Gaucher Disease Clinical Trial
Official title:
A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 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.
This was a Phase 2, open-label study in participants with Gaucher disease, a lysosomal storage disorder. Afegostat tartrate (also known as AT2101 or isofagomine tartrate) is designed to act as a pharmacological chaperone by selectively binding to misfolded β-glucocerebrosidase (GCase) and helping it fold correctly, intended to restore GCase activity. The study consisted of a 14-day screening period, a 28-day treatment period, and a 7-day wash-out period. Participants received 1 of 4 dosing regimens for afegostat tartrate. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
|
||
| Completed |
NCT04430881 -
A National Study in Patients With Unexplained Splenomegaly
|
||
| Completed |
NCT02536937 -
A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
| Completed |
NCT02536911 -
A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
| Completed |
NCT01411228 -
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
|
Phase 3 | |
| Terminated |
NCT04094181 -
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
|
||
| Completed |
NCT00391625 -
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
|
Phase 1/Phase 2 | |
| Completed |
NCT03625882 -
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
|
||
| Active, not recruiting |
NCT05526664 -
Omics Gaucher Study: Multiomic Approach
|
||
| Completed |
NCT02536755 -
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
|
Phase 3 | |
| Recruiting |
NCT01344096 -
Thrombocytopathy in Gaucher Disease Patients
|
N/A | |
| Completed |
NCT01881633 -
A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
|
Phase 1 | |
| Recruiting |
NCT06116071 -
Biomarkers Related to Bone in Pediatric Gaucher Disease
|
||
| Recruiting |
NCT01951989 -
Intra-monocyte Imiglucerase Kinetics in Gaucher Disease
|
Phase 2 | |
| Completed |
NCT00258778 -
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
|
Phase 1 | |
| Recruiting |
NCT04388969 -
World Data on Ambroxol for Patients With GD and GBA Related PD
|
||
| Recruiting |
NCT05992532 -
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
|
||
| Terminated |
NCT04145037 -
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
|
Phase 1/Phase 2 | |
| Completed |
NCT00302146 -
Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
|
||
| Active, not recruiting |
NCT02605603 -
SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
|