Clinical trials: Histiocytosis,Langerhans-Cell
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Non-Hodgkin Lymphoma, Multiple Myeloma
In-Human CXCR4 Imaging of Hematologic and Solid Tumors Using [68Ga]-Pentixafor-PET
The purpose of this study is to determine the uptake of the imaging agent [68Ga]-pentixafor with PET/CT scans in people with monoclonal gammopathy of undetermined significance (MGUS), smoldering multiple myeloma (SMM), and…
- Conditions
- Non-Hodgkin Lymphoma, Multiple Myeloma, Histiocytic Neoplasms, Erdheim-Chester Disease
- Trial name
- NCT05093335
- Testing
- [68Ga]-Pentixafor
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
Langerhans Cell Histiocytosis (LCH), Histiocytoses Erdheim-chester Disease
Lenalidomide for Adult Histiocyte Disorders
This research study is studying a chemotherapy drug Lenalidomide as a possible treatment for one of three histiocyte disorders: Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), or histiocytic sarcoma (HS).
- Conditions
- Langerhans Cell Histiocytosis (LCH), Histiocytoses Erdheim-chester Disease, Histiocytic Sarcoma (HS)
- Trial name
- NCT02523040
- Testing
- Lenalidomide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Dana-Farber Cancer Institute
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Gaucher Disease, Acid SphingoMyelinase Deficiency
PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and oncohematology clinics, and adult internal medicine and hematology.
- Conditions
- Gaucher Disease, Acid SphingoMyelinase Deficiency, ASMD, Niemann-Pick Diseases
- Trial name
- PREDIGA-2 (NCT05641103)
- Testing
- Patient with splenomegaly or splenectomy
- Where
- 14 sites in Spain
- Volunteers
- 122 people
- Sponsor
- Fundación Española de Hematología y Hemoterapía
Langerhans Cell Histiocytosis
Safety and Efficacy of CD207 Targeted CAR-T Cell Therapy in Patients With R/R Langerhans Cell Histiocytosis
This is a single-arm study to evaluate the efficacy and safety of CD207 targeted CAR-T cell therapy in relapsed and refractory langerhans cell histiocytosis.
- Trial name
- NCT05477446
- Testing
- CD207 CAR-T cells
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Beijing Friendship Hospital
Langerhans Cell Histiocytosis
A Combination of Vemurafenib, Cytarabine and 2-chlorodeoxyadenosine in Children With LCH and BRAF V600E Mutation
Langerhans cell histiocytosis (LCH) is a disease caused by clonal expansion, proliferation, and dissemination of cells that are phenotypically close to Langerhans cells in different tissues and organs.
- Trial name
- NCT03585686
- Testing
- Vemurafenib, Cytarabine, 2-chlorodeoxyadenosine
- Where
- Russia
- Volunteers
- 12 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Macrophage Activation Syndrome, Primary Hemophagocytic Lymphohistiocytosis
Retrospective Study of Immunotherapy Related Toxicities and Factors Impacting Outcomes in Children and Adults With Cancer
Immunotherapy is changing the landscape of cancer therapy. Particularly unique to immunotherapy is the toxicity profile, which differs from chemotherapy-based strategies and can be associated with inflammatory responses and/or…
- Trial name
- NCT03827343
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Cancer Institute (NCI)
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical…
- Trial name
- NCT04877132
- Testing
- olipudase alfa (GZ402665)
- Sponsor
- Sanofi
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
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
Childhood Langerhans Cell Histiocytosis, Leukemia
Umbilical Cord Blood and Placental Blood Transplantation in Treating Patients With Hematologic Cancer or Aplastic Anemia
RATIONALE: Umbilical cord blood or placental blood transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy that was used to kill cancer cells.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00008164
- Testing
- umbilical cord blood transplantation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Herbert Irving Comprehensive Cancer Center
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
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 Type C Disease
Miglustat in Niemann-Pick Type C Disease
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a…
- Trial name
- NCT00517153
- Testing
- miglustat
- Volunteers
- 29 people
- Sponsor
- Actelion
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
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
Langerhans Cell Histiocytosis (LCH)
Luvometinib in Pediatric SS-LCH With Special-site Single/Multifocal Bone Lesions
Langerhans cell histiocytosis (LCH) is the most common type of histiocytic disorder in children, affecting about 2.6 to 8.9 out of every million kids each year.
- Trial name
- NCT07371182
- Testing
- Luvometinib
- Where
- 11 sites in China
- Volunteers
- 62 people
- Sponsor
- West China Second University Hospital
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