A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis Clinical Trial

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

Status Completed

Clinical Trial Summary

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.

Clinical Trial Description

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. ;

Study Design

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

• NCT number: NCT00800501
• Study type: Interventional
• Source: Newron Sweden AB
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: December 2008
• Completion date: June 2011