Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
more
I am looking for
added new label for I am looking for
more
Advanced Filters
Found 31 clinical trials
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ (SEED)

Primary Objective To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ To

cerezyme
imiglucerase
neuronopathic gaucher disease
  • 0 views
  • 06 May, 2022
  • 5 locations
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

replacement therapy
  • 11 views
  • 15 May, 2022
  • 1 location
GSL Synthetase Inhibitor or in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor

Immune checkpoint blockade has made great but unsatisfied success in treating cancers. One important reason is the hijacked HLA (Human Leukocyte Antigen) antigen presentation. Eliglustat could inhibit glycosphingolipids synthesis and restore HLA-I antigen presentation and transform the immunogenicity of tumor cells. Therefore,GSL synthetase inhibitor eliglustat monotherapy or in combination with …

cancer
solid tumor
  • 0 views
  • 14 May, 2022
  • 1 location
Gaucher Disease Outcome Survey (GOS)

The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of

velaglucerase alfa
  • 131 views
  • 30 Jan, 2022
  • 1 location
Prodromal Parkinsonian Features in GBA1 Mutation Carriers

before the appearance of the disease, and also to compare them to a group of diagnosed Gaucher patients and a group of healthy people who are not carriers of Gaucher disease. A group of those

parkinson's disease
Accepts healthy volunteers
  • 0 views
  • 08 Mar, 2022
  • 1 location
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of

deficiency
alglucerase
imiglucerase
eliglustat
type 1 gaucher disease
  • 522 views
  • 18 Apr, 2022
  • 284 locations
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of

deficiency
alglucerase
imiglucerase
eliglustat
type 1 gaucher disease
  • 0 views
  • 01 Apr, 2022
  • 283 locations
Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

genetic disorder
palliative care
genetic disorders
stem cell transplantation
hematopoetic
  • 363 views
  • 13 Nov, 2021
  • 1 location
Gaucherite - A Study to Stratify Gaucher Disease

The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict

cancer
enzyme therapy
  • 117 views
  • 28 Jul, 2021
  • 8 locations
A Long-term Follow-up Study of Gaucher Disease

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy

replacement therapy
substrate reduction therapy
  • 26 views
  • 30 Sep, 2021
  • 1 location