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

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01384162
• Other study ID #: sNN0029-002
• Status: Terminated
• Phase: Phase 1/Phase 2
• First received: June 23, 2011
• Last updated: January 26, 2016
• Start date: June 2009
• Est. completion date: October 2015

Study information

• Verified date: January 2016
• Source: Newron Sweden AB
• Contact: n/a
• Is FDA regulated: No
• Health authority: Belgium: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: October 2015
• Est. primary completion date: October 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 75 Years

Eligibility

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 Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• sNN0029
ICV infusion

Locations

Country	Name	City	State
Belgium	University Hospital Leuven, Department of Neurology	Leuven

Sponsors (3)

Lead Sponsor	Collaborator
Newron Sweden AB	ICON Clinical Research, Medtronic

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	Disease activity as measured by ALS functional rating scale (FRS) - VEGF concentrations in CSF - Possible VEGF antibodies in plasma		Multiple assessments over 120 weeks	No