Clinical trials: Histiocytosis,Langerhans-Cell
Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic
New Candidate Criteria for Diagnosis of Macrophage Activation Syndrome
Macrophage activation syndrome(MAS) is a complication of bone marrow suppression, coagulopathy and CNS dysfunction which occurs in rheumatic diseases.
- Trial name
- MAS-D (NCT01095146)
- Where
- India
- Volunteers
- 50 people
- Sponsor
- Amrita Institute of Medical Sciences & Research Center
Histiocytosis, Langerhans-Cell
Biomarkers for Diagnostic, Prognostic and of Response to Treatment in Adult Langerhans Cell Histiocytosis
Adult Langerhans histiocytosis (LCH) is a rare disease of unknown etiology, characterized by the activation of the MAPK (Mitogen-activated protein kinases) pathway, driven by various somatic mutations in the specific lesions of…
- Trial name
- BIOHISTIO (NCT06197204)
- Testing
- Blood sampling, Biopsy
- Where
- France
- Volunteers
- 570 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Psychiatric Adults Patients
Screening of Niemann-Pick Disease, Type C in a Psychiatric Population
Niemann-Pick disease, Type C is a rare genetic disorder characterized by a failing in intracellular cholesterol transport, inducing an accumulation of sphingolipids in the brain.
- Trial name
- NPCPsy (NCT02841358)
- Testing
- Blood sampling, Biopsy
- Where
- France
- Volunteers
- 22 people
- Sponsor
- University Hospital, Grenoble
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
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
Recurrent Ependymoma, Recurrent Ewing Sarcoma
Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well ivosidenib works in treating patients with solid tumors, including central nervous system tumors, lymphomas and histiocytic disorders that have not responded to (refractory) or…
- Conditions
- Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis
- Trial name
- NCT04195555
- Testing
- Ivosidenib
- Where
- 174 sites in United States, Australia and 1 more
- Volunteers
- 3 people
- Sponsor
- National Cancer Institute (NCI)
Histiocytosis, Langerhans-cell
Reduced Intensity Hematopoietic Cell Transplantation for Patients With Resistant Langerhans Cell Histiocytosis
RATIONALE: Giving a monoclonal antibody, such as alemtuzumab, and chemotherapy drugs, such as fludarabine and melphalan, before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem…
- Trial name
- NCT00618540
- Testing
- alemtuzumab, fludarabine phosphate, melphalan
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Histiocytosis, Histiocytosis, Langerhans-Cell
Adult Pulmonary Langerhans Cell Histiocytosis: a National Registry-based Prospective Cohort Study
The long-term outcomes of adult patients with pulmonary Langerhans cell histiocytosis (PLCH), particularly survival, is largely unknown.
- Conditions
- Histiocytosis, Histiocytosis, Langerhans-Cell, Histiocytosis Pulmonary
- Trial name
- NCT04665674
- Testing
- Standard of care
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hemophagocytic Lymphohistiocytosis, Epstein-Barr Virus
Segmental Resection Combined With DEP Regimen for EBV-HLH Patients With Intestinal Involvement
EBV-HLH is a rare disease with high mortality, especially for those with intestinal involvement. In order to reduce disease burden and improve survival of these patients, we conduct a prospective observational study to explore…
- Conditions
- Hemophagocytic Lymphohistiocytosis, Epstein-Barr Virus, Bowel Resection
- Trial name
- NCT06131489
- Testing
- Segmental bowel resection combined with the DEP regimen
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Beijing Friendship Hospital
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal…
- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1
- Trial name
- RICHI (NCT01998633)
- Testing
- Hematopoietic Stem Cell Transplant
- Where
- 22 sites in United States, Canada
- Volunteers
- 47 people
- Sponsor
- Medical College of Wisconsin
Primary Hemophagocytic Lymphohistiocytosis
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term…
- Trial name
- NCT03312751
- Testing
- Emapalumab
- Where
- 27 sites in United States, Canada and 6 more
- Volunteers
- 35 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophagocytic Lymphohistiocytosis (HLH)
All-Trans Retinoic Acid for the Treatment of Hemophagocytic Lymphohistiocytosis
This study is designed to evaluate the safety and preliminary efficacy of all-trans retinoic acid (ATRA) as an initial treatment for patients with active hemophagocytic lymphohistiocytosis (HLH).
- Trial name
- NCT07626398
- Testing
- ATRA
- Volunteers
- 30 people
- Sponsor
- Beijing Anzhen Hospital
Sphingomyelin Lipidosis
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
- Trial name
- NCT02004704
- Testing
- GZ402665
- Where
- 9 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
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
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations (mutations) in cell cycle genes…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03526250
- Testing
- Laboratory Biomarker Analysis, Palbociclib, Pharmacological Study
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 23 people
- Sponsor
- National Cancer Institute (NCI)
Niemann-Pick Disease, Type C1
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
- Trial name
- NCT03201627
- Testing
- Lithium Carbonate
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
HLH, Hemophagocytic Lymphohistiocytoses
Establishing a National Egyptian HLH Registry (NEHR)
Establishing a National Egyptian HLH Registry (NEHR)
- Trial name
- NEHR (NCT07800234)
- Where
- 2 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Ain Shams University
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Septic Shock, Acute Respiratory Distress Syndrome
CytOSorb TreatMent Of Critically Ill PatientS Registry
Registry intended to provide a data repository and reporting infrastructure for the surveillance of CytoSorb device use in real-world critical care settings, and to serve as an objective, comprehensive, and scientifically-based…
- Conditions
- Septic Shock, Acute Respiratory Distress Syndrome, Trauma, Rhabdomyolysis
- Trial name
- COSMOS (NCT05146336)
- Testing
- CytoSorb
- Where
- 28 sites in Austria, Germany and 4 more
- Volunteers
- 3,000 people
- Sponsor
- CytoSorbents, Inc