Clinical trials: Gaucher Disease, Type 2
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.
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia…
- Trial name
- NCT05447494
- Testing
- Low-dose CAN103, High-dose CAN103
- Where
- China
- Volunteers
- 40 people
- Sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.
Brain Tumor, Pediatric, Medulloblastoma, Childhood
GD2-CAR T Cells for Pediatric Brain Tumours
The purpose of this study is to test the safety and efficacy of iC9-GD2-CAR T-cells, a third generation (4.1BB-CD28) CAR T cell treatment targeting GD2 in paediatric or young adult patients affected by relapsed/refractory…
- Conditions
- Brain Tumor, Pediatric, Medulloblastoma, Childhood, Embryonal Tumor, High Grade Glioma
- Trial name
- NCT05298995
- Testing
- GD2-CART01 (iC9-GD2-CAR T-cells)
- Where
- Italy
- Volunteers
- 54 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
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
Neuroblastoma
3RD GENERATION GD2 SPECIFIC CHIMERIC ANTIGEN RECEPTOR TRANSDUCED AUTOLOGOUS NATURAL KILLER T-CELLS FOR NEUROBLASTOMA
This research study is for patients that have a cancer called Neuroblastoma that has either come back after treatment or did not respond to the standard medicines used to treat it.
- Trial name
- GINAKIT (NCT02439788)
- Testing
- cyclophosphamide, Fludarabine, GINAKIT Cells
- Where
- United States
- Sponsor
- Baylor College of Medicine
Type 2 Gaucher Disease
Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease
This study will use an experimental method of delivering the enzyme glucocerebrosidase directly into the brain of a patient with Gaucher disease to treat disease symptoms.
- Trial name
- NCT00244582
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Recurrent Neuroblastoma, Refractory Neuroblastoma
Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma
This phase I trial studies the side effects and best dose of lenalidomide when given together with dinutuximab with or without isotretinoin in treating younger patients with neuroblastoma that does not respond to treatment or…
- Trial name
- NCT01711554
- Testing
- Dinutuximab, Isotretinoin, Laboratory Biomarker Analysis
- Where
- 15 sites in United States, Canada
- Volunteers
- 27 people
- Sponsor
- National Cancer Institute (NCI)
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
Sarcoma
Trivalent Ganglioside Vaccine With Immunological Adjuvant or Immunological Adjuvant Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free
Sarcoma patients are at high risk for their cancer to recur even when the sarcoma has been removed surgically or treated with radiation or chemotherapy.
- Trial name
- NCT01141491
- Testing
- Trivalent ganglioside vaccine, OPT-821
- Where
- 13 sites in United States
- Volunteers
- 136 people
- Sponsor
- MabVax Therapeutics, Inc.
Relapsed Neuroblastoma, Refractory Neuroblastoma
C7R-GD2.CART Cells for Patients With Relapsed or Refractory Neuroblastoma and Other GD2 Positive Cancers (GAIL-N)
This study is for patients with neuroblastoma, sarcoma, uveal melanoma, breast cancer, or another cancer that expresses a substance on the cancer cells called GD2.
- Conditions
- Relapsed Neuroblastoma, Refractory Neuroblastoma, Relapsed Osteosarcoma, Relapsed Ewing Sarcoma
- Trial name
- NCT03635632
- Testing
- C7R-GD2.CART cells
- Where
- 2 sites in United States
- Volunteers
- 94 people
- Sponsor
- Baylor College of Medicine
Herpes Simplex
Safety Study of Herpes Simplex Vaccine in HSV Seronegative and Seropositive Females Between 10 and 17 Years Old
Main goal of this study is to compare the occurrence of serious adverse events (SAEs) between the herpes simplex (gD2-AS04) vaccine group and the Saline control group throughout the study period (up to month 12).
- Trial name
- NCT00224484
- Testing
- GSK208141, Havrix (investigational formulation)
- Where
- 151 sites in United States, Australia and 16 more
- Volunteers
- 5,960 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
B Cell Lymphoma (BCL)
Multi-CAR-T Cells Targeting B Cell Lymphomas
This study aims to evaluate safety and efficacy of a combination of 4th generation chimeric antigen receptor gene-modified T cells targeting B cell surface molecules including CD19 and alternative CARTs as booster and…
- Trial name
- NCT04429438
- Testing
- 4SCAR19 and 4SCAR20/22/70/PSMA/13/79b/GD2
- Where
- China
- Volunteers
- 11 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Neuroblastoma
Multi-modular Chimeric Antigen Receptor Targeting GD2 in Neuroblastoma
MAGNETO is a single-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children and teenagers aged 1-16 years with relapsed or refractory neuroblastoma.
- Trial name
- MAGNETO (NCT05990751)
- Testing
- GD2 CAR T cells
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University College, London
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 (NBL), Ewing Sarcoma
GD2IL18CART Combined With Bevacizumab for Relapsed and Refractory GD2-positive Solid Tumors
This study will test a chimeric antigen receptor (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
Cancer Disease
GD2/CD70 Bi-specific CAR-T Cell Therapy
The purpose of this study is to assess the feasibility, safety and efficacy of GD2/CD70 bi-specific CAR-T cell therapy in patients with GD2 and/or CD70 positive tumor.
- Trial name
- NCT05438368
- Testing
- bi-4SCAR GD2/CD70 T cells
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Neuroblastoma
Monoclonal Antibody 3F8 and Sargramostim in Treating Patients With Neuroblastoma
RATIONALE: Monoclonal antibodies, such as monoclonal antibody 3F8, can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Trial name
- NCT00072358
- Testing
- anti-GD2 murine IgG3 monoclonal antibody 3F8
- Where
- United States
- Volunteers
- 291 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neuroblastoma
GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma
This research study combines two different ways of fighting cancer: antibodies and Natural Killer T cells (NKT). Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer.
- Trial name
- GINAKIT2 (NCT03294954)
- Testing
- GINAKIT Cells, GINAKIT cells + Etanercept
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Baylor College of Medicine
Neuroblastoma
Humanized 3F8 Monoclonal Antibody (Hu3F8) in Patients With High-Risk Neuroblastoma and GD2-Positive Tumors
The purpose of this study is to find out if "humanized 3F8" (Hu3F8) is safe for treating neuroblastoma and other cancers. A phase 1 study means the investigators are trying to find out what side effects happen when higher and…
- Trial name
- NCT01419834
- Testing
- Humanized 3F8 Monoclonal Antibody (Hu3F8)
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Memorial Sloan Kettering Cancer Center