Clinical trials: Gaucher Disease, Type 2
Solid Tumor
Study on GD2 Positive Solid Tumors by 4SCAR-GD2
Patients with refractory and/or recurrent solid tumor have poor prognosis despite complex multimodel therapy and therefore, novel approaches are urgently needed.
- Trial name
- NCT02992210
- Testing
- 4SCAR-GD2
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Gaucher Disease
SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
This project is expected to elucidate role of different therapeutic interventions: SRT in comparison to ERT in influencing immune aspects of GD pathology, as well as bone involvement.
- Trial name
- NCT02605603
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Recurrent Childhood Neuroblastoma, Recurrent Childhood Osteosarcoma
Testing a New Immune Cell Therapy, GD2-Targeted Modified T-cells (GD2CART), in Children, Adolescents, and Young Adults With Relapsed/Refractory Osteosarcoma and Neuroblastoma, GD2-CAR PERSIST Trial
This phase I trial investigates the side effects and determines the best dose of an immune cell therapy called GD2CART, as well as how well it works in treating patients with osteosarcoma or neuroblastoma that has come back…
- Conditions
- Recurrent Childhood Neuroblastoma, Recurrent Childhood Osteosarcoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma
- Trial name
- NCT04539366
- Testing
- Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration
- Where
- 6 sites in United States
- Volunteers
- 73 people
- Sponsor
- National Cancer Institute (NCI)
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
Diffuse Midline Glioma, H3 K27M-Mutant
CAR T Cells to Target GD2 for DMG
The CARMIGO Trial is a single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and young adults aged 2-16 years with Diffuse Midline Glioma…
- Trial name
- CARMIGO (NCT05544526)
- Testing
- GD2 CAR T cells
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University College, London
Neuroblastoma, Melanoma
A Phase I Trial Of The Humanized Anti-GD2 Antibody In Children And Adolescents With Neuroblastoma, Osteosarcoma, Ewing Sarcoma and Melanoma
Relapsed and/or refractory neuroblastoma, osteosarcoma, Ewing sarcoma and melanoma are considered difficult to treat and cure. For this study we are testing the use of a new experimental (investigational) antibody called…
- Conditions
- Neuroblastoma, Melanoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT00743496
- Testing
- Anti-GD2 antibody
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- St. Jude Children's Research Hospital
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
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
Malignant Disease
GD2/CD56 Bi-specific CAR-T Cell Therapy
The purpose of this clinical trial is to assess the feasibility, safety and efficacy of anti-GD2/CD56 bi-specific CAR-T cell therapy in patients with GD2 and/or CD56 positive cancer.
- Trial name
- NCT05437328
- Testing
- bi-4SCAR GD2/CD56 T cells
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
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
Advanced Unresectable, Metastatic
Biomarker-Guided Dual-Target CAR-T Cells for Advanced Solid Tumors
This is a multicenter, open-label, Phase 1/2 master protocol evaluating autologous dual-target CAR-T cell therapy in adults with advanced solid cancers.
- Trial name
- SELECT-2CAR (NCT07523529)
- Testing
- Autologous dual-target CAR-T cells selected from the predefined target library., Fludarabine, Cyclophosphamide
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Beijing Biotech
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 example 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…
- Conditions
- Small Cell Lung Cancer (SCLC), Relapsed/Refractory SCLC, Extensive-stage SCLC
- Trial name
- NCT07744256
- Testing
- EB-DART-NK01, Lymphodepleting chemotherapy
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Beijing Biotech
Gaucher Disease, Type 3
An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
The purpose of this study is to evaluate the efficacy and safety of autologous hemotopoietic stem cell (HSC) gene therapy, AVR-RD-02, compared to enzyme replacement therapy, for the treatment of Gaucher disease Type 3 in male and…
- Trial name
- NCT05815004
- Testing
- Gene therapy, Enzyme Replacement Agent
- Sponsor
- AVROBIO
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
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
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
Sarcoma, Osteosarcoma
A Phase I Trial of T Cells Expressing an Anti-GD2 Chimeric Antigen Receptor in Children and Young Adults With GD2+ Solid Tumors
Background GD2 is a well-characterized tumor antigen in neuroblastoma, which is also expressed on osteosarcomas and some other sarcomas.
- Conditions
- Sarcoma, Osteosarcoma, Neuroblastoma, Melanoma
- Trial name
- NCT02107963
- Testing
- Anti-GD2-CAR engineered T cells, AP1903, Cyclophosphamide
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Cancer Institute (NCI)
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
Melanoma (Skin), Neuroblastoma
Biological Therapy in Treating Children With Refractory or Recurrent Neuroblastoma or Other Tumors
RATIONALE: Biological therapies such as hu14.18-interleukin-2 fusion protein use different ways to stimulate the immune system and stop cancer cells from growing.
- Conditions
- Melanoma (Skin), Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00003750
- Testing
- hu14.18-IL2 fusion protein
- Where
- 59 sites in United States, Australia and 1 more
- Volunteers
- 28 people
- Sponsor
- Children's Oncology Group
Gaucher Disease, Type 2
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2).
- Trial name
- NCT04411654
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Prevail Therapeutics