Clinical trials: Gaucher Disease Type 3
Gaucher's Disease
PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
Gaucher disease is a lysosomal storage disease resulting from glucocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages.
- Trial name
- NCT00001410
- Testing
- Lysodase
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Institute of Mental Health (NIMH)
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.
Stage IV Melanoma
A Study of a Monoclonal Antibody, KW-2871, in Patients With Advanced Melanoma
This is a dose escalating cohort study to determine the maximum tolerated dose (MTD) of KW-2871 (in dose cohorts of 60, 80, and 100 mg/m2) when administered with a specified premedication regimen (ranitidine, diphenhydramine, and…
- Trial name
- NCT00199342
- Testing
- KW-2871
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Kyowa Kirin, Inc.
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)