Trial Outcomes & Findings for Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease (NCT NCT00400634)
NCT ID: NCT00400634
Last Updated: 2022-11-10
Results Overview
The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.
COMPLETED
PHASE2
58 participants
Change from Baseline to 12 Month Visit
2022-11-10
Participant Flow
58 subjects were recruited over a 10.5 month period at 11 centers in the US
Participant milestones
| Measure |
CERE-120 Treatment Group
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10\^11 vg)
|
Sham Surgery Control Group
Subjects who were randomized to undergo sham surgery (partial burr holes)
|
|---|---|---|
|
Overall Study
STARTED
|
38
|
20
|
|
Overall Study
COMPLETED
|
34
|
19
|
|
Overall Study
NOT COMPLETED
|
4
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease
Baseline characteristics by cohort
| Measure |
CERE-120 Treatment Group
n=38 Participants
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10\^11 vg)
|
Sham Surgery Control Group
n=20 Participants
Subjects who were randomized to undergo sham surgery (partial burr holes)
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
60.1 years
STANDARD_DEVIATION 7.56 • n=5 Participants
|
57.3 years
STANDARD_DEVIATION 8.3 • n=7 Participants
|
59.1 years
STANDARD_DEVIATION 8.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
38 participants
n=5 Participants
|
20 participants
n=7 Participants
|
58 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Change from Baseline to 12 Month VisitPopulation: Subjects who completed the end-of-study visit at month 12 and had no important protocol deviations that potentially could have affected the efficacy assessment of the study drug.
The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.
Outcome measures
| Measure |
CERE-120 Treatment Group
n=34 Participants
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10\^11 vg)
|
Sham Surgery Control Group
n=19 Participants
Subjects who were randomized to undergo sham surgery (partial burr holes)
|
|---|---|---|
|
UPDRS Part III OFF
|
-7.21 units on a scale
Interval -10.34 to -4.09
|
-6.91 units on a scale
Interval -11.16 to -2.66
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Change from Baseline to 18 Month VisitPopulation: Subjects who completed the 12-month double-blind posttreatment period of the study continued to undergo double-blind assessments every 3 months until the last subject had completed the end-of-study visit at month 12. The LOCF method was used to impute data at month 18 for the subjects who had blinded data through month 15.
The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.
Outcome measures
| Measure |
CERE-120 Treatment Group
n=19 Participants
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10\^11 vg)
|
Sham Surgery Control Group
n=11 Participants
Subjects who were randomized to undergo sham surgery (partial burr holes)
|
|---|---|---|
|
UPDRS Part III OFF
|
-11.96 units on a scale
Standard Error 7.068
|
-4.34 units on a scale
Standard Error 2.479
|
Adverse Events
CERE-120 Treatment Group
Sham Surgery Control Group
Serious adverse events
| Measure |
CERE-120 Treatment Group
n=38 participants at risk
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10\^11 vg)
|
Sham Surgery Control Group
n=20 participants at risk
Subjects who were randomized to undergo sham surgery (partial burr holes)
|
|---|---|---|
|
Vascular disorders
Death
|
5.3%
2/38 • Number of events 2 • 23 months
|
0.00%
0/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
spinal compression fracture
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Glioblastoma
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate Cancer
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Renal and urinary disorders
Urinary Retention
|
5.3%
2/38 • Number of events 2 • 23 months
|
0.00%
0/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Lumbar Stenosis
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Nervous system disorders
Cerebral Hemorrhage
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Nervous system disorders
Gait Disturbance
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Nerve Root Compression
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Surgical and medical procedures
Device Failure
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Esophageal Cancer
|
2.6%
1/38 • Number of events 1 • 23 months
|
0.00%
0/20 • 23 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm Progression
|
0.00%
0/38 • 23 months
|
5.0%
1/20 • Number of events 1 • 23 months
|
|
Nervous system disorders
Fall
|
0.00%
0/38 • 23 months
|
5.0%
1/20 • Number of events 1 • 23 months
|
|
Gastrointestinal disorders
Gastroenteritis
|
0.00%
0/38 • 23 months
|
5.0%
1/20 • Number of events 1 • 23 months
|
|
Cardiac disorders
Chest Pain
|
0.00%
0/38 • 23 months
|
5.0%
1/20 • Number of events 1 • 23 months
|
Other adverse events
| Measure |
CERE-120 Treatment Group
n=38 participants at risk
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10\^11 vg)
|
Sham Surgery Control Group
n=20 participants at risk
Subjects who were randomized to undergo sham surgery (partial burr holes)
|
|---|---|---|
|
Nervous system disorders
Headache
|
73.7%
28/38 • 23 months
|
50.0%
10/20 • 23 months
|
|
Nervous system disorders
Dyskinesia
|
23.7%
9/38 • 23 months
|
30.0%
6/20 • 23 months
|
|
Nervous system disorders
Parkinson's Disease
|
21.1%
8/38 • 23 months
|
20.0%
4/20 • 23 months
|
|
Nervous system disorders
Dizziness
|
10.5%
4/38 • 23 months
|
20.0%
4/20 • 23 months
|
|
Nervous system disorders
Hypoaesthesia
|
2.6%
1/38 • 23 months
|
20.0%
4/20 • 23 months
|
|
Nervous system disorders
Tremor
|
5.3%
2/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Nervous system disorders
Freezing Phenomena
|
5.3%
2/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Nervous system disorders
Balance Disorder
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Nervous system disorders
Brain Edema
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Nervous system disorders
Cognitive Disorder
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Injury, poisoning and procedural complications
Post Procedural Pain
|
28.9%
11/38 • 23 months
|
35.0%
7/20 • 23 months
|
|
Injury, poisoning and procedural complications
Headache Postoperative
|
13.2%
5/38 • 23 months
|
25.0%
5/20 • 23 months
|
|
Injury, poisoning and procedural complications
Incision Site Complication
|
15.8%
6/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Injury, poisoning and procedural complications
Fall
|
10.5%
4/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Injury, poisoning and procedural complications
Procedural Hypotension
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Gastrointestinal disorders
Nausea
|
34.2%
13/38 • 23 months
|
30.0%
6/20 • 23 months
|
|
Gastrointestinal disorders
Constipation
|
10.5%
4/38 • 23 months
|
15.0%
3/20 • 23 months
|
|
Gastrointestinal disorders
Diarrhoea
|
7.9%
3/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Gastrointestinal disorders
Vomiting
|
7.9%
3/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Gastrointestinal disorders
Dyspepsia
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
13.2%
5/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.9%
3/38 • 23 months
|
15.0%
3/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
13.2%
5/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Shoulder Pain
|
5.3%
2/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Psychiatric disorders
Insomnia
|
21.1%
8/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Psychiatric disorders
Depression
|
10.5%
4/38 • 23 months
|
15.0%
3/20 • 23 months
|
|
Psychiatric disorders
Anxiety
|
5.3%
2/38 • 23 months
|
20.0%
4/20 • 23 months
|
|
Psychiatric disorders
Confusional State
|
2.6%
1/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Psychiatric disorders
Hallucinations
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Infections and infestations
Urinary Tract Infection
|
10.5%
4/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Infections and infestations
Nasopharyngitis
|
7.9%
3/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
5.3%
2/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Infections and infestations
Bronchitis
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Infections and infestations
Sinusitis
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
General disorders
Fatigue
|
7.9%
3/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
General disorders
Face Edema
|
5.3%
2/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
General disorders
Gait Disturbance
|
5.3%
2/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
General disorders
Edema
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
General disorders
Edema Peripheral
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Renal and urinary disorders
Micturation Urgency
|
10.5%
4/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Renal and urinary disorders
Hematuria
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Renal and urinary disorders
Pollakiuria
|
5.3%
2/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Renal and urinary disorders
Urinary Incontinence
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Renal and urinary disorders
Urinary Retention
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Renal and urinary disorders
Renal Failure
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.2%
5/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Skin and subcutaneous tissue disorders
Periorbital Edema
|
7.9%
3/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
7.9%
3/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Investigations
Blood Testosterone Decreased
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Respiratory, thoracic and mediastinal disorders
Sleep Apnea Syndrome
|
5.3%
2/38 • 23 months
|
0.00%
0/20 • 23 months
|
|
Vascular disorders
Hypertension
|
7.9%
3/38 • 23 months
|
10.0%
2/20 • 23 months
|
|
Vascular disorders
Hypotension
|
5.3%
2/38 • 23 months
|
5.0%
1/20 • 23 months
|
|
Eye disorders
Vision Blurred
|
2.6%
1/38 • 23 months
|
10.0%
2/20 • 23 months
|
Additional Information
Raymond T. Bartus, Executive Vice President and Cheif Scientific Officer
Ceregene, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60