Clinical trials: Histiocytosis,Langerhans-Cell
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
HLH, Hemophagocytic Lymphohistiocytoses
Prospective Validation of the OHI Index
Hemophagocytic lymphohistiocytosis (HLH) associated with hematologic malignancies (HM-HLH) is a syndrome with an abysmal prognosis (10-30% 5 years overall survival).
- Conditions
- HLH, Hemophagocytic Lymphohistiocytoses, Hemophagocytic Syndrome, Hematologic Malignancy
- Trial name
- HLH (NCT05882175)
- Where
- 2 sites in Israel
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Meir Medical Center
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
Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment.
- Conditions
- Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma, Erdheim-Chester Disease, Rosai Dorfman Disease
- Trial name
- NACHO-COBI (NCT04079179)
- Testing
- Cobimetinib
- Where
- 12 sites in United States
- Volunteers
- 90 people
- Sponsor
- Carl Allen
BRAF V600E Mutation
Dabrafenib and Trametinib in People With BRAF V600E Mutation Positive Lesions in Erdheim Chester Disease
Erdheim-Chester Diseases (ECD) is a very rare non-Langerhans cell histiocytosis of unknown origin and pathogenesis. It has been reported mainly in adult males over the age of 40 years, although cases have been reported in females…
- Trial name
- NCT02281760
- Testing
- Dabrafenib Mesylate, Trametinib Dimethyl Sulfoxide
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Hemophagocytic Lymphohistiocytosis
Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohistiocytosis
This observational study is designed to determine the levels of pro-inflammatory markers in patients diagnosed with M-HLH, and to assess whether the cytokine profiles bear an IFNγ signature.
- Trial name
- NCT03259230
- Testing
- Blood Draws, Data Collection
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Swedish Orphan Biovitrum
Fabry Disease, Gaucher Disease
Complement Activation in the Lysosomal Storage Disorders
The lysosomal storage disorders (LSDs) are monogenic disorders associated with inflammation affecting multiple organs, and early death.
- Conditions
- Fabry Disease, Gaucher Disease, Niemann-Pick Disease, Type C, Lysosomal Storage Diseases
- Trial name
- CATALYST (NCT04189601)
- Testing
- Complement measurements
- Where
- Australia
- Volunteers
- healthy volunteers welcome
- Sponsor
- Melbourne Health
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
Langerhans Cell Histiocytosis
LCH in Adults: a Collaborative, Prospective-retrospective, Observational Study
This is a multicenter, retrospective and prospective, observational, no profit study including adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient.
- Trial name
- NCT04627090
- Testing
- Data collection
- Where
- 8 sites in Italy
- Volunteers
- 186 people
- Sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Nasopharyngeal Carcinoma (NPC)
Brain Alterations Based on Multiplex MR Imaging in Nasopharyngeal Carcinoma Patients After Treatment, and Their Correlation With Clinical Cognitive Scores.
The objective of this study is to examine the potential impact of the treatment before, during, and/or after radiotherapy on the brain in patients with nasopharyngeal carcinoma (NPC) using Multiplex (MTP) MR imaging sequence…
- Trial name
- NPC (NCT07011329)
- Testing
- Changes in the brain MTP parameters after radiotherapy
- Where
- China
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hubei Cancer Hospital
Lymphoma, Hemophagocytic Lymphohistiocytoses
Describing Treatment Outcomes and Responses in Lymphoma-associated Hemophagocytic Lymphohistiocytosis
The purpose of the study is to describe Hemophagocytic lymphohistiocytosis (HLH) in patients with lymphoma to identify possible therapeutic strategies to improve overall survival of the patients with lymphoma associated…
- Trial name
- NCT05898477
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- University of Alabama at Birmingham
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
Langerhans Cell Histiocytosis, Erdheim-Chester Disease
The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
The purpose of this study was to assess safety, efficacy and PK in adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) given HLX208 (BRAF V600E inhibitor).
- Conditions
- Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD
- Trial name
- NCT05092815
- Testing
- HLX208
- Where
- China
- Volunteers
- 25 people
- Sponsor
- Shanghai Henlius Biotech
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Granulomatosis With Polyangiitis, Hemophagocytic Syndrome
Study on Pharmacokinetics of Meperizumab Injection and NUCALA® in Healthy Male Volunteers
The trial was designed as a single-center, randomized, double-blind, single-dose parallel controlled phase I study to evaluate the similarity of pharmacokinetics and safety of Meperizumab injection and NUCALA® in healthy male…
- Trial name
- NCT05353179
- Testing
- Meperizumab injection, NUCALA®
- Where
- China
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Niemann-Pick Disease, Type C
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).
- Trial name
- NCT02612129
- Testing
- arimoclomol
- Where
- 14 sites in United States, Denmark and 7 more
- Volunteers
- 50 people
- Sponsor
- ZevraDenmark
Autoimmune Lymphoproliferative Syndrome, Autoimmune Cytopenia
Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation
The main objective of this study is to generate diagnosis and therapeutic-decision tools through the identification of molecular causes of PIDs with autoimmunity/inflammation and the variability in disease outcome at the…
- Conditions
- Autoimmune Lymphoproliferative Syndrome, Autoimmune Cytopenia, Autoimmune Diseases, Autoimmune Anemia
- Trial name
- ATRACTion (NCT04902807)
- Testing
- Collection of samples
- Where
- France
- Volunteers
- 325 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France