Clinical trials: Histiocytosis,Langerhans-Cell
Histiocytosis, Langerhans Cell Histiocytosis
Histiocytic Disorder Follow-up Study
The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific…
- Conditions
- Histiocytosis, Langerhans Cell Histiocytosis, Erdheim-Chester Disease, Rosai Dorfman Disease
- Trial name
- NCT05915208
- Where
- United States
- Volunteers
- 6,000 people
- Sponsor
- University of Alabama at Birmingham
Hemophagocytic Lymphohistiocytosis, B Cell Lymphoma
Zanubrutinib Combined With Rituximab in the Treatment of Secondary HLH in B-cell Lymphoma
For patients who met the inclusion criteria, treatment regimens were administered: Rituximab 375 mg/m², intravenously, once weekly for 4 weeks. Zanubrutinib 160 mg, orally, twice daily for 4 weeks.
- Trial name
- NCT07270835
- Testing
- Rituximab, Zanubrutinib
- Where
- China
- Volunteers
- 40 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Rosai-Dorfman Disease, Langerhans Cell Histiocytosis
Central China Rosai-Dorfman Disease Registry
This is a research study among patients with Rosai-Dorfman disease.
- Trial name
- CCR-DDR (NCT05284942)
- Testing
- Mycophenolate mofetil combined with oral prednisone
- Where
- 2 sites in China
- Volunteers
- 20 people
- Sponsor
- Tongji Hospital
Advanced Tumors, Melanoma
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patient With Advanced Tumors
This is a First-in-Human, Open Label, Phase I/II Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patients with Advanced Tumors, composed of a Phase I dose escalation and dose expansion stage and a…
- Conditions
- Advanced Tumors, Melanoma, Non-Small-Cell Lung Cancer, Erdheim-Chester Disease
- Trial name
- NCT04198818
- Testing
- HH2710
- Where
- 3 sites in United States, China
- Volunteers
- 37 people
- Sponsor
- Haihe Biopharma Co., Ltd.
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 Disease, Type C
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
This is a prospective non-therapeutic observational study in NP-C patients. The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical…
- Trial name
- NCT02435030
- Where
- 16 sites in Denmark, France and 6 more
- Volunteers
- 35 people
- Sponsor
- ZevraDenmark
Gaucher Disease, Acid SphingoMyelinase Deficiency
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and oncohematology clinics, and adult internal medicine and hematology.
- Trial name
- NCT05992532
- Where
- 23 sites in Spain
- Volunteers
- 210 people
- Sponsor
- Fundación Española de Hematología y Hemoterapía
Niemann-Pick Type C Disease
Miglustat in Niemann-Pick Type C Disease
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a…
- Trial name
- NCT00517153
- Testing
- miglustat
- Volunteers
- 29 people
- Sponsor
- Actelion
Acid Sphingomyelinase Deficiency (ASMD)
Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
Acid sphingomyelinase Deficiency known as Neiman _PICK disease is a group of rare genetic diseases. This study includes analysis of clinical manifestations in patients with ASMD and investigations done for diagnosis of these…
- Trial name
- NCT07274826
- Where
- Egypt
- Volunteers
- 7 people
- Sponsor
- Sohag University
Sphingomyelin Lipidosis
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Primary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult…
- Trial name
- ASCEND (NCT02004691)
- Testing
- Olipudase alfa
- Where
- 24 sites in United States, Argentina and 15 more
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Haematological Malignancy, Malignant Neoplasm
DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers.
This clinical trial is looking at two drugs called dabrafenib and trametinib. Dabrafenib and trametinib are approved as standard of care treatment for adult patients with melanoma (a type of skin cancer) or lung cancer and in…
- Conditions
- Haematological Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders, Neoplasms by Histologic Type
- Trial name
- DETERMINE (NCT07440290)
- Testing
- Dabrafenib, Trametinib
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
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
Niemann-Pick Disease, Niemann-Pick Disease, Type C
Biomarker for Niemann Pick Type C Disease (BioNPC)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)
- Trial name
- BioNPC (NCT01306604)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
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
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
Hemophagocytic Lymphohistiocytoses
A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetic Characteristics, and Immunogenicity of Plonmarlimab in Subjects With Rheumatic and Immunological Disease-associated Haemophagocytic Lymphohistiocytosis (HLH) (Also Known as Macrophage Activation Syndrome (MAS))
This study adopts an open-label, single-arm, multicenter design to evaluate the efficacy, safety, tolerability, immunogenicity, and PK characteristics of Plonmarlimab administration in patients with rheumatic and immunological…
- Trial name
- NCT07034209
- Testing
- Plonmarlimab
- Where
- 6 sites in China
- Volunteers
- 18 people
- Sponsor
- TJ Biopharma Co., Ltd.
Langerhans Cell Histiocytosis
Study of Sequential Administration of Oral 6-Thioguanine After Methotrexate in Patients With LCH
The objective of this study is to determine the incidence of complete and partial response and the duration of response in patients with Langerhans Cell Histiocytosis (LCH) treated with sequential administration of oral…
- Trial name
- NCT00588536
- Testing
- Methotrexate, 6-Thioguanine, Leucovorin Calcium
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Hemophagocytic Lymphohistiocytosis
Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis
Despite good progress during the last decade, hemophagocytic lymphohistiocytosis (HLH) remains difficult to treat. Two different treatment regimens have been used successfully.
- Trial name
- NCT01104025
- Testing
- ATG, rabbit, Etoposide, Methotrexate
- Where
- 15 sites in United States, Canada
- Volunteers
- 31 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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)