Clinical trials: Histiocytosis
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
Niemann-Pick Disease, Type C1
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 2 different dose levels and what effects it has on people who…
- Trial name
- NCT02939547
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Cyclo Therapeutics, Inc.
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
Septic Shock, Acute Respiratory Distress Syndrome
CytOSorb TreatMent Of Critically Ill PatientS Registry
Registry intended to provide a data repository and reporting infrastructure for the surveillance of CytoSorb device use in real-world critical care settings, and to serve as an objective, comprehensive, and scientifically-based…
- Conditions
- Septic Shock, Acute Respiratory Distress Syndrome, Trauma, Rhabdomyolysis
- Trial name
- COSMOS (NCT05146336)
- Testing
- CytoSorb
- Where
- 28 sites in Austria, Germany and 4 more
- Volunteers
- 3,000 people
- Sponsor
- CytoSorbents, Inc
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 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
Melanoma, Thyroid Cancer
Phase I/IIa Study to Evaluate the Safety, PK, PD, and Preliminary Efficacy of PLX8394 in Patients With Advanced Cancers.
The study objective is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered PLX8394 in patients with advanced solid tumors.
- Conditions
- Melanoma, Thyroid Cancer, Colorectal Cancer, Non-small Cell Lung Cancer
- Trial name
- NCT02012231
- Testing
- PLX8394
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Fore Biotherapeutics
Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Lymphoma Non-Hodgkin, Agressive Lymphoma
A Study of CC-97540, CD19-targeted NEX-T Chimeric Antigen Receptor (CAR) T Cells, in Subjects With Relapsed or Refractory B-cell Non-Hodgkin Lymphoma
This is a Phase 1, first-in-human, open-label, multicenter study of CC-97540, CD19-targeted NEX-T chimeric antigen receptor (CAR) T cells, in subjects with relapsed or refractory B-cell non-Hodgkin lymphoma.
- Conditions
- Lymphoma Non-Hodgkin, Agressive Lymphoma, Diffuse-large B-cell Lymphoma (DLBCL)
- Trial name
- NCT04231747
- Testing
- CC-97540
- Where
- 14 sites in United States, Canada
- Volunteers
- 24 people
- Sponsor
- Juno Therapeutics, a Subsidiary of Celgene
Niemann-Pick Disease, Type C
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844.
- Trial name
- NCT04958642
- Testing
- Adrabetadex
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 66 people
- Sponsor
- Mandos LLC
Sphingomyelin Lipidosis
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Primary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult…
- Trial name
- ASCEND (NCT02004691)
- Testing
- Olipudase alfa
- Where
- 24 sites in United States, Argentina and 15 more
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
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 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
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
Leukemia, Lymphoma
BL22 Immunotoxin In Treating Young Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Non-Hodgkin's Lymphoma
RATIONALE: BL22 immunotoxin can locate tumor cells and kill them without harming normal cells. BL22 immunotoxin may be effective in treating relapsed or refractory acute lymphoblastic leukemia and non-Hodgkin's lymphoma.
- Trial name
- NCT00077493
- Testing
- BL22 immunotoxin, antibody-drug conjugate therapy, immunotoxin therapy
- Where
- United States
- Volunteers
- 95 people
- Sponsor
- MedImmune LLC
Niemann-Pick Disease, Type C
Early Access Program With Arimoclomol in US Patients With NPC
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy.
- Trial name
- NCT04316637
- Testing
- Arimoclomol
- Where
- 13 sites in United States
- Sponsor
- ZevraDenmark
Hemophagocytic Syndrome
Evaluate the Efficacy and Safety of PEG-rhG-CSF in Patients With Hemophagocytic Syndrome
Hemophagocytic syndrome (HPS), also known as hemophagocytic lymphohistiocytosis (HLH), is an immune mediated life-threatening disease. There is no uniform recommendation for salvage treatment of HLH.
- Trial name
- NCT04500886
- Testing
- PEG-rhG-CSF, rhG-CSF
- Where
- China
- Volunteers
- 50 people
- Sponsor
- CSPC Baike (Shandong) Biopharmaceutical Co., Ltd.