Table 3.

Current Clinical Trials Enrolling patients with Gaucher Disease

Name (Investigational product)	Type	Details	Eligibility	Identifier	Location
GuardOne (AVR-RD-02, AvroBio)	Gene Therapy	Phase 1/2 Lentiviral Vector	Type 1 GD, Ages 16-35	NCT04145037	Calgary, Canada; Melbourne, Australia
PROVIDE (PR001, Prevail Therapeutics/Eli Lilly)	Gene Therapy	Phase 1/2 AAV9 Vector	Type 2 GD, Ages up to 24 months	NCT04411654	New York, NY, USA; Pittsburgh, PA, USA; Minneapolis, MN, USA; Oakland, CA, USA
PROPEL (PR001A, Prevail Therapeutics/Eli Lilly)	Gene Therapy	Phase 1/2a	Parkinson disease patients with at least one pathogenic GBA1 mutation	NCT04127578	Chicago, IL, USA; Orlando FL, USA; New York, NY, USA
ELIKIDS (Eliglustat, Sanofi-Genzyme)	Drug Therapy	Phase 3	Type 1 and 3 GD, Ages 2-18, For safety and efficacy in pediatric patients	NCT03485677	Argentina, Canada, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
Ambroxol	Drug Therapy	Clinical Trial	Type 1 GD, Ages 18-75, suboptimal response to ERT	NCT03950050	Jerusalem, Israel
LEAP2IT (venglustat, Sanofi-Genzyme)	Drug Therapy	Phase 3	Type 1 and 3 GD, Ages 12 and older, in combination with Cerezyme	NCT02843035	New Haven, CT, USA; Dallas, TX, USA; Mainz, Germany; Minato-Ku, Japan; Cambridge, London, Salford, United Kingdom
N-acetylcysteine	Drug Therapy	Clinical Trial	Type 1 GD, Ages 18 and older	NCT02583672	Minneapolis, MN, USA; New York, NY, USA

GD – Gaucher Disease, ERT – Enzyme Replacement Therapy