Clinical trials: Langerhans-Cell Histiocytosis
Rosai-Dorfman Disease
Lenalidomide and Dexamethasone for Rosai-Dorfman Disease
Rosai-Dorfman Disease (RDD) is a rare, heterogeneous histiocytic disorder. Because of the rarity of RDD and a lack of prospective randomized trials, the treatment strategy for RDD is mostly based on retrospective study.
- Trial name
- NCT04924647
- Testing
- lenalidomide and dexamethasone
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Peking Union Medical College Hospital
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
Pulmonary Langerhans Cell Histiocytosis
Assessment of Safety of Air Travel in Patients With Pulmonary Langerhans Cell Histiocytosis
The aim of this study is to conduct survey-based assessments for the safety of air travel in patients with pulmonary Langerhans cell histiocytosis (PLCH).
- Trial name
- NCT03052101
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- University of Cincinnati
Hemophagocytic Lymphohistiocytosis
Comparison of Two Etoposide Initiation Strategies for Severe Hemophagocytic Lymphohistiocytosis
Hemophagocytic lymphohistiocytosis (HLH) is an immune-mediated disorder characterized by hyperactivation of the immune system, leading to a cytokine storm responsible for organ failures.
- Trial name
- TIC-TAC-SAM (NCT07497438)
- Testing
- Etoposide (Early strategy), Etoposide (Delayed strategy)
- Where
- France
- Volunteers
- 176 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
I Cell Disease, Fucosidosis
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory…
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy
- Trial name
- NCT00005900
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Fairview University Medical Center
Niemann-Pick Diseases
Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
- Trial name
- NCT04469894
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- National Niemann-Pick Disease Foundation
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
Langerhans Cell Histiocytosis of Lung
Evaluation of Efficacy and Tolerance of Cladribine in Symptomatic Pulmonary Langerhans Cell Histiocytosis
ECLA is a phase II, multicenter study testing sub cutaneous cladribine 0.1mg/kg/j during 5 days, administrated every month for 4 courses, in symptomatic adult patients with pulmonary Langerhans cell histiocytosis and impairment…
- Trial name
- ECLA (NCT01473797)
- Testing
- Cladribine
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Splenomegaly, Splenectomy
Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease
The goal of this clinical trial is to optimise and facilitate screening for Acid SphingoMyelinase Deficiency (ASMD) disease, by evaluating acid sphingomyelinase activity and, where appropriate, LysoSM levels in a cohort of 200…
- Conditions
- Splenomegaly, Splenectomy, Thrombopenia, Interstitial Lung Disease (ILD)
- Trial name
- Niemann-PID (NCT06869499)
- Testing
- Blood sampling for dosage
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Wladimir MAUHIN, Dr
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Langerhans Cell Histiocytosis
Evaluation of Exercise Capacity and Exercise Limitation in Patients With Pulmonary Langerhans Cell Histiocytosis
Pulmonary Langerhans Histiocytosis Cells (PLCH) is characterized by infiltration of Langerhans cells and formation of loose granulomas with lymphocytic infiltrate and formation of nodular and cystic lesions on chest CT, and is…
- Trial name
- NCT02665546
- Where
- Brazil
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- InCor Heart Institute
Niemann-Pick Disease, Type C
Safety and Efficacy of Miglustat in Chinese NPC Patients
This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study.
- Trial name
- NCT03910621
- Testing
- Miglustat
- Where
- 2 sites in China
- Volunteers
- 17 people
- Sponsor
- Actelion
Langerhans Cell Histiocytosis
Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH
Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.
- Trial name
- NCT07187193
- Testing
- Cytarabine (Ara-C), Thalidomide (100mg)
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
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
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia
Umbilical Cord Blood Transplant With Added Sugar and Chemotherapy and Radiation Therapy in Treating Patients With Leukemia or Lymphoma
This phase II trial studies how well an umbilical cord blood transplant with added sugar works with chemotherapy and radiation therapy in treating patients with leukemia or lymphoma.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia, Acute Leukemia, Acute Lymphoblastic Leukemia
- Trial name
- NCT03096782
- Testing
- Anti-Thymocyte Globulin, Busulfan, Clofarabine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- M.D. Anderson Cancer Center
Hemophagocytic Lymphohistiocytosis
DEP-Ru Regimen as a Salvage Therapy for HLH
This study aimed to investigate the efficacy and safety of ruxolitinib together with liposomal doxorubicin, etoposide and methylprednisolone (DEP-Ru) as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis.
- Trial name
- NCT03533790
- Testing
- DEP-Ru
- Where
- China
- Volunteers
- 80 people
- Sponsor
- Beijing Friendship Hospital
Leukemia
Alemtuzumab or Tocilizumab in Combination With Etoposide and Dexamethasone for the Treatment of Adult Patients With Hemophagocytic Lymphohistiocytosis
The goal of this clinical research study is to compare the effect of adding either alemtuzumab or tocilizumab to the drug combination of etoposide and dexamethasone in controlling HLH.
- Trial name
- NCT02385110
- Testing
- Alemtuzumab, Etoposide, Dexamethasone
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- M.D. Anderson Cancer Center
Histiocytosis, Langerhans-Cell
Cardiovascular Risk Factors and LCH in Adults
Langerhans-cell histiocytosis (LCH) is a rare disease with features of chronic inflammation and hypopituitarism, conditions associated with increased risk of cardiovascular diseases.
- Trial name
- NCT00483925
- Where
- Greece
- Volunteers
- healthy volunteers welcome
- Sponsor
- Laikο General Hospital, Athens
Hemophagocytic Lymphohistiocytoses
Prospective Cohort for Adult Hemophagocytosis
This prospective study enrolls subjects who have clinical and laboratory manifestations related with hemophagocytic lymphohistiocytosis.
- Trial name
- NCT03117010
- Testing
- Steroids, Etoposide
- Where
- South Korea
- Volunteers
- 81 people
- Sponsor
- Samsung Medical Center
Langerhans Cell Histiocytosis
Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib
The purpose of this Phase II clinical trial is to establish the safety and effectiveness of trametinib, a targeted therapy, for the treatment of newly or recently diagnosed Langerhans Cell Histiocytosis (LCH) among pediatric…
- Trial name
- NCT06582745
- Testing
- Trametinib
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Cook Children's Health Care System