An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis

An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump

This is an open label safety and tolerability continuation study of intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis that have previously participated in study sNN0029-001. The intention of the study is to investigate safety and tolerability of intracerebroventricular administration of sNN0029 and whether it can improve motor function and prolong survival in patients with ALS.

Study Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: sNN0029

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Belgium
  • Leuven, Belgium, 3000
    • University Hospital Leuven, Department of Neurology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria (Appendix B).
2. Previous participation in study sNN0029-001 with completion of 12 weeks study drug administration.
3. To have completed the investigations associated with safety in study sNN0029-001 without development of clinically significant safety concerns.
4. Patient has been given written and verbal information about the continuation study, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.
5. Patient has given oral and / or signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the ICF on the patient's behalf.

Exclusion Criteria:

1. Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.
2. Proliferative retinopathy.
3. Non-proliferative retinopathy of moderate severity or higher.
4. Concurrent clinically significant dementia as determined by the investigator.
5. Concurrent clinically significant depression as determined by the investigator.
6. Need for administration of any antiplatelet or anticoagulant medication (e.g. aspirin, Plavix, non-steroidal anti-inflammatory drugs [NSAIDs]). Low dose aspirin or occasional use of NSAIDs is allowed (See Appendix E).
7. Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator.
8. For female patients, ongoing pregnancy or planned pregnancy
9. Breast feeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: sNN0029, ICV infusion	Drug: sNN0029; ICV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety and tolerability through assessments of adverse events, vital signs, clinical laboratory tests, MRI of brain, fundus photography and device performance as characterized by catheter tip placement and infusion accuracy	multiple assessments over 120 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Disease activity as measured by ALS functional rating scale (FRS) - VEGF concentrations in CSF - Possible VEGF antibodies in plasma	Multiple assessments over 120 weeks

Collaborators and Investigators

• Sponsor: Newron Sweden AB
• Collaborators: Medtronic; ICON Clinical Research
• Investigators: Principal Investigator: Wim Robberecht, MD, PhD, University Hospital Leuven, Department of Neurology

Study record dates

Study Major Dates

• Study Start: June 1, 2009
• Primary Completion (Actual): October 1, 2015
• Study Completion (Actual): October 1, 2015

Study Registration Dates

• First Submitted: June 23, 2011
• First Submitted That Met QC Criteria: June 27, 2011
• First Posted (Estimate): June 28, 2011

Study Record Updates

• Last Update Posted (Estimate): January 27, 2016
• Last Update Submitted That Met QC Criteria: January 26, 2016
• Last Verified: January 1, 2016

More Information

Terms related to this study

• Keywords: Safety; ALS; Implantable; Infusion; Intracerebroventricular; VEGF165
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: sNN0029-002