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

NCT01384162 | Terminated Phase 1 DMC

• 1 other identifier: sNN0029-002
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; VEGF165; Safety; Intracerebroventricular; Infusion; Implantable

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Disease activity as measured by ALS functional rating scale (FRS) - VEGF concentrations in CSF - Possible VEGF antibodies in plasma

  Multiple assessments over 120 weeks

Study Arms (1)

sNN0029, ICV infusion

EXPERIMENTAL

Drug: sNN0029

Interventions

sNN0029 DRUG

ICV infusion

sNN0029, ICV infusion

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).
• Previous participation in study sNN0029-001 with completion of 12 weeks study drug administration.
• To have completed the investigations associated with safety in study sNN0029-001 without development of clinically significant safety concerns.
• 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.
• 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.

You may not qualify if:

• Hypertension defined as blood pressure \>160 mmHg systolic or \>90 mmHg diastolic.
• Proliferative retinopathy.
• Non-proliferative retinopathy of moderate severity or higher.
• Concurrent clinically significant dementia as determined by the investigator.
• Concurrent clinically significant depression as determined by the investigator.
• 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).
• Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator.
• For female patients, ongoing pregnancy or planned pregnancy
• Breast feeding

Sponsors & Collaborators

• Newron Sweden AB: lead
• Medtronic: collaborator
• ICON Clinical Research: collaborator

Study Sites (1)

University Hospital Leuven, Department of Neurology

Leuven, 3000, Belgium

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Wim Robberecht, MD, PhD

  University Hospital Leuven, Department of Neurology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 23, 2011
• First Posted: June 28, 2011
• Study Start: June 1, 2009
• Primary Completion: October 1, 2015
• Study Completion: October 1, 2015
• Last Updated: January 27, 2016

Record last verified: 2016-01

Locations

BE (1)