Clinical trials: Histiocytosis,Langerhans-Cell
Advanced Malignant Neoplasm, Castleman Disease
Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease
This phase I trial studies the side effects and best dose of bevacizumab and temsirolimus alone or in combination with valproic acid or cetuximab in treating patients with a malignancy that has spread to other places in the body…
- Conditions
- Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease
- Trial name
- NCT01552434
- Testing
- Bevacizumab, Cetuximab, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 154 people
- Sponsor
- M.D. Anderson Cancer Center
Langerhans Cell Histiocytosis (LCH)
Real-world Study of Darafenib or Trametinib and Clofarabine for High-risk/Recurrent/Refractory Langerhans Cell Histiocytosis in Children
Langerhans cell histiocytosis (LCH) is the most common histiocytosis in children, with an incidence of 2.6-8.9 per million. It is an inflammatory myeloid tumor with varied symptoms.
- Trial name
- NCT07022834
- Testing
- Dabrafenib or Trametinib and Clofarabine
- Where
- China
- Volunteers
- 20 people
- Sponsor
- West China Second University Hospital
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
Investigation of Ferritin in Critically Ill Patients With Hemophagocytic Lymphohistiocytosis
Retrospective analysis of ferritin, outcome and HLH-criteria in critically ill patients.
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS), Hemophagocytic Syndromes
- Trial name
- FERRITS (NCT02854943)
- Volunteers
- 2,623 people
- Sponsor
- Gunnar Lachmann
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
Niemann-Pick Disease, Type C
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC).
- Trial name
- NCT05163288
- Testing
- N-Acetyl-L-Leucine
- Where
- 14 sites in United States, Australia and 6 more
- Volunteers
- 53 people
- Sponsor
- IntraBio Inc
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
L-DEP as an Initial Treatment for EBV-HLH
This study aimed to investigate the efficacy and safety of Pegaspargase together with liposomal doxorubicin, etoposide and high dose methylprednisolone (L-DEP) as an initial treatment for Epstein Barr virus-induced hemophagocytic…
- Trial name
- NCT02912702
- Testing
- Pegaspargase, doxorubicin hydrochloride liposome injection, etoposide
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Beijing Friendship Hospital
Hemophagocytic Lymphohistiocytoses, Lymphoma
Sensitivity and Specificity of Cytokines in the Diagnosis of Lymphoma-associated HLH
Lymphoma-associated hemophagocytic lympohistiocytosis is a refractory immune disorder with high mortality. Early identification and diagnosis of lymphoma-associated HLH has become the key to improve the prognosis of…
- Trial name
- NCT05600959
- Testing
- cytokine
- Where
- China
- Volunteers
- 89 people
- Sponsor
- Beijing Friendship Hospital
Hemophagocytic Lymphohistiocytoses
Ruxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)
This phase II trial tests the effects of ruxolitinib in combination with a de-intensified HLH-94 drug regimen has on patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH), a disorder caused by dysregulated immune…
- Trial name
- NCT06160791
- Testing
- Ruxolitinib, Etoposide, Dexamethasone
- Where
- 4 sites in United States
- Volunteers
- 54 people
- Sponsor
- Jerry Lee, MD, MSc, MPhil
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
Advanced Malignant Solid Neoplasm, Recurrent Ependymoma
Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well larotrectinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with NTRK fusions that may have spread from where it first started to…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma
- Trial name
- NCT03213704
- Testing
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan
- Where
- 124 sites in United States, Puerto Rico
- Volunteers
- 9 people
- Sponsor
- National Cancer Institute (NCI)
Asmd, Visceral Type
Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency
The goal of this study is to assess the ability of MRI techniques to detect early stages of lipid accumulation in the liver of ASMD patients with the chronic visceral subtype compared to healthy subjects.
- Trial name
- MONACO (NCT05904366)
- Testing
- MR Spectroscopy (MRS), MR Elastography (MRE)
- Where
- Netherlands
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Eline C. B. Eskes
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma
- Trial name
- NCT00084695
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Milton S. Hershey Medical Center
Lysosomal Storage Disorders, Gaucher Disease
Investigating Lysosomal Storage Diseases in Minority Groups
Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US.
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease
- Trial name
- NCT02120235
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Hemophagocytic Lymphohistiocytoses
Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis
This observational study is designed to provide a better understanding of the potential use of interferon gamma (IFNγ) and IFNγ-inducible chemokines as markers of HLH disease activity in adults, and the potential of IFNγ to…
- Trial name
- NCT06047210
- Testing
- Blood Draws Data Collection
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Swedish Orphan Biovitrum
Lysosomal Storage Disorders
Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
The lysosome is a specialized part of the cell that functions to degrade metabolic wastes in the cell. Defects in the functioning of the lysosome result in accumulation and subsequent storage of such metabolic wastes.
- Trial name
- NCT02000310
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
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
Dementia, Neurodegenerative Diseases
Retinal Hyperspectral Imaging in Neurodegenerative Diseases
Hyperspectral retinal imaging is a non-invasive imaging modality in which a series of images of the retina are captured using light of different wavelengths.
- Conditions
- Dementia, Neurodegenerative Diseases, Alzheimer Disease, Parkinson Disease
- Trial name
- NCT07545473
- Testing
- Hyperspectral camera
- Where
- Australia
- Volunteers
- 930 people, healthy volunteers welcome
- Sponsor
- Center for Eye Research Australia
Niemann-Pick Disease, Type C
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick Disease, Type C (NPC), a genetic disorder that results in progressive…
- Trial name
- NCT00344331
- Where
- United States
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.