Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Ceregene | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | Intracerebral administration of CERE-120 |
|
| 2 | Sham Comparator | Sham Neurosurgery |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) | Drug | CERE-120 5.4 x 10^11 vg |
| |
| Measure | Description | Time Frame |
|---|---|---|
| UPDRS Part III OFF | 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. | Change from Baseline to 12 Month Visit |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| UPDRS Part III OFF | 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. |
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Joao Siffert, M.D. | Ceregene | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama, Birmingham | Birmingham | Alabama | 35233 | United States | ||
| University of California, San Francisco |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 20970382 | Result | Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20. |
Not provided
Not provided
Not provided
58 subjects were recruited over a 10.5 month period at 11 centers in the US
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | CERE-120 Treatment Group | Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg) |
| FG001 | Sham Surgery Control Group | Subjects who were randomized to undergo sham surgery (partial burr holes) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Sham Surgery |
| Procedure |
Bilateral partial thickness burr holes placed, no intraparenchymal injections |
|
| Change from Baseline to 18 Month Visit |
| San Francisco |
| California |
| 94143-0138 |
| United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Mount Sinai School of Medicine | New York | New York | 10029 | United States |
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Pennsylvania Hospital | Philadelphia | Pennsylvania | 19107 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | CERE-120 Treatment Group | Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg) |
| BG001 | Sham Surgery Control Group | Subjects who were randomized to undergo sham surgery (partial burr holes) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | UPDRS Part III OFF | 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. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Change from Baseline to 12 Month Visit |
|
|
| ||||||||||||||||||||||||||||
| Other Pre-specified | UPDRS Part III OFF | 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. | 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. | Posted | Least Squares Mean | Standard Error | units on a scale | Change from Baseline to 18 Month Visit |
|
23 months
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | CERE-120 Treatment Group | Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg) | 13 | 38 | 38 | 38 | ||
| EG001 | Sham Surgery Control Group | Subjects who were randomized to undergo sham surgery (partial burr holes) | 4 | 20 | 20 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Death | Vascular disorders | MedDRA (10.0) | Systematic Assessment | Neither death was considered related to CERE-120 |
|
| spinal compression fracture | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment | Not considered related to CERE-120 |
|
| Glioblastoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Prostate Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Urinary Retention | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Lumbar Stenosis | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Cerebral Hemorrhage | Nervous system disorders | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120, Related to surgical procedure |
|
| Gait Disturbance | Nervous system disorders | MedDRA (10.0) | Systematic Assessment | Not considered related to CERE-120 |
|
| Nerve Root Compression | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Device Failure | Surgical and medical procedures | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Esophageal Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (10.0) | Systematic Assessment | Considered not related to CERE-120 |
|
| Neoplasm Progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (10.0) | Systematic Assessment |
| |
| Fall | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Gastroenteritis | Gastrointestinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Chest Pain | Cardiac disorders | MedDRA (10.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Dyskinesia | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Parkinson's Disease | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Freezing Phenomena | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Balance Disorder | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Brain Edema | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Cognitive Disorder | Nervous system disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Post Procedural Pain | Injury, poisoning and procedural complications | MedDRA (10.0) | Systematic Assessment |
| |
| Headache Postoperative | Injury, poisoning and procedural complications | MedDRA (10.0) | Systematic Assessment |
| |
| Incision Site Complication | Injury, poisoning and procedural complications | MedDRA (10.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (10.0) | Systematic Assessment |
| |
| Procedural Hypotension | Injury, poisoning and procedural complications | MedDRA (10.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Shoulder Pain | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Confusional State | Psychiatric disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Hallucinations | Psychiatric disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA (10.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (10.0) | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA (10.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (10.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (10.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (10.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Face Edema | General disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Gait Disturbance | General disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Edema | General disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Edema Peripheral | General disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Micturation Urgency | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Hematuria | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Urinary Incontinence | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Urinary Retention | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Renal Failure | Renal and urinary disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Periorbital Edema | Skin and subcutaneous tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Pruritis | Skin and subcutaneous tissue disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Blood Testosterone Decreased | Investigations | MedDRA (10.0) | Systematic Assessment |
| |
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Sleep Apnea Syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (10.0) | Systematic Assessment |
| |
| Vision Blurred | Eye disorders | MedDRA (10.0) | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Raymond T. Bartus, Executive Vice President and Cheif Scientific Officer | Ceregene, Inc. | 858-458-8823 | rtbartus@ceregene.com |
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
Not provided
Not provided
| Male |
|
| Units | Counts |
|---|---|
| Participants |
|
|