Clinical trials: Gaucher's Disease Type I
Gaucher Disease
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
Primary Objective: Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to <18 years old) with Gaucher disease type 1 and type 3.
- Trial name
- NCT06523517
- Testing
- Eliglustat Tartrate Capsules
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Peking Union Medical College Hospital
Gaucher Disease, Type 1
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00954460
- Testing
- velaglucerase alfa
- Where
- 32 sites in United States
- Sponsor
- Shire
Gaucher Disease
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00478647
- Testing
- GA-GCB (velaglucerase alfa)
- Where
- 15 sites in United States, Israel and 3 more
- Volunteers
- 40 people
- Sponsor
- Shire
Gaucher Disease, Type 1, MRI
Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
This study with standardized reading MRIs, will provide for the first time objective and quantified data on organomegaly (liver and spleen volumes) as well as bone alteration (Bone Marrow Burden11) of French patients treated with…
- Trial name
- EIROS (NCT03333447)
- Testing
- MRI
- Where
- France
- Volunteers
- 20 people
- Sponsor
- CEN Biotech
Gaucher Disease Type 1
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate…
- Trial name
- GD (NCT06818838)
- Testing
- LY-M001
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Lingyi Biotech Co., Ltd.
Gaucher Disease, Type 1, Type 1 Gaucher Disease
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy.
- Conditions
- Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
- Trial name
- NCT00433147
- Testing
- Afegostat tartrate
- Where
- 10 sites in United States
- Volunteers
- 30 people
- Sponsor
- Amicus Therapeutics
Gaucher Disease, Gaucher Disease, Type 1
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD.
- Trial name
- NCT05487599
- Testing
- LY3884961
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 8 people
- Sponsor
- Prevail Therapeutics
Gaucher Disease
A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease
The purpose of this study was to evaluate the safety, tolerability, efficacy, immunogenicity, PD and PK characteristics of LY-M001 injection in children with GD1 aged 6 years ≤ age < 18 years.
- Trial name
- NCT06528080
- Testing
- LY-M001
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
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 1
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Ambroxol hydrochloride, an over-the-counter antitussive available in many markets , was identified as an interesting pharmacological chaperone.
- Trial name
- NCT03950050
- Testing
- Ambroxol
- Where
- Israel
- Volunteers
- 40 people
- Sponsor
- Shaare Zedek Medical Center
Gaucher Disease
A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition
Substances in the body, so-called biomarkers, can help predict the severity of Gaucher disease (GD)-related bone problems in adults.
- Trial name
- NCT05702814
- Testing
- No Intervention
- Where
- Spain
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Takeda
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
Gaucher's Disease
Bone Response to Enzyme Replacement in Gaucher's Disease
The purpose of this study is to examine how the skeleton responds to repeated doses of enzyme replacement therapy in patients with type I Gaucher's disease who have had their spleens removed.
- Trial name
- NCT00001416
- Testing
- CEREDASE™
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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
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.
Gaucher Disease
The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease
The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients
- Trial name
- NCT01161914
- Testing
- Cerezyme®, ISU302
- Sponsor
- ISU Abxis Co., Ltd.
Gaucher Disease
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar…
- Trial name
- NCT02574286
- Testing
- Velaglucerase alfa, Vitamin D
- Where
- 15 sites in United States, Israel and 2 more
- Volunteers
- 21 people
- Sponsor
- Shire
Gaucher Disease, Type 1
A Screening Study Evaluating Disease Status of Gaucher Type I Patients
A screening study to identify and characterize the disease status of patients with Gaucher Type 1 disease for potential inclusion in a subsequent phase 3 clinical study.
- Trial name
- NCT00795197
- Where
- 3 sites in United States, India
- Sponsor
- Genzyme, a Sanofi Company
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