Clinical trials: Histiocytosis,Langerhans-Cell
Sphingomyelin Lipidosis
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
Primary Objective: To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD To describe…
- Trial name
- NCT04106544
- Testing
- Investigational Procedures
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 84 people
- Sponsor
- Sanofi
Hemophagocytic Lymphohistiocytoses
INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)
The purpose of this observational study is to collect data on the natural history of disease of patients with Hemophagocytic Lymphohistiocytosis (HLH) including diagnosis, treatments, responses, and outcomes.
- Trial name
- NCT05277272
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Nonneoplastic Condition
Combination Chemotherapy Followed By Donor Stem Cell Transplant in Treating Patients With Hemophagocytic Lymphohistiocytosis
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of hemophagocytic lymphohistiocytosis cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00334672
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- 13 sites in United Kingdom
- Volunteers
- 288 people
- Sponsor
- Children's Cancer and Leukaemia Group
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
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH treatment trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back…
- Conditions
- Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
- Trial name
- NCT03213652
- Testing
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan
- Where
- 126 sites in United States, Australia and 2 more
- Volunteers
- 13 people
- Sponsor
- National Cancer Institute (NCI)
Erdheim-Chester Disease, Langerhans Cell Histiocytosis
Uptake and Biodistribution of 18F-fluorocholine in Histiocytic Disorders by PET Imaging and Biopsy Measurement
The main purpose of this study is to discover how 18F-cholineFCH distributes in the body, and see if it can distinguish active histiocytes (tumor) from inflammatory (non- tumor) cells.
- Conditions
- Erdheim-Chester Disease, Langerhans Cell Histiocytosis, Histiocytic Disorders
- Trial name
- NCT02608619
- Testing
- F-choline PET Scan, FDG-PET, biopsy
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
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
Cancer
Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors
This study aims to determine the efficacy of daily sirolimus and celecoxib, with low dose etoposide alternating with cyclophosphamide for pediatric participants with relapsed or refractory tumors.
- Trial name
- AflacST1502 (NCT02574728)
- Testing
- Sirolimus, Celecoxib, Etoposide
- Where
- 6 sites in United States
- Volunteers
- 46 people
- Sponsor
- Emory University
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
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
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Background: Niemann-Pick disease type C (NPC) is a genetic disorder that results in progressive loss of nervous system function by affecting the membranes of nerve cells. There is no known cure for NPC.
- Trial name
- NCT00975689
- Testing
- N-Acetyl Cysteine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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)
Advanced Tumors, Melanoma
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patient With Advanced Tumors
This is a First-in-Human, Open Label, Phase I/II Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patients with Advanced Tumors, composed of a Phase I dose escalation and dose expansion stage and a…
- Conditions
- Advanced Tumors, Melanoma, Non-Small-Cell Lung Cancer, Erdheim-Chester Disease
- Trial name
- NCT04198818
- Testing
- HH2710
- Where
- 3 sites in United States, China
- Volunteers
- 37 people
- Sponsor
- Haihe Biopharma Co., Ltd.
Rosai-Dorfman Disease, Langerhans Cell Histiocytosis
Central China Rosai-Dorfman Disease Registry
This is a research study among patients with Rosai-Dorfman disease.
- Trial name
- CCR-DDR (NCT05284942)
- Testing
- Mycophenolate mofetil combined with oral prednisone
- Where
- 2 sites in China
- Volunteers
- 20 people
- Sponsor
- Tongji Hospital
Acid Sphingomyelinase Deficiency
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632…
- Trial name
- NCT06949358
- Testing
- Olipudase alfa
- Where
- 2 sites in France
- Volunteers
- 3 people
- Sponsor
- Sanofi
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
Niemann-Pick Disease, Type C
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT03471143
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Washington University School of Medicine
Hemophagocytic Lymphohistiocytosis
L-DEP as an Initial Treatment for EBV-HLH
This study aimed to investigate the efficacy and safety of Pegaspargase together with liposomal doxorubicin, etoposide and high dose methylprednisolone (L-DEP) as an initial treatment for Epstein Barr virus-induced hemophagocytic…
- Trial name
- NCT02912702
- Testing
- Pegaspargase, doxorubicin hydrochloride liposome injection, etoposide
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Beijing Friendship Hospital