PR001 ( DrugBank: - )

---

2 diseases

ID	Disease name (Link within this page)	Number of trials
6	Parkinson disease	1
19	Lysosomal storage disease	1

6. Parkinson disease

---

Clinical trials : 2,298 /  Drugs : 2,202 - (DrugBank : 350) / Drug target genes : 188 - Drug target pathways : 202

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04127578 (ClinicalTrials.gov)	January 3, 2020	14/10/2019	A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation	A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation	Parkinson Disease	Biological: PR001;Drug: Methylprednisolone;Drug: Sirolimus;Drug: Prednisone;Drug: Rituximab	Prevail Therapeutics	Eli Lilly and Company	Recruiting	35 Years	80 Years	All	12	Phase 1/Phase 2	United States;Israel

19. Lysosomal storage disease

---

Clinical trials : 854 /  Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04411654 (ClinicalTrials.gov)	June 29, 2021	11/5/2020	Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)	An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants With Type 2 Gaucher Disease	Gaucher Disease, Type 2	Biological: PR001;Drug: Methylprednisolone;Drug: Sirolimus;Drug: Prednisone	Prevail Therapeutics	Eli Lilly and Company	Recruiting	N/A	24 Months	All	15	Phase 1/Phase 2	United States;United Kingdom