Clinical trials: Gaucher Disease, Type 2
Refractory Neuroblastoma, Soft Tissue Cancer
A Study Of Naxitamab , Granulocyte Macrophage Colony Stimulating Factor For Patients With Relapsed /Refractory , Soft Tissue or Anti GD2 Immunotherapy Refractory Neuroblastoma
The purpose of this study is to evaluate safety and efficacy of naxitamab, granulocyte macrophage Colony Stimulating Factor (GM CSF) and Isofosfamide/Carboplatin/Etoposide (NICE) for Patients With Relapsed /Refractory, soft…
- Trial name
- NICE (NCT06438614)
- Testing
- HITS, Naxitamab + GM-CSF cycles, NICE
- Where
- Spain
- Volunteers
- 47 people
- Sponsor
- Fundació Sant Joan de Déu
Neuroblastoma, GD2 Antibody (Naxitamab)
Adding Of Naxitamb In Induction Therapy For High Risk Neuroblastoma
To explore the efficacy of adding anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy for newly diagnosed high risk neuroblastoma patients.
- Trial name
- NCT06574698
- Testing
- Addition of anti-GD2 immunotherapy (Naxitamab) at the stage of induction chemotherapy
- Where
- China
- Volunteers
- 64 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Relapsed or Refractory Neuroblastoma
A Phase I Trial of Anti-GD2 T-cells (1RG-CART)
The purpose of this first in human study is to determine the safety and feasibility of 1RG-CART therapy in patients with relapsed or refractory neuroblastoma.
- Trial name
- NCT02761915
- Testing
- Leukapheresis, Cyclophosphamide, Fludarabine
- Where
- United Kingdom
- Volunteers
- 17 people
- Sponsor
- Cancer Research UK
Melanoma (Skin)
Monoclonal Antibody Therapy in Treating Patients With Stage III or Stage IV Melanoma
RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Trial name
- NCT00004184
- Testing
- monoclonal antibody 4B5 anti-idiotype vaccine, sargramostim, alum adjuvant
- Volunteers
- 50 people
- Sponsor
- University of Alabama at Birmingham
Small Cell Lung Cancer (SCLC), Relapsed/Refractory SCLC
Adaptive Phase 1/2 Study of Dual-Target CAR-NK Cells in Relapsed/Refractory Small Cell Lung Cancer (SCLC)
This study is an open-label, multi-center, adaptive Phase 1/2 trial evaluating the safety, feasibility, and preliminary antitumor activity of allogeneic dual-target CAR-NK cell products in adults with relapsed or refractory small…
- Conditions
- Small Cell Lung Cancer (SCLC), Relapsed/Refractory SCLC, SCLC, Extensive Stage
- Trial name
- DART-NK-SCLC (NCT07480213)
- Testing
- EB-DART-NK01 (DLL3/CD56 CAR-NK cells), Lymphodepleting chemotherapy
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Beijing Biotech
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase.
- Conditions
- Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00358150
- Testing
- Eliglustat tartrate
- Where
- 17 sites in United States, Argentina and 4 more
- Volunteers
- 26 people
- Sponsor
- Genzyme, a Sanofi Company
High-Risk Neuroblastoma
Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5
This clinical trial will evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly-diagnosed patients with high-risk neuroblastoma. We aim to recruit 10 patients over the next 2 years.
- Trial name
- NCT07537400
- Testing
- Naxitamab
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Shaare Zedek Medical Center
Diffuse Intrinsic Pontine Glioma, High Grade Glioma
C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B)
In this study, there are two treatment groups called Cohort 1 and Cohort 2. Cohort 1 is for patients with diffuse midline glioma, diffuse intrinsic pontine glioma, medulloblastoma, or another rare high-grade glioma that expresses…
- Conditions
- Diffuse Intrinsic Pontine Glioma, High Grade Glioma, Embryonal Tumor, Ependymal Tumor
- Trial name
- NCT04099797
- Testing
- C7R-GD2.CART cells (IV and ICV infusion)
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Baylor College of Medicine
Neuroblastoma, Sarcoma
Monoclonal Antibody A1G4 Plus BCG in Treating Patients With Cancer
RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Combining monoclonal antibody A1G4 with BCG may kill more tumor cells.
- Trial name
- NCT00003023
- Testing
- BCG vaccine, monoclonal antibody A1G4 anti-idiotype vaccine
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Bone Sarcoma, Embryonal Rhabdomyosarcoma
Chemo-immunotherapy in Patients Under 18 Years of Age With Bone and Soft Tissue Sarcomas
Data from 40 patients are planned to be included in the study to randomize approximately 10 patients. This study involves patients with advanced/metastatic GD2-positive rhabdomyosarcoma after progression on 1st line of…
- Conditions
- Bone Sarcoma, Embryonal Rhabdomyosarcoma, Alveolar Rhabdomyosarcoma
- Trial name
- FollowTheDIN (NCT06669013)
- Testing
- dinutuximab beta
- Where
- Russia
- Volunteers
- 40 people
- Sponsor
- N.N. Petrov National Medical Research Center of Oncology
GM1 Gangliosidosis, GM2 Gangliosidosis
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2).
- Conditions
- GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease
- Trial name
- RETRIEVE (NCT04470713)
- Where
- 17 sites in United States, Belgium and 8 more
- Volunteers
- 226 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Neuroblastoma
Bivalent Vaccine With Escalating Doses of the Immunological Adjuvant OPT-821, in Combination With Oral β-glucan for High-Risk Neuroblastoma
In the first part of this study we found the highest dose of the vaccine that did not have too many side effects. We are now trying to find out what effects the vaccine has when given at the same dose to all patients.
- Trial name
- NCT00911560
- Testing
- adjuvant OPT-821 in a vaccine containing two antigens (GD2L and GD3L) covalently linked to KLH, oral β-glucan
- Where
- United States
- Volunteers
- 374 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Gaucher's Disease
A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth
A study to assess the absorption of eliglustat through the mouth in healthy subjects and the safety of any systemic exposure resulting from oral surface absorption of eliglustat in healthy subjects.
- Trial name
- Acronym (NCT06193304)
- Testing
- Eliglustat
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Sanofi
Neuroblastoma, Bone Marrow, Sympathetic Nervous System
Anti-GD2 3F8 Antibody and Allogeneic Natural Killer Cells for High-Risk Neuroblastoma
Funding Source - FDA OOPD FDR004128 The goal of this study is to see if it is safe and feasible to give chemotherapy, natural killer (NK) cells, and an antibody called 3F8.
- Trial name
- NCT00877110
- Testing
- cyclophosphamide, vincristine, topotecan ,allogeneic NK cells & 3F8
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Glioma of Brain, CAR-T Cell Immunotherapy
CAR-T Cell Immunotherapy for GD2 Positive Glioma Patients
The purpose of this study is to evaluate the safety and effectiveness of CAR-T cell immunotherapy in treating with GD2 positive glioma patients.
- Trial name
- NCT04406610
- Testing
- GD2 CAR-T immunotherapy
- Sponsor
- Fuda Cancer Hospital, Guangzhou
Neuroblastoma
NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial
This is a phase II study looking at patient response to treatment with the combination dinutuximab, temozolomide, irinotecan, and GM-CSF.
- Trial name
- NCT06450041
- Testing
- Universal Donor (UD) TGFβi NK Cells, Temozolomide, Irinotecan
- Where
- 13 sites in United States
- Volunteers
- 62 people
- Sponsor
- New Approaches to Neuroblastoma Therapy Consortium
Neuroblastoma (NBL), Ewing Sarcoma
GD2IL18CART Combined With Bevacizumab for Relapsed and Refractory GD2-positive Solid Tumors
This study will test a CAR T-cell treatment combined with bevacizumab in children and young adults with relapsed or refractory neuroblastoma, Ewing sarcoma, or osteosarcoma.
- Conditions
- Neuroblastoma (NBL), Ewing Sarcoma, Osteosarcoma
- Trial name
- GD2CART-BEV (NCT07897968)
- Testing
- Chimeric Antigen Receptor (CAR) Therapy Infusion, Bevacizumab (Avastin)
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Universität Münster
Advanced Solid Tumor
Anti-GD2 ADC M3554 in Advanced Solid Tumors
The purpose of this study is to establish the recommended doses and further evaluate the safety and preliminary antitumor activity of M3554 in participants with soft tissue sarcoma (STS) and glioblastoma, IDH-wildtype.
- Trial name
- NCT06641908
- Testing
- M3554
- Where
- 11 sites in United States, Belgium and 4 more
- Volunteers
- 65 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
MPS I, MPS II
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses…
- Conditions
- MPS I, MPS II, MPS IVA, MPS VI
- Trial name
- PEARL (NCT04532047)
- Testing
- Aldurazyme (laronidase)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, San Francisco
Gaucher Disease, Type 1
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00430625
- Testing
- VPRIV ®,
- Where
- 5 sites in Argentina, Israel and 3 more
- Volunteers
- 25 people
- Sponsor
- Shire