A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis
This study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted catheter and an implanted SynchroMed® II pump. Patients with Amyotrophic Lateral Sclerosis will be enrolled.
Amyotrophic Lateral Sclerosis
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Double-blind, Randomised, Parallel Group Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump|
- Safety and tolerability through assessment of adverse events, ECGs, vital signs, clinical laboratory variables, MRI of brain and spinal cord, CSF sampling, and device performance as characterized by catheter tip placement and infusion accuracy. [ Time Frame: Multiple assessments over 3 months ] [ Designated as safety issue: No ]
- Time course of Amyotrophic Lateral Sclerosis Functional Rating Scale, Quality of life as measured by the EQ-5D rating scale [ Time Frame: Multiple assessments over 3 months ] [ Designated as safety issue: No ]
|Study Start Date:||December 2008|
|Study Completion Date:||June 2011|
|Primary Completion Date:||June 2011 (Final data collection date for primary outcome measure)|
Continuous ICV infusion at one of three dose levels
|Placebo Comparator: Placebo||
Continuous ICV infusion
Vascular endothelial growth factor (VEGF) is an endogenous human protein fundamental to the development of the vascular and nervous systems in the body. A role for VEGF in ALS has been suggested from observations in animal models of the disease as well as observations of a dysregulation of VEGF production in patients with ALS.
NeuroNova intends to investigate whether intracerebroventricular administration of VEGF165 in the form of the drug product sNN029 can improve motor function and prolong survival in patients with ALS, and in this first study the safety and tolerability of treatment for 3 months will be evaluated.
Assessments will include:
- Electrocardiograms, vital signs and clinical laboratory tests
- Adverse events and withdrawals related to adverse events
- Possible pathological changes in the brain, spinal cord or retina identified through magnetic resonance imaging and funduscopy
- Possible signs of intracranial bleeding or loss of blood-brain-barrier integrity through measurements of bilirubin and albumin levels in cerebrospinal fluid collected through lumbar and cervical puncture
- Device performance as characterized by catheter tip placement (determined by imaging) and infusion accuracy (pump residual volume)
The secondary objective of this study is:
To explore the effect of ICV administration of sNN0029 on the time course of Amyotrophic Lateral Sclerosis related parameters including:
- Disease activity as measured by Amyotrophic Lateral Sclerosis Functional Rating Scale
- Quality of life as measured by the EQ-5D rating scale
- To explore the levels of VEGF165 in cerebrospinal fluid collected through lumbar and cervical puncture.
|University Hospital Leuven, Department of Neurology|
|Leuven, Belgium, 3000|
|Principal Investigator:||Wim Robberecht, MD PhD||University Hospital Leuven, Department of Neurology|