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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Terminated

Phase

Phase 1

Locations

Belgium

Study Type

Interventional

Intervention

sNN0029

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, VEGF165, Safety, Intracerebroventricular, Infusion, Implantable

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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

Sites / Locations

University Hospital Leuven, Department of Neurology

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

sNN0029, ICV infusion

Arm Description

Outcomes

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

Secondary Outcome Measures

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

Full Information

NCT ID

NCT01384162

First Posted

June 23, 2011

Last Updated

January 26, 2016

Sponsor

Newron Sweden AB

Collaborators

Medtronic, ICON Clinical Research

1. Study Identification

Unique Protocol Identification Number

NCT01384162

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

January 2016

Overall Recruitment Status

Terminated

Why Stopped

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).

Study Start Date

June 2009 (undefined)

Primary Completion Date

October 2015 (Actual)

Study Completion Date

October 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Newron Sweden AB

Collaborators

Medtronic, ICON Clinical Research

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

Keywords

ALS, VEGF165, Safety, Intracerebroventricular, Infusion, Implantable

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

sNN0029, ICV infusion

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

sNN0029

Intervention Description

ICV infusion

Primary Outcome Measure Information:

Title

Time Frame

multiple assessments over 120 weeks

Secondary Outcome Measure Information:

Title

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Wim Robberecht, MD, PhD

Organizational Affiliation

University Hospital Leuven, Department of Neurology

Official's Role

Principal Investigator

Facility Information:

Facility Name

University Hospital Leuven, Department of Neurology

City

Leuven

ZIP/Postal Code

3000

Country

Belgium

12. IPD Sharing Statement

Learn more about this trial

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

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