Clinical trials: Histiocytosis,Langerhans-Cell
Haemophagocytic Lymphohistiocytosis
Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)
The purpose of this project is to study the survival of patients until Haematopoietic Stem Cell Transplantation following the use of Ruxolitinib as first-line treatment associated to corticosteroids in primary HLH.
- Trial name
- R-HLH (NCT05762640)
- Testing
- Ruxolitinib
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
NHL With Hemophagocytic Lymphohistiocytosis
Trial of DA-EPOCH Regimen for NHL With HLH
The major purpose of this clinical study is to assess clinical outcome of dose-adjusted EPOCH regimen for patients with non-Hodgkin's lymphoma(NHL)-associated hemophagocytic lymphohistiocytosis
- Trial name
- NCT01818908
- Testing
- DA-EPOCH
- Where
- 4 sites in China
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- The First Affiliated Hospital with Nanjing Medical University
Blood And Marrow Transplantation, Leukemia
Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies
The goal of this clinical research study is to learn if it is safe and feasible to transplant changed cord blood for patients with leukemia or lymphoma. Researchers also want to learn if this can help to control the disease.
- Conditions
- Blood And Marrow Transplantation, Leukemia, Lymphoma, Transplantation Infection
- Trial name
- NCT01471067
- Testing
- Melphalan, Fludarabine, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- M.D. Anderson Cancer Center
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
Adrenal Gland Pheochromocytoma, Carcinoma In Situ
Project: Every Child for Younger Patients With Cancer
This study gathers health information for the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and…
- Conditions
- Adrenal Gland Pheochromocytoma, Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma
- Trial name
- NCT02402244
- Testing
- Cytology Specimen Collection Procedure, Medical Chart Review
- Where
- 279 sites in United States, Australia and 4 more
- Volunteers
- 75,000 people
- Sponsor
- Children's Oncology Group
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Primary Hemophagocytic Lymphohistiocytosis
Emapalumab Efficacy in Children With Primary Hemophagocytic Lymphohistiocytosis
The goal of this retrospective study is to assess efficacy and safety of emapalumab prescribed for the treatment in children with refractory from of pHLH.
- Trial name
- CGM EMA (NCT06587191)
- Where
- Russia
- Volunteers
- 7 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma
Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a…
- Conditions
- Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma
- Trial name
- NCT04284774
- Testing
- Tipifarnib
- Where
- 172 sites in United States, Puerto Rico
- Volunteers
- 5 people
- Sponsor
- National Cancer Institute (NCI)
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
Adult Langerhans Cell Histiocytosis, Childhood Langerhans Cell Histiocytosis
Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy drugs and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Adult Langerhans Cell Histiocytosis, Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma
- Trial name
- NCT00008216
- Testing
- Peripheral Blood Stem Cell Transplantation
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Columbia University
Hemophagocytic Lymphohistiocytoses, Cytokine Storm
Cytokine Guided Risk Stratification and Treatment in Pediatric Hemophagocytic Lymphohistiocytosis
Hemophagocytic lymphohistiocytosis (HLH) is a rapidly fatal disease caused by immune-dysregulation characterized by hypercytokinemia, with about 30%-40% of patients suffering death in children.
- Trial name
- NCT05491304
- Testing
- Dexamethasone, Etoposide, Ruxolitinib
- Where
- China
- Volunteers
- 400 people
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
GM2 Gangliosidosis, Niemann-Pick Disease, Type C
Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C…
- Trial name
- RAINBOW (NCT05758922)
- Testing
- AZ-3102 (Dose 1), AZ-3102 (Dose 2)
- Where
- 3 sites in Brazil
- Volunteers
- 13 people
- Sponsor
- Azafaros B.V.
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
Acid Sphingomyelinase Deficiency (ASMD), Niemann Pick Disease
National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
The goal of this study is to describe the natural history of ASMD in adult and paediatric patients with or without specific treatment in order to assess the impact of the disease on their daily lives and quality of life.
- Trial name
- FASMD (NCT06985212)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Wladimir MAUHIN, Dr
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.
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
Langerhans Cell Histiocytosis (LCH)
Modified LCH-III Regimen With or Without Luvometinib for Multisystem Pediatric Langerhans Cell Histiocytosis
Langerhans cell histiocytosis (LCH) is the most common histiocytic disorder in children, caused by excessive proliferation and accumulation of Langerhans cells (a type of immune cell) in various body tissues.
- Trial name
- NCT07431060
- Testing
- Prednisone, Vincristine, Mercaptopurine
- Where
- 11 sites in China
- Volunteers
- 120 people
- Sponsor
- West China Second University Hospital
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
Langerhans Cell Histiocytosis
Denosumab for the Treatment of Adult LCH
This study is aiming to evaluate the efficacy of denosumab among adult patients suffering from Langerhans Cell Histiocytosis (LCH).
- Trial name
- NCT03270020
- Testing
- Denosumab 70 MG/ML [Xgeva]
- Where
- Greece
- Volunteers
- 10 people
- Sponsor
- Hellenic Society for the Study of Bone Metabolism