Clinical trials: Histiocytosis,Langerhans-Cell
Primary Haemophagocytic Lymphohistiocytosis
Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis.
- Trial name
- NCT01818492
- Testing
- NI-0501
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 45 people
- Sponsor
- Swedish Orphan Biovitrum
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
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
Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor
Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well ulixertinib works in treating patients with solid tumors that have spread to other places in the body (advanced), non-Hodgkin lymphoma, or histiocytic disorders that have a…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Ependymal Tumor, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03698994
- Testing
- Pharmacokinetic Study, Ulixertinib
- Where
- 107 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
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
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
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders
Stem Cell Transplant for Immunologic or Histiocytic Disorders
This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of…
- Conditions
- Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome
- Trial name
- NCT00176865
- Testing
- Stem Cell Transplant, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
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
Hemophagocytic Syndrome (HPS)
A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
This is a pilot study to determine the efficacy of Ruxolitinib in secondary hemophagocytic syndrome. The primary objective is to assess the efficacy of ruxolitinib 15 mg PO twice daily in patients with HPS.
- Trial name
- NCT02400463
- Testing
- Ruxolitinib
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Michigan Rogel Cancer Center
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
Langerhans Cell Histiocytosis
Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
- Trial name
- NCT02425904
- Testing
- Clofarabine
- Where
- 15 sites in United States, Canada
- Volunteers
- 25 people
- Sponsor
- Dana-Farber Cancer Institute
Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic…
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01273766
- Testing
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Wake Forest University Health Sciences
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
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
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
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)
Hemophagocytic Lymphohistiocytosis
A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under…
- Trial name
- NCT02069899
- Testing
- Emapalumab
- Where
- 16 sites in United States, France and 3 more
- Volunteers
- 58 people
- Sponsor
- Swedish Orphan Biovitrum
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma
- Trial name
- NCT03220035
- Testing
- Laboratory Biomarker Analysis, Vemurafenib
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 4 people
- Sponsor
- National Cancer Institute (NCI)
Hurler's Syndrome, Maroteaux-Lamy Syndrome
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell…
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis
- Trial name
- NCT00668564
- Testing
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Masonic Cancer Center, University of Minnesota