A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00800501
• Date of registration: 29/11/2008
• Prospective Registration: Yes
• Primary sponsor: Newron Sweden AB
• Public title: A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis
• Scientific title: A Double-blind, Randomised, Parallel Group Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis, Using an Implanted Catheter and SynchroMed® II Pump
• Date of first enrolment: December 2008
• Target sample size: 18
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00800501
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

Belgium

Contacts

• Name: Wim Robberecht, MD PhD
• Affiliation: University Hospital Leuven, Department of Neurology

Key inclusion & exclusion 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.

2. Age 18 to 75 years inclusive.

3. If patients are being treated with riluzole, they must have been on a stable dose for
at least 30 days.

4. Ophthalmological examination at screening with normal findings regarding vascular
structure and function.

5. MRI/magnetic resonance angiography (MRA) examination of the brain and cervical spinal
cord at screening with no findings of tumors or potential sources of pathological
bleedings, or abnormality that may interfere with the assessments of safety or
efficacy or that would, in the judgement of the investigator, represent a surgical
risk to the subject.

6. Values of coagulation parameters including platelet count, normalized prothrombin
complex (PK-INR), activated partial thromboplastin time (APTT) within normal ranges.

7. Patient is medically able to undergo the surgery required for stereotactic
implantation of the catheter and infusion pump.

8. Patient has been given written and verbal information, has had the opportunity to ask
questions about the study, and understands the time and procedural commitments.

9. Patient has given 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. Impaired respiratory function judged to pose a risk to the patient during anaesthesia
for the device implantation.

2. Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.

3. Diagnosis of diabetes mellitus.

4. Proliferative retinopathy.

5. Non-proliferative retinopathy of moderate severity or higher.

6. Concurrent clinically significant dementia as determined by the investigator.

7. Concurrent clinically significant depression as determined by the investigator.

8. History of structural brain disease other than ALS, including tumours and
hyperplasia.

9. 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 ICV delivery or device implants medically unsuitable.

10. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that
is not managed optimally. Physicians should specifically investigate anatomical
factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or
other abnormalities), underlying disorders of the coagulation cascade, platelet
function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver
disease, or other medical conditions), and the administration of any antiplatelet or
anticoagulant medication (e.g., aspirin, Plavix, NSAIDs) in the pre- or perioperative
period. Any of those conditions or drugs could place a patient at an increased risk
for intraoperative or postoperative bleeding.

11. Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.

12. Presence of cardiac pacemakers, spinal cord stimulators, implantable programmable
intraspinal drug pumps, or any other device that may interfere or interact with the
programmer, without prior approval by Medtronic.

13. Clinically significant abnormalities in hematology or clinical chemistry parameters
as assessed by the investigator.

14. Ongoing medical condition that according to the investigator would interfere with the
conduct and assessments in the study. Examples are medical disability (e.g., severe
degenerative arthritis, compromised nutritional state, peripheral neuropathy) that
would interfere with the assessment of safety and efficacy of investigational product
or device performance, or would compromise the ability of the subject to undergo
study procedures (e.g., MRI), or to give informed consent.

15. Participation in another clinical trial with an investigational drug or device within
3 months prior to Screening visit.

16. For female subjects, ongoing pregnancy or planned pregnancy during the period of
treatment with study drug.

17. Breast feeding during the period of treatment with study drug.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Placebo

Drug: sNN0029

Primary Outcome(s)

Safety and tolerability through assessment of adverse events, ECGs, vital signs, clinical laboratory variables, MRI of brain and spinal cord, CSF sampling, and device performance as characterized by catheter tip placement and infusion accuracy. [Time Frame: Multiple assessments over 3 months]

Secondary Outcome(s)

Time course of Amyotrophic Lateral Sclerosis Functional Rating Scale, Quality of life as measured by the EQ-5D rating scale [Time Frame: Multiple assessments over 3 months]

Secondary ID(s)

sNN0029-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Medtronic

ICON Clinical Research