges for people preparing to return to work after allogeneic stem cell transplantation. The aim of this study is to evaluate "proof of concept" by: 1. examining the feasibility and acceptability of the WorkS intervention and the study procedures, and 2. exploring the preliminary effects of WorkS for improving patient-reported return-to-work self-efficacy, work status, quality of life, and financial toxicity.
Pediatric patients undergoing stem cell transplant (SCT) are hospitalized for extended periods and are at high risk for sleep disturbances. In order to begin to address the environmental issues that SCT recipients face during inpatient hospitalizations, investigators will conduct a single arm pilot study of a program entitled 'Developing Restful Environments and Management Strategies' (DREAMS). The program will provide children receiving SCT and families with information and a kit that includes tools which may support sleep and circadian health during an inpatient hospitalization.
The purpose of this Phase 1, first in human open-label study is to assess the safety and tolerability of TRX-103 in patients with hematological malignancies undergoing HLA-mismatched related or unrelated hematopoietic stem cell transplantation (HSCT). It is anticipated that up to 36 Subjects will be enrolled during a 18-24 month enrollment period. TRX-103 will be infused one time post HSCT.
This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor specific antibodies (DSA) levels to low enough levels to permit patients to proceed with allogeneic peripheral blood transplant (alloBMT). Those being asked to participate have high DSA levels that puts those being asked to participate at high risk of rejecting the available donor's blood stem cells and making those being asked to participate ineligible to receive a stem cell transplant.
To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.
This clinical trial tests the feasibility, implementation and acceptability of chaplain delivered compassion meditation in order to improve spiritual care for patients receiving stem cell transplantation. Hospital chaplains play a vital role in delivering emotional and spiritual care to a broad range of both religious and non-religious patients for a wide variety of stressors, and extensive research indicates that spiritual consults impact patient outcomes and satisfaction. Compassion meditation is a secularized, research-based mindfulness and compassion meditation program designed to expand and strengthen compassion for self and others. Practices include training in attentional stability and increased emotional awareness, as well as targeted reflections to appreciate one's relationship with self and others. By centering the mind, controlling debilitating ruminative thoughts, and cultivating personal resiliency and an inclusive and more accurate understanding of others. Engaging in chaplain delivered compassion meditation may improve the spiritual care for patients receiving stem cell transplantation.
This randomized clinical trial is evaluating the impact of a positive psychology intervention (PATH) on anxiety symptoms, depression symptoms, and quality of life in survivors of hematopoietic stem cell transplant (HSCT) compared to usual care.
This phase I trial tests the safety, side effects, best dose, and effectiveness of 225Ac-DOTA-Anti-CD38 daratumumab monoclonal antibody in combination with fludarabine, melphalan and total marrow and lymphoid irradiation (TMLI) as conditioning treatment for donor stem cell transplant in patients with high-risk acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS). Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Radioimmunotherapy is treatment with a radioactive substance that is linked to a monoclonal antibody, such as daratumumab, that will find and attach to cancer cells. Radiation given off by the radioisotope my help kill the cancer cells. Chemotherapy drugs, such as fludarabine and melphalan, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. TMLI is a targeted form of body radiation that targets marrow, lymph node chains, and the spleen. It is designed to reduce radiation-associated side effects and maximize therapy effect. Actinium Ac 225-DOTA-daratumumab combined with fludarabine, melphalan and TMLI may be safe, tolerable, and/or effective as conditioning treatment for donor stem cell transplant in patients with high-risk AML, ALL, and MDS.
This phase II trial tests how well ibrutinib works in preventing chronic graft-versus-host disease (GVHD) in patients undergoing donor (allogeneic) hematopoietic cell transplantation (HCT). An allogeneic hematopoietic cell transplantation (allo-HCT) is a treatment in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical donor. When healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets. However, sometimes the transplanted cells from a donor can attack the body's normal cells (called GVHD). Giving ibrutinib after the transplant may stop that from happening. Ibrutinib is in a class of medications called kinase inhibitors. It works by blocking a protein in the blood called Bruton's tyrosine kinase (BTK). By blocking BTK, ibrutinib inhibits certain immune cells that play a role in cGVHD. Giving ibrutinib after an allo-HCT may prevent the development of chronic GVHD.
In an effort to reduce graft versus host disease (GVHD) and enhance graft versus leukemia (GVL) effect post allogenic hematopoietic stem cell transplantation (AHSCT), recent research has focused on host immune cell depletion. Frame shifting anti-thymocyte globulin (ATG) backwards to earlier days before days 0 can result in deeper host and less graft T-cell depletion, leading to better immune reconstitution. Preliminary data where 80% of the ATG dose is given on days -6,-5,-4 and 20% given on day -1, showed effective prevention of severe acute GVHD, chronic GVHD and favorable early immune reconstitution. We hypothesize that our 2 step ATG dosing platform when combined with standard tacrolimus and mini methotrexate can prevent grade III-IV acute GVHD and chronic GVHD, resulting in improvement of GVHD/relapse free survival at one year post transplant.
This study is designed as a single arm open label traditional Phase I, 3+3, study of CD4-redirected chimeric antigen receptor engineered T-cells (CD4CAR) in patients with relapsed or refractory AML. The study will evaluate safety in this patient population and also the presence of efficacy signal described by elimination of residual disease to qualify patients for stem cell transplant.