A Phase I, Randomised, Double-blind, Placebo-controlled Study in Patients With Amyotrophic Lateral Sclerosis to Further Assess the Safety and Tolerability of Intracerebroventricular Administration of sNN0029 Infusion Solution

Newron Sweden AB2 sites in 2 countries15 target enrollmentSeptember 2014

ConditionsAmyotrophic Lateral Sclerosis

InterventionssNN0029 Placebo

DrugssNN0029 Placebo

Overview

Phase: Phase 1
Intervention: sNN0029
Conditions: Amyotrophic Lateral Sclerosis
Sponsor: Newron Sweden AB
Enrollment: 15
Locations: 2
Primary Endpoint: Number of Adverse Events (AEs)
Status: Terminated
Last Updated: 10 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a phase I, multicentre randomised, double-blind, placebo-controlled trial to assess the safety and tolerability of continuous i.c.v. administration of sNN0029 infusion solution at a dose of 4µg/day in patients with Amyotrophic Lateral Sclerosis (ALS).

Registry

clinicaltrials.gov

Start Date

September 2014

End Date

October 2015

Last Updated

10 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Newron Sweden AB

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised World Federation of Neurology (WFN) El Escorial criteria.
•If patients are being treated with riluzole, they must have been on a stable dose for at least the past 30 days prior to screening.
•The patient is, in the opinion of the investigator, medically fit to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.

Exclusion Criteria

•Impaired respiratory function judged to pose a risk to the patient during anaesthesia for the device implantation.
•Hypertension defined as blood pressure \>160 mmHg systolic or \>90 mmHg diastolic.
•Values for coagulation parameters including platelet count, normalised prothrombin complex (PK-INR), activated partial thromboplastin time (APTT) outside normal ranges.
•Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.
•Diagnosis of diabetes mellitus.
•History of structural brain disease other than ALS, including tumours and hyperplasia.
•An MRI of the brain and cervical spine, and an Magnetic Resonance Angiography (MRA) of the brain with findings of tumours or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgment of the investigator, represent a surgical risk to the patient. If an MRI and/or MRA has been performed within 1 month prior to screening, the results from that examination can be used.
•Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection), alters wound healing (e.g., including bleeding disorders), or renders chronic i.c.v. delivery or device implants medically unsuitable.
•Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that cannot be not managed optimally due to:
•i. anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities), ii. underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions) iii. administration of any antiplatelet or anticoagulant medication in the preoperative period

Arms & Interventions

sNN0029 (VEGF)

4 µg/d of sNN0029 administered by continuous intracerebral infusion during12 weeks

Intervention: sNN0029

Placebo

Placebo administered by continuous intracerebral infusion during12 weeks

Intervention: Placebo

Outcomes

Primary Outcomes

Number of Adverse Events (AEs)

Time Frame: 12 weeks

The safety and tolerability of i.c.v. administration of sNN0029 infusion solution at a dose of 4 µg/day delivered via a Medtronic SynchroMed® II Infusion System will be evaluated by comparing tabulated number of events over 12 weeks by body system, preferred term and by severity and relationship to study medication/device. Serious Adverse Events/Serious Adverse Device Events will also be presented in separate tabulations.