Clinical trials: Gaucher Disease, Type 2
Neuroblastoma, Osteosarcoma
Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma
The body has different ways of fighting infections and disease. No single way seems perfect for fighting cancer. This research study combines two different ways of fighting disease: antibodies and T cells.
- Trial name
- NCT03721068
- Testing
- iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Gaucher Disease Type 3
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
- Trial name
- NCT07715084
- Testing
- AZ-3102
- Where
- 7 sites in India, Pakistan and 1 more
- Volunteers
- 20 people
- Sponsor
- Azafaros B.V.
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.
Melanoma
Combination Immunotherapy Targeting Melanoma
The purpose of this study is to assess the feasibility, safety and efficacy of combination immunotherapy based on CAR T cells, cytotoxic T lymphocytes (CTLs), and dendritic cell (DC) vaccines modified with GM-CSF and B7-2 (CD86)…
- Trial name
- NCT06739226
- Testing
- Antigen-specific CAR T, CTL and DCvac to treat melanoma.
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Neuroblastoma
Monoclonal Antibody Therapy in Treating Children With Metastatic Neuroblastoma in Second Remission
RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Trial name
- NCT00002458
- Testing
- monoclonal antibody 3F8
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neuroblastoma
A Study on the Efficacy of GD2-CAR T Cells in the Treatment of Neuroblastoma
Neuroblastoma (NB) is a malignant tumor of the sympathetic nervous system.Chemotherapy and autologous hematopoietic stem cell transplantation are the main treatments for neuroblastoma, and the prognosis of patients with high-risk…
- Trial name
- NCT06684639
- Testing
- GD2-CAR-T cell
- Where
- China
- Volunteers
- 30 people
- Sponsor
- The General Hospital of Western Theater Command
Gaucher's Disease Type I, Gaucher's Disease Type III
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Primary Objective: Evaluated the safety and pharmacokinetics of eliglustat in pediatric participants (≥2 to <18 years old). Secondary Objective: Evaluated the efficacy of eliglustat and quality of life in pediatric participants…
- Trial name
- ELIKIDS (NCT03485677)
- Testing
- Eliglustat GZ385660, Imiglucerase GZ437843
- Where
- 21 sites in Argentina, Canada and 8 more
- Volunteers
- 57 people
- Sponsor
- Sanofi
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
Neuroblastoma, Effects of Immunotherapy
Anti-GD2 4th Generation CART Cells Targeting Refractory and/or Recurrent Neuroblastoma
Patients with refractory and/or recurrent neuroblastoma have poor prognosis despite complex multimodel therapy and therefore, novel approaches are urgently needed.
- Trial name
- 4SCAR-GD2 (NCT02765243)
- Testing
- Anti-GD2 CART
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Zhujiang Hospital
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
Melanoma
Safety Study of Human IgM (MORAb-028) to Treat Metastatic Melanoma
This research is being done to find out the safety of MORAb-028 in subjects with metastatic melanoma.
- Trial name
- NCT01123304
- Testing
- MORAb028
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Morphotek
Gaucher Disease
A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase.
- Trial name
- NCT02053896
- Volunteers
- 5 people
- Sponsor
- ISU Abxis Co., Ltd.
Epithelial Ovarian Cancer, Epithelial Ovarian Cancer, Fallopian Tube or Peritoneum
Dual-Target CAR-NK Cells in Recurrent or Refractory Epithelial Ovarian Cancer
This study evaluates the safety, tolerability, and preliminary anti-tumor activity of EB-DUALNK, a dual-target chimeric antigen receptor natural killer (CAR-NK) cell therapy, in adults with recurrent or refractory epithelial…
- Trial name
- EB-DUALNK-OV (NCT07589543)
- Testing
- EB-DUALNK, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 42 people
- Sponsor
- Beijing Biotech
Lysosomal Storage Disorders, Gaucher Disease
Investigating Lysosomal Storage Diseases in Minority Groups
Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US.
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease
- Trial name
- NCT02120235
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Melanoma
Determine Toxicity and Antibody Responses With a KLH Conjugated Bivalent Vaccine Containing GD2 Lactone, GD3 Lactone With Immunological Adjuvant QS-DG or OPT-821 in Patients With Disease Free AJCC Stage III or IV Cutaneous Melanoma
Vaccines contain substances that help us make antibodies. Different antibodies help protect us against a variety of harmful things. GD2 and GD3 gangliosides are substances found on the surface of most melanoma cells.
- Trial name
- NCT00597272
- Testing
- KLH conjugates with GD2L and GD3L
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
Glioma of Spinal Cord, Glioma of Brainstem
GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas (DIPG) & Spinal Diffuse Midline Glioma(DMG)
The primary purpose of this study is to test whether CAR T cells targeting GD2 (GD2CART) can be successfully made and safely given to children and adults with H3K27M-mutant diffuse midline glioma (DMG).
- Trial name
- NCT04196413
- Testing
- GD2 CAR T cells, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 97 people
- Sponsor
- Stanford University
SCLC, Malignant Melanoma
GD2-SADA:177Lu-DOTA Complex in Patients With Solid Tumors Known to Express GD2
Patients with Small Cell Lung Cancer, High Risk Neuroblastoma, Sarcoma and Malignant Melanoma will be treated with GD2-SADA:177Lu-DOTA complex(The IMP is a two-step radioimmunotherapy, delivered as two separate products GD2-SADA…
- Conditions
- SCLC, Malignant Melanoma, Sarcoma, High Risk Neuroblastoma
- Trial name
- NCT05130255
- Testing
- GD2-SADA (0.3 mg/kg) and 177Lu-DOTA (30 mCi), GD2-SADA (3 mg/kg) 177Lu-DOTA (30 mCi), GD2-SADA (0.3 mg/kg) 177Lu-DOTA (200 mCi)
- Where
- 8 sites in United States
- Volunteers
- 23 people
- Sponsor
- Y-mAbs Therapeutics