72 Recruiting Studies Using Sirolimus

Active clinical trials that use this intervention, sourced directly from the NIH registry.

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Trials Using Sirolimus

🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic

Phase III Trial of Sirolimus in IBM

📍 University of Kansas Medical Center — Kansas City, Kansas

Inclusion Body Myositis

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Sickle Cell Anemia

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Sickle Cell Disease

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🔵 Active, Not Recruiting Early Phase1 💊 Investigational Drug / Biologic

Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Chronic Granulomatous Disease

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🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic

Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant

📍 City of Hope Medical Center — Duarte, California

Allogeneic Hematopoietic Stem Cell Transplant Recipient

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🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic

Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial

📍 Stanford University — Palo Alto, California

LAM

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

📍 UCSF Benioff Children's Hospital, 747 52nd St — Oakland, California

Gaucher Disease, Type 2

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🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic

This Study Consists of Two Study Parts Conducted Under a Single IRB. Part I: Short Term ApoE-dependent Cerebral Blood Flow Response to Sirolimus in Cognitively Normal Adults Part II: Short Term ApoE-dependent Cerebral Blood Flow and Lung Perfusion Response to Sirolimus in Cognitively Normal Adults

📍 University of Missouri-Columbia — Columbia, Missouri

Genetic Predisposition to Disease

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia

📍 City of Hope Medical Center — Duarte, California

Adult Acute Lymphoblastic Leukemia in Remission

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Chronic Granulomatous Disease Transplant

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🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic

TMLI and Alemtuzumab for Treatment of Sickle Cell Disease

📍 City of Hope Medical Center — Duarte, California

Sickle Cell Disease

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Haploidentical PBMC Transplant for Severe Congenital Anemias

📍 National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland

Sickle Cell Anemia

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

📍 Fred Hutch/University of Washington Cancer Consortium — Seattle, Washington

Aplastic Anemia

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

📍 Mayo Clinic, 13400 E. Shea Boulevard — Scottsdale, Arizona

Parkinson Disease

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

📍 Roswell Park Cancer Institute — Buffalo, New York

Acute Myeloid Leukemia in Remission

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Sickle Cell Disease

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🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic

Sirolimus or Everolimus or Temsirolimus and Vorinostat in Advanced Cancer

📍 University of Texas MD Anderson Cancer Center — Houston, Texas

Advanced Cancer

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Sickle Cell Disease

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation

📍 St. Jude Children's Research Hospital — Memphis, Tennessee

Acute Lymphoblastic Leukemia (ALL)

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Severe Aplastic Anemia

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)

📍 University of Illinois at Chicago — Chicago, Illinois

Sickle Cell Disease

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🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Optimizing PTCy Dose and Timing

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Graft Versus Host Disease

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease

📍 University of Arkansas for Medical Sciences — Little Rock, Arkansas

Castleman Disease

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia

📍 Banner University Medical Center - Tucson — Tucson, Arizona

Acute Lymphoblastic Leukemia

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder

📍 MD Anderson Cancer Center — Houston, Texas

Familial Platelet Disorder

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🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic

Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft

📍 Stanford University — Stanford, California

Allogeneic Hematopoietic Cell Transplantation (HCT)

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🔵 Active, Not Recruiting Phase2 / Phase3 💊 Investigational Drug / Biologic

Assessing the Efficacy of Sirolimus in Patients With COVID-19 Pneumonia for Prevention of Post-COVID Fibrosis

📍 University of Chicago — Chicago, Illinois

Pulmonary Fibrosis

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors

📍 Phoenix Children's Hospital — Phoenix, Arizona

Cancer

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🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic

Virotherapy and Natural History Study of KHSV-Associated Multricentric Castleman s Disease With Correlates of Disease Activity

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Lymphoproliferative Disorder

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🟡 Not Yet Recruiting Phase2 💊 Investigational Drug / Biologic

Cemiplimab for Kidney Transplant Recipients With Advanced Cutaneous Squamous Cell Carcinoma

📍 Washington University School of Medicine — St Louis, Missouri

Kidney Transplant Recipient

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🟡 Not Yet Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Ventricular mTOR Inhibition to Prevent Hydrocephalus After Brain Hemorrhage

📍 Massachusetts General Hospital Lunder 4 OR for adult surgeries — Boston, Massachusetts

Post-hemorrhagic Hydrocephalus (PHH)

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🟡 Not Yet Recruiting Phase4 💊 Investigational Drug / Biologic

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

📍 Baylor College of Medicine — Houston, Texas

Duchenne Muscular Dystrophy

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🟡 Not Yet Recruiting Phase2 💊 Investigational Drug / Biologic

Intestinal & Multivisceral Transplantation for Unresectable Mucinous Carcinoma Peritonei (TRANSCAPE)

📍 Cleveland Clinic Digestive Disease & Surgery Institute (DDSI), Case Comprehensive Cancer Center — Cleveland, Ohio

Secondary Malignant Neoplasm of Retroperitoneum

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🟡 Not Yet Recruiting Phase1 💊 Investigational Drug / Biologic

Effects of Sirolimus on Asymptomatic ApoE4 Carriers

📍 University of Missouri — Columbia, Missouri

Genetic Predisposition to Disease

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

📍 Alberta Children's Hospital — Calgary, Alberta

Sickle Cell Disease

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Allo HSCT Using RIC and PTCy for Hematological Diseases

📍 Masonic Cancer Center at University of Minnesota — Minneapolis, Minnesota

Acute Myelogenous Leukemia

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation

📍 City of Hope — Duarte, California

Peripheral Blood Stem Cell Transplantation

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Lysosomal Diseases

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors

📍 Aflac Cancer & Blood Disorders Centers — Atlanta, Georgia

Solid Tumor

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome

📍 City of Hope Medical Center — Duarte, California

Acute Myeloid Leukemia With Complex Karyotype

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody

📍 Children's National Hospital — Washington D.C., District of Columbia

Sickle Cell Disease

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🟢 Recruiting Phase4 💊 Investigational Drug / Biologic

Cosibelimab for CSCC in Patients With Kidney Transplant or Hematologic Malignancy

📍 Brigham and Women's Hospital — Boston, Massachusetts

Cutaneous Squamous Cell Carcinoma

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

📍 University of Illinois at Chicago — Chicago, Illinois

Sickle Cell Disease

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Effect of Sirolimus on Molecular Alterations in Cerebral Aneurysms

📍 University of Miami — Miami, Florida

Cerebral Aneurysm

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🟢 Recruiting Observational / Diagnostic ⚙️ Medical Device Protocol

MagicTouch™Sirolimus-Coated Balloon for Treatment of Coronary Artery Lesions in Small Vessels

📍 Dignity Health - Mercy Gilbert Medical Center — Gilbert, Arizona

Coronary Artery Disease

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

📍 Yogi Chopra — Toronto, Ontario

Thalassemia in Children

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Allo HSCT for High Risk Hemoglobinopathies

📍 Masonic Cancer Center — Minneapolis, Minnesota

Graft Failure

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🟢 Recruiting Observational / Diagnostic ⚙️ Medical Device Protocol

MagicTouch for Treatment of In-Stent Restenosis in Coronary Artery Lesions

📍 Cardiology, PC - Princeton Baptist Medical Center — Birmingham, Alabama

In-Stent Restenosis

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Weekly Sirolimus Therapy

📍 Medical University of South Carolina — Charleston, South Carolina

Venous Malformation

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

📍 University of Alabama at Birmingham — Birmingham, Alabama

Tuberous Sclerosis Complex

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🟢 Recruiting Phase1 💊 Investigational Drug / Biologic

Phase I Trial Integrating HLA-Haploidentical Anti-CD19 CAR-T Cells With Post-Transplantation Cyclophosphamide-Based HLA-Haploidentical Hematopoietic Cell Transplantation

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Hematologic Malignancies

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

MTOR Inhibitors in Older Adults

📍 UT Southwestern Medical Center — Dallas, Texas

Aging

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Total Body Irradiation and Astatine-211-Labeled BC8-B10 Monoclonal Antibody for the Treatment of Nonmalignant Diseases

📍 Fred Hutch/University of Washington Cancer Consortium — Seattle, Washington

Non-Malignant Neoplasm

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators

📍 Lurie Children's Hospital — Chicago, Illinois

X-Linked Myotubular Myopathy

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Advancing Transplantation Outcomes in Children

📍 University of Alabama at Birmingham (Site # 71038) — Birmingham, Alabama

Kidney Transplant

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Cyclophosphamide and Sirolimus for the Treatment of Metastatic, RAI-refractory, Differentiated Thyroid Cancer

📍 University of Michigan Cancer Center — Ann Arbor, Michigan

Metastatic Thyroid Cancer

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease

📍 St Jude Children's Research Hospital — Memphis, Tennessee

Sickle Cell Disease

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🟢 Recruiting Interventional Protocol 📊 Observational Clinical Research

Safety and Durability of Sirolimus for Treatment of LAM

📍 Stanford University Medical Center — Stanford, California

Lymphangioleiomyomatosis

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Chronic Granulomatous Disease (CGD)

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🟢 Recruiting Phase1 💊 Investigational Drug / Biologic

Rapalog Pharmacology (RAP PAC) Study

📍 University of Wisconsin — Madison, Wisconsin

Aging

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🟢 Recruiting Phase1 💊 Investigational Drug / Biologic

225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome

📍 City of Hope Medical Center — Duarte, California

Acute Lymphoblastic Leukemia

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Chronic Granulomatous Disease

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RECRUITING Phase1 / Phase2 💊 Investigational Drug / Biologic

Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer

📍 UC San Diego Moores Cancer Center — La Jolla, California

Clinical Stage III Cutaneous Melanoma AJCC v8

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Domatinostat With Sirolimus for Relapsed, Refractory Sarcoma and Osteosarcoma

📍 Moffitt Cancer Center — Tampa, Florida

Sarcoma

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Hematologic Neoplasms

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🟢 Recruiting Phase1 💊 Investigational Drug / Biologic

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

📍 Columbia University Irving Medical Center — New York, New York

Sickling Disorder Due to Hemoglobin S

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🟢 Recruiting Phase1 💊 Investigational Drug / Biologic

Sirolimus Pre-conditioning on T Cell Activity and T-cell Engaging Bispecific Antibody Efficacy in Multiple Myeloma

📍 University of Iowa Health Care — Iowa City, Iowa

Multiple Myeloma

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

CD40L-HyperIgM Syndrome

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

The Role of Sirolimus in Preventing Functional Decline in Older Adults

📍 UT Southwestern Medical Center — Dallas, Texas

Aging

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)

📍 Moffitt Cancer Center — Tampa, Florida

Acute Leukemia

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🟢 Recruiting Phase2 💊 Investigational Drug / Biologic

Optimizing GVHD Prophylaxis After Allogeneic Hematopoietic Cell Transplantation

📍 University of Nebraska Medical Center — Omaha, Nebraska

Hematological Malignancies

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🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic

Phase I/II Study to Reduce Post-transplantation Cyclophosphamide Dosing for Older or Unfit Patients Undergoing Bone Marrow Transplantation for Hematologic Malignancies

📍 National Institutes of Health Clinical Center — Bethesda, Maryland

Hematologic Neoplasms

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