. 2018 Dec 12;16(1):166–175. doi: 10.1007/s13311-018-00694-0

Table 1.

Completed clinical trials in Parkinson’s disease

Disease	Vector	Delivery route	Anatomic target	Dose	Primary outcome measure(s)	Time to follow-up	Adverse events	Current status
Parkinson’s disease	AAV2-GAD	Intraparenchymal	Subthalamic nucleus	1 × 10^12 vg	Change in UPDRS scores	12 months	Serious adverse event: bowel obstruction in AAV2-GAD group; not attributed to treatment	Terminated (due to financial reasons; NCT00643890)
Parkinson’s disease	AAV-hAADC-2	Intraparenchymal (CED)	Putamen	9 × 10^10 vg 3 × 10^11 vg	Safety and tolerability of AAV-hAADC-2 as measured by adverse events	6 months	Asymptomatic intracranial hemorrhage reported postop	Completed (NCT00229736)
Parkinson’s disease	AAV2-NTN (CERE-120)	Intraparenchymal (CED)	Putamen and substantia nigra	2.4 × 10^11 vg (substantia nigra) 1.0 × 10^12 vg (putamen)	Change from baseline in UPDRS motor examination	3 years	Two patients experienced hemorrhages with transient symptoms	Active, not recruiting (NCT00985517)

Disease

Vector

Delivery route

Anatomic target

Dose

Primary outcome measure(s)

Time to follow-up

Adverse events

Current status

Parkinson’s disease

AAV2-GAD

Intraparenchymal

Subthalamic nucleus

1 × 10^12 vg

Change in UPDRS scores

12 months

Serious adverse event: bowel obstruction in AAV2-GAD group; not attributed to treatment

Terminated (due to financial reasons; NCT00643890)

Parkinson’s disease

AAV-hAADC-2

Intraparenchymal (CED)

Putamen

9 × 10^10 vg

3 × 10^11 vg

Safety and tolerability of AAV-hAADC-2 as measured by adverse events

6 months

Asymptomatic intracranial hemorrhage reported postop

Completed (NCT00229736)

Parkinson’s disease

AAV2-NTN (CERE-120)

Intraparenchymal (CED)

Putamen and substantia nigra

2.4 × 10^11 vg (substantia nigra)

1.0 × 10^12 vg (putamen)

Change from baseline in UPDRS motor examination

3 years

Two patients experienced hemorrhages with transient symptoms

Active, not recruiting (NCT00985517)

AADC = aromatic amino acid decarboxylase; CED = convection-enhanced delivery; GAD = glutamate decarboxylase; GDNF = glial derived neurotropic factor; UPDRS = Unified Parkinson’s Disease Rating Scale