Clinical trials: Gaucher Disease, Type 2
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.
- Conditions
- Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00364858
- Testing
- Cerezyme
- Where
- 26 sites in United States, Brazil and 5 more
- Volunteers
- 95 people
- Sponsor
- Genzyme, a Sanofi Company
Neuroblastoma
Therapy for Children With Advanced Stage Neuroblastoma
Neuroblastoma is the most common extracranial solid tumor in childhood, with nearly 50% of patients presenting with widespread metastatic disease.
- Trial name
- NCT01857934
- Testing
- cyclophosphamide, topotecan, hu14.18K322A
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- St. Jude Children's Research Hospital
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
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
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
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
Gaucher Disease
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and post pubertal at Screening with a confirmed…
- Trial name
- NCT04145037
- Testing
- AVR-RD-02
- Where
- 5 sites in United States, Canada
- Volunteers
- 8 people
- Sponsor
- AVROBIO
SCLC
Safety and Clinical Activity of Nivatrotamab in Relapsed/Recurrent Metastatic Small-cell Lung Cancer
Adult patients with small-cell lung cancer (SCLC) will be treated with nivatrotamab a monoclonal anti GD2×CD3 bispecific antibody to investigate the safety and tolerability of the drug.
- Trial name
- NCT04750239
- Testing
- Nivatrotamab
- Where
- 7 sites in United States
- Volunteers
- 3 people
- Sponsor
- Y-mAbs Therapeutics
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
Gaucher Disease
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Primary Objective: Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies.
- Trial name
- EXOSKEL (NCT02536755)
- Testing
- Eliglustat, GZ385660
- Where
- 4 sites in Canada, Russia and 1 more
- Volunteers
- 31 people
- Sponsor
- Genzyme, a Sanofi Company
Brain and Central Nervous System Tumors, Intraocular Melanoma
Iodine I 131 Monoclonal Antibody 3F8 in Treating Patients With Central Nervous System Cancer or Leptomeningeal Cancer
RATIONALE: Radiolabeled monoclonal antibodies, such as iodine I 131 monoclonal antibody 3F8, can find tumor cells and carry tumor-killing substances to them without harming normal cells.
- Conditions
- Brain and Central Nervous System Tumors, Intraocular Melanoma, Lung Cancer, Melanoma (Skin)
- Trial name
- NCT00445965
- Testing
- DNA analysis, immunologic technique, pharmacological study
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neuroblastoma
Study of Donor Derived, Multi-virus-specific, Cytotoxic T-Lymphocytes for Relapsed/Refractory Neuroblastoma
This is a single-center, investigator-initiated, single-arm, pilot study of post-allogeneic transplant, adoptive immunotherapy for the treatment of patients with relapsed/refractory neuroblastoma expressing the mesenchymal tumor…
- Trial name
- STALLONe (NCT01460901)
- Testing
- GD2 CAR modified Tri-virus specific cytotoxic t-cells
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Mercy Hospital Kansas City
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
Anti-GD2 3F8 Monoclonal Antibody and GM-CSF for High-Risk Neuroblastoma
The purpose of this study is to be able to supply an experimental combination of drugs called 3F8 and GM-CSF (also called sargramostim) to patients with high-risk neuroblastoma who may benefit from treatment.
- Trial name
- NCT02100930
- Testing
- Anti-GD2 3F8 Monoclonal Antibody, GM-CSF (granulocyte-macrophage colony-stimulating factor), oral isotretinoin
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neuroblastoma, Osteosarcoma
Study of the Safety and Efficacy of Humanized 3F8 Bispecific Antibody (Hu3F8-BsAb) in Patients With Relapsed/Refractory Neuroblastoma, Osteosarcoma and Other Solid Tumor Cancers
The purpose of this study is to test the safety of a study drug called humanized 3F8 bispecific antibody (Hu3F8-BsAb).
- Conditions
- Neuroblastoma, Osteosarcoma, Other Solid Tumor Cancers
- Trial name
- NCT03860207
- Testing
- Humanized 3F8 Bispecific Antibody, Blood draw
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Y-mAbs Therapeutics
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
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.