Clinical trials: Histiocytosis
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Lymphoma, Non-Hodgkin, HIV Infections
A Study of Azidothymidine (AZT) in the Treatment of HIV Infection in Patients Receiving a Bone Marrow Transplant
To determine whether zidovudine (AZT) in conjunction with bone marrow transplantation prevents the reinfection of donor hematopoietic/lymphoid cells in patients with positive HTLV III antibody and large cell/diffuse histiocytic…
- Trial name
- NCT00002284
- Testing
- Zidovudine
- Where
- United States
- Sponsor
- Glaxo Wellcome
Lymphoma, B-Cell, Small Lymphocytic Lymphoma
A Study of Nemtabrutinib (MK-1026) in Participants With Relapsed or Refractory Hematologic Malignancies (ARQ 531-101/MK-1026-001)
This study aims to evaluate the safety, tolerability, pharmacodynamic, and pharmacokinetic (PK) of nemtabrutinib (formerly ARQ 531) tablets in selected participants with relapsed or refractory hematologic malignancies.
- Conditions
- Lymphoma, B-Cell, Small Lymphocytic Lymphoma, Chronic Lymphocytic Leukemia, Waldenstrom Macroglobulinemia
- Trial name
- NCT03162536
- Testing
- Nemtabrutinib
- Where
- 10 sites in United States
- Volunteers
- 190 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
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
Diffuse Large B-cell Lymphoma
Biocell Natural Killer Mixture in Diffuse Large B Cell Lymphoma (DLBCL) Patients
Primary objective: To compare the event free survival for 3years of R-CHOP plus Biocell Natural Killer Cell to R-CHOP therapy with DLBCL patients.
- Trial name
- NKCell (NCT00846157)
- Testing
- NKM injection
- Where
- South Korea
- Volunteers
- 276 people
- Sponsor
- NKBio Co.Ltd.
Leukemia, Lymphoma
Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Cord Blood Transplant for Hematologic Malignancy
Ex vivo expanded human myeloid progenitor cells (hMPCs; CLT-008) have the potential to accelerate neutrophil recovery in patients receiving myeloablative conditioning as part of an umbilical cord blood transplant for hematologic…
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Plasma Cell Neoplasm
- Trial name
- NCT00891137
- Testing
- human myeloid progenitor cells
- Where
- 8 sites in United States
- Volunteers
- 30 people
- Sponsor
- Cellerant Therapeutics
B Cell Lymphoma, NHL, Adult
SynKIR-310 for Relapsed/Refractory B-NHL
This first-in-human (FIH) trial is designed to assess the safety, feasibility and preliminary efficacy of a single intravenous (IV) dose of SynKIR-310 administered to participants with relapsed/refractory B-NHL.
- Conditions
- B Cell Lymphoma, NHL, Adult, Mantle Cell Lymphoma, Relapsed Non-Hodgkin Lymphoma
- Trial name
- NCT06544265
- Testing
- SynKIR-310
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Verismo Therapeutics
EBV-induced Lymphomas, EBV-associated Malignancies
Biological Therapy in Treating Patients at High-Risk or With Lymphoma, Lymphoproliferative Disease, or Malignancies
The purpose of this phase I/II trial is to study the side effects and best dose of biological therapy to treat patients at high-risk or with Epstein-Barr virus-associated lymphoma or lymphoproliferative disease.
- Conditions
- EBV-induced Lymphomas, EBV-associated Malignancies, Transplant Patients With EBV Viremia at High Risk of Developing a Recurrent EBV Lymphoma
- Trial name
- NCT00002663
- Testing
- Epstein-Barr virus-specific cytotoxic T lymphocytes (EBV-CTLs)
- Where
- United States
- Volunteers
- 58 people
- Sponsor
- Atara Biotherapeutics
Niemann-Pick Type C Disease
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.
- Trial name
- NCT03643562
- Testing
- Adrabetadex
- Where
- 17 sites in United States
- Volunteers
- 27 people
- Sponsor
- Mandos LLC
Farber's Lipogranulomatosis, Ceramidase Deficiency
Biomarker for Farber Disease (BioFarber)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Farber disease from the blood
- Conditions
- Farber's Lipogranulomatosis, Ceramidase Deficiency, Hepatomegaly, Splenomegaly
- Trial name
- BioFarber (NCT02298634)
- Where
- 3 sites in Egypt, Germany and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma
MORAb-004 in Treating Young Patients With Recurrent or Refractory Solid Tumors or Lymphoma
This phase I trial studies the side effects and best dose of MORAb-004 in treating young patients with recurrent or refractory solid tumors or lymphoma.
- Conditions
- Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Childhood Burkitt Lymphoma
- Trial name
- NCT01748721
- Testing
- anti-endosialin/TEM1 monoclonal antibody MORAb-004, pharmacological study, laboratory biomarker analysis
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Morphotek
Diffuse Large B Cell Lymphoma, Large B-cell Lymphoma
Long-term Follow-up Study of Lentiviral-based Gene-edited Immune Cell Therapy
According to health authorities guidances (FDA 2006, EMA(European Medicines Agency) 2009) for gene therapy clinical trials, observing subjects for delayed adverse events for 15 years is recommended.
- Conditions
- Diffuse Large B Cell Lymphoma, Large B-cell Lymphoma, Primary Mediastinal Large B Cell Lymphoma, Follicular Lymphoma Grade 3A
- Trial name
- NCT05377307
- Testing
- Pell's lentiviral-based gene-edited immune cell therapy
- Where
- 5 sites in Taiwan
- Volunteers
- 49 people
- Sponsor
- Pell Bio-Med Technology Co., Ltd.
Niemann-Pick Disease, Type C
Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with…
- Trial name
- NCT03687476
- Testing
- VTS-270
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
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
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
This study aims to evaluate safety information in patients with HLH/MAS in Still's disease exposed to Gamifant.
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS), Still Disease
- Trial name
- NCT07909005
- Testing
- Local laboratory testing, Local Lab Tests
- Where
- 18 sites in United States
- Volunteers
- 70 people
- Sponsor
- Swedish Orphan Biovitrum
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.
DLBCL - Diffuse Large B Cell Lymphoma, CAR T Cell Therapy
DALY II Japan/MB-CART2019.1 for DLBCL
DALY II Japan is a phase II, multi-center, single arm study to evaluate the efficacy, safety, and pharmacokinetics of zamtocabtagene autoleucel (MB-CART2019.1) in patients with relapsed and/or refractory diffuse large B cell…
- Trial name
- DALY II Japan (NCT07288879)
- Testing
- MB-CART2019.1
- Where
- 5 sites in Japan
- Volunteers
- 31 people
- Sponsor
- Miltenyi Biomedicine GmbH
Brain and Central Nervous System Tumors, Lymphoma
DX-8951f in Treating Children With Advanced Solid Tumors or Lymphomas
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of DX-8951f in treating children who have advanced solid…
- Conditions
- Brain and Central Nervous System Tumors, Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00004212
- Testing
- filgrastim, exatecan mesylate
- Where
- 4 sites in United States
- Sponsor
- Daiichi Sankyo
Brain and Central Nervous System Tumors, Leukemia
Chemotherapy in Treating Young Patients With Recurrent or Refractory Meningeal Leukemia, Lymphoma, or Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of liposomal cytarabine in treating young patients who have…
- Conditions
- Brain and Central Nervous System Tumors, Leukemia, Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00003073
- Testing
- liposomal cytarabine
- Where
- 6 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Pacira Pharmaceuticals, Inc
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.