Clinical trials: X-Linked Lymphoproliferative Disorders
X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)
A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2.
- Trial name
- NCT06309823
- Testing
- MAS825
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- The Hospital for Sick Children
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
Leukemia, Myeloid, Acute, Leukemia, B-cell
Precision Exercise in Children With Malignant Hemopathies
In the early years of life and during adolescence, physical activity is crucial for good development of motor skills. It is even more so for those children and young people who are forced to undergo anti-cancer therapies and…
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin
- Trial name
- SportTherapy (NCT04090268)
- Testing
- Sport Therapy
- Where
- Italy
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders
T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders
The hypothesis is to determine if a preparative regimen of busulfan, cyclophosphamide, and antithymocyte globulin (ATG) plus allogeneic stem cell transplantation will be effective in the treatment of immune deficiencies and…
- Conditions
- Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome
- Trial name
- NCT00176826
- Testing
- Stem Cell Transplant, Myeloablative conditioning regimen
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
SCID, Omenn's Syndrome
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
- Conditions
- SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome
- Trial name
- NCT01652092
- Testing
- Alemtuzumab 0.3 mg, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Non-ischemic Cardiomyopathy, Cardiomyopathies
Cardiovascular Multimodality Imaging Study
Determining the etiology of cardiomyopathy is of high clinical importance for optimal treatment strategy and prediction of prognosis.
- Conditions
- Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy
- Trial name
- NCT06610019
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Montefiore Medical Center
Common Variable Immunodeficiency, Agammaglobulinemia
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period.
- Trial name
- NCT00520494
- Testing
- Vivaglobin
- Where
- 6 sites in Canada, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies. II.
- Conditions
- Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease
- Trial name
- NCT00006054
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
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…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Granuloma, Granulomatous Disease, Chronic
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that…
- Conditions
- Granuloma, Granulomatous Disease, Chronic, Leukocyte Disease, Genetic Disease, X-Linked
- Trial name
- NCT00001405
- Where
- United States
- Volunteers
- 850 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency, Common Variable Hypogammaglobulinemia
Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
The main objective of this study is to see if GAMMAPLEX is efficacious with respect to Food and Drug Administration (FDA) minimal requirements (no more than 1 serious, acute, bacterial infection per subject per year) in subjects…
- Conditions
- Primary Immunodeficiency, Common Variable Hypogammaglobulinemia, X-linked Hypogammaglobulinemia, Hypogammaglobulinemia
- Trial name
- NCT00278954
- Testing
- Gammaplex (Intravenous immunoglobulin)
- Where
- 7 sites in United States
- Volunteers
- 50 people
- Sponsor
- Bio Products Laboratory
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders
Stem Cell Transplant for Immunologic or Histiocytic Disorders
This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of…
- Conditions
- Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome
- Trial name
- NCT00176865
- Testing
- Stem Cell Transplant, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Multiple Myeloma, Acute Myeloid Leukemia
National Longitudinal Cohort of Hematological Diseases
Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China.
- Conditions
- Multiple Myeloma, Acute Myeloid Leukemia, Hemophilia, Hemophilia A
- Trial name
- NICHE (NCT04645199)
- Where
- China
- Volunteers
- 2,300 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma
T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts
Subjects are being asked to participate in this study because treatment of their disease requires them to receive a stem cell transplant.
- Conditions
- Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma, Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Trial name
- MOHEL (NCT00368355)
- Testing
- Ara-C, Cyclophosphamide, Campath-1H
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Baylor College of Medicine