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

This study has been terminated.
(Issues with development and supply of infusion system for delivery of IMP. Lack of favorable benefit risk ratio in sNN0029-003 study (review of interim data).)
Sponsor:
Collaborators:
Medtronic
ICON Clinical Research
Information provided by (Responsible Party):
Newron Sweden AB
ClinicalTrials.gov Identifier:
NCT01384162
First received: June 23, 2011
Last updated: January 26, 2016
Last verified: January 2016
  Purpose
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.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: sNN0029
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Newron Sweden AB:

Primary Outcome Measures:
  • 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 [ Time Frame: multiple assessments over 120 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Disease activity as measured by ALS functional rating scale (FRS) - VEGF concentrations in CSF - Possible VEGF antibodies in plasma [ Time Frame: Multiple assessments over 120 weeks ] [ Designated as safety issue: No ]

Enrollment: 15
Study Start Date: June 2009
Study Completion Date: October 2015
Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: sNN0029, ICV infusion Drug: sNN0029
ICV infusion

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01384162

Locations
Belgium
University Hospital Leuven, Department of Neurology
Leuven, Belgium, 3000
Sponsors and Collaborators
Newron Sweden AB
Medtronic
ICON Clinical Research
Investigators
Principal Investigator: Wim Robberecht, MD, PhD University Hospital Leuven, Department of Neurology
  More Information

Responsible Party: Newron Sweden AB
ClinicalTrials.gov Identifier: NCT01384162     History of Changes
Other Study ID Numbers: sNN0029-002 
Study First Received: June 23, 2011
Last Updated: January 26, 2016
Health Authority: Belgium: Institutional Review Board

Keywords provided by Newron Sweden AB:
ALS
VEGF165
Safety
Intracerebroventricular
Infusion
Implantable

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on July 26, 2016