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

Newron

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: sNN0029

Study type: Interventional

Funder types: Industry

Identifiers

NCT01384162

sNN0029-002

Details and patient eligibility

About

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.

Enrollment: 15 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

sNN0029, ICV infusion

Experimental group

Treatment:

Drug: sNN0029