Clinical trials: Gaucher Disease, Type 2
Gaucher Disease, Acid SphingoMyelinase Deficiency
PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and oncohematology clinics, and adult internal medicine and hematology.
- Conditions
- Gaucher Disease, Acid SphingoMyelinase Deficiency, ASMD, Niemann-Pick Diseases
- Trial name
- PREDIGA-2 (NCT05641103)
- Testing
- Patient with splenomegaly or splenectomy
- Where
- 14 sites in Spain
- Volunteers
- 122 people
- Sponsor
- Fundación Española de Hematología y Hemoterapía
Unresectable Melanoma, Metastatic Cutaneous Melanoma
Dual-Target CSPG4/GD2 CAR-NK Cells for Advanced Melanoma
This is a first-in-human, open-label, multicenter phase 1/2 study evaluating the safety, feasibility, recommended phase 2 dose (RP2D), and preliminary antitumor activity of allogeneic dual-target CSPG4/GD2 CAR-NK cells…
- Conditions
- Unresectable Melanoma, Metastatic Cutaneous Melanoma, Metastatic Uveal Melanoma
- Trial name
- DUET-MEL (NCT07627698)
- Testing
- EB-DTKN-401 allogeneic dual-target CSPG4/GD2 CAR-NK cells, Fludarabine, Cyclophosphamide
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Beijing Biotech
Brain and Central Nervous System Tumors, Intraocular Melanoma
Monoclonal Antibody Therapy in Treating Patients With Leptomeningeal Cancer
RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells.
- Conditions
- Brain and Central Nervous System Tumors, Intraocular Melanoma, Lung Cancer, Melanoma (Skin)
- Trial name
- NCT00003022
- Testing
- iodine I 131 monoclonal antibody 3F8
- Where
- United States
- 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
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.
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
Metabolic Disease, Fabry Disease
Getting Global Rare Disease Insights Through Technology Study
This project is a randomized controlled trial to use a mobile health journal, called Zamplo (formerly known as MyHealthJournal or ZoeInsights), to record patient reported outcomes (PROM) in patients with metabolic disorders.
- Conditions
- Metabolic Disease, Fabry Disease, Gaucher Disease, Pompe Disease
- Trial name
- GRIT (NCT04758130)
- Testing
- Zamplo Digital Health Platform
- Where
- Canada
- Volunteers
- 150 people
- Sponsor
- M.A.G.I.C. Clinic LTD
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
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
Melanoma
Vaccine Enriched, Autologous, Activated T-Cells Directed to Tumor in Patients With Relapsed/Refractory Melanoma
The researchers will investigate if modified T-cells from a patients own system can be utilized to find and destroy metastatic melanoma tumor and thus improve patient outcomes.
- Trial name
- MARVSmALo (NCT02482532)
- Testing
- tvs-CTL Vaccine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Gary Doolittle
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
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
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
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
Gaucher Disease
A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers
Primary Objective: To compare the pharmacokinetics of Abcertin to the reference product, EU-sourced Cerezyme, after single intravenous administration of 60 IU/kg.
- Trial name
- NCT04787887
- Testing
- Abcertin, EU-sourced Cerezyme
- Where
- Australia
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- ISU Abxis Co., Ltd.
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 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
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
Type I Gaucher Disease
Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.
- Trial name
- NCT01463215
- Testing
- Ambroxol
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Exsar Corporation