Clinical trials: Langerhans Cell Histiocytosis
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
Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis
A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back…
- Trial name
- NCT05828069
- Testing
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy
- Where
- 123 sites in United States, Australia and 1 more
- Volunteers
- 48 people
- Sponsor
- National Cancer Institute (NCI)
Hemophagocytic Lymphohistiocytosis (HLH)
Treatment of Familiar Lymphohistiocytosis
The purpose of this project is to study the number of surviving patients until hematopoietic stem cell transplantation (HSCT) after first line treatment of hemophagocytic lymphohistiocytosis (HLH) by Alemtuzumab
- Trial name
- C-HLH (NCT02472054)
- Testing
- Alemtuzumab, Methyl Prednisolone (MP), Cyclosporin A (CSA)
- Where
- France
- Volunteers
- 29 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Haematological Malignancy, Malignant Neoplasm
DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers.
This clinical trial is looking at two drugs called dabrafenib and trametinib. Dabrafenib and trametinib are approved as standard of care treatment for adult patients with melanoma (a type of skin cancer) or lung cancer and in…
- Conditions
- Haematological Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders, Neoplasms by Histologic Type
- Trial name
- DETERMINE (NCT07440290)
- Testing
- Dabrafenib, Trametinib
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Niemann-Pick Diseases
Induced Pluripotent Stem Cells for Niemann Pick Disease
Establishment of individualized human cellular disease models based on induced pluripotent stem cells that reflect the broad heterogeneous phenotypic spectrum of Niemann Pick disease
- Trial name
- IPSNPABC (NCT03883750)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Histiocytosis
Determination of Molecular Status, the Efficacy and Safety of Fluorodeoxyglucose in PET-CT Imaging
Prospective, low intervention, open, single-center, non-commercial clinical trial to improve diagnostics in patients with histiocytosis by assessing the molecular profile of the tumor tissues, monitoring its presence in…
- Trial name
- HISTIOGEN (NCT04943211)
- Testing
- fluorodeoxyglucose (18F-FDG)
- Where
- Poland
- Volunteers
- 150 people
- Sponsor
- Anna Raciborska
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.
Erdheim-Chester Disease
Subtypes and Prognostic Factors in Erdheim-Chester Disease
Erdheim-Chester Disease (ECD) is a rare form of histiocytosis characterized by the proliferation of blood cells, known as histiocytes, which infiltrate various organs and tissues, often causing irreversible damage.
- Trial name
- NCT06317246
- Testing
- Investigation of BRAF mosaicism
- Where
- 2 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Meyer Children's Hospital IRCCS
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
Niemann-Pick Disease, Type C
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
- Trial name
- NCT03759639
- Testing
- IB1001
- Where
- 9 sites in United States, Germany and 3 more
- Volunteers
- 33 people
- Sponsor
- IntraBio Inc
Acid Sphingomyelinase Deficiency (ASMD)
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
Primary Objective: To describe the lung, spleen and liver outcomes of olipudase alfa Secondary Objectives: To describe the patient's characteristics To describe conditions of olipudase alfa use To describe safety data related to…
- Trial name
- OPERA (NCT05359276)
- Testing
- Olipudase alfa
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Sanofi
Histiocytosis
Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory Histiocytosis
Prospective, interventional, open, randomized, single-center, non-commercial clinical trial to optimize treatment and dosage of vemurafenib in juvenile patients with histiocytosis resistant to conventional therapy and in whom the…
- Trial name
- BRAVO (NCT04943198)
- Testing
- Vemurafenib
- Where
- Poland
- Volunteers
- 25 people
- Sponsor
- Anna Raciborska
Hemophagocytic Lymphohistiocytosis (HLH)
Dose-modified Emapalumab and Ruxolitinib (E-Ru) Regimens for Hemophagocytic Lymphohistiocytosis
This study is trying to evaluate the efficacy and safety of dose-modified Emapalumab and Ruxolitinib (E-Ru) regimens for the treatment of active hemophagocytic lymphohistiocytosis.
- Trial name
- NCT06951971
- Testing
- dose-modified E-Ru, Salvage treatment and follow-up
- Where
- China
- Volunteers
- 30 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Hemophagocytic Syndrome
Children and Adult Hemophagocytic Syndrome (HLHa)
Different study of HLHa patients : Diagnosis criteria, because criteria are based on pediatric genetic studies. Physiopathological studies: genetic studies have demonstrated the role of CD8+ cells, in particular because they have…
- Trial name
- HLH-genes (NCT02113917)
- Testing
- Identification of biological markers
- Where
- France
- Volunteers
- 204 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
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
Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma
T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts
Subjects are being asked to participate in this study because treatment of their disease requires them to receive a stem cell transplant.
- Conditions
- Acute Lymphoblastic Leukemia, Non Hodgkins Lymphoma, Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Trial name
- MOHEL (NCT00368355)
- Testing
- Ara-C, Cyclophosphamide, Campath-1H
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Baylor College of Medicine