A follow-on Study to Assess Long-term Safety and Tolerability of i.c.v Administration of sNN0029 in Patients With ALS

• Register: ClinicalTrials.gov
• Last refreshed on: 8 February 2016
• Main ID: NCT02269436
• Date of registration: 08/10/2014
• Prospective Registration: Yes
• Primary sponsor: Newron Sweden AB
• Public title: A follow-on Study to Assess Long-term Safety and Tolerability of i.c.v Administration of sNN0029 in Patients With ALS
• Scientific title: A Multi-centre, Open-label, follow-on Study to Assess Long-term Safety and Tolerability of Intracerebroventricular Administration of sNN0029 in Patients With Amyotrophic Lateral Sclerosis
• Date of first enrolment: January 2015
• Target sample size: 11
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02269436
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Belgium, Netherlands

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Previous participation in sNN0029-003 with completion of 12 weeks study without
clinically significant safety concerns

2. Intact continuity of the Medtronic SynchroMed® II Infusion System as judged by X-ray
of head and abdominal area

3. Clinical diagnosis of ALS classified as definite, or probable with or without
additional laboratory evidence, according to the revised WFN El Escorial criteria

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 informed consent form on the patient's behalf

Exclusion Criteria:

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

2. Ophthalmological examination (fundus photography, visual acuity and perimetry) with
any clinically significant findings that imply safety concerns for this study.

3. Diagnosis of diabetes mellitus

4. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that
cannot be not managed optimally due to:

- 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)

5. Presence of additional risk factors for thromboembolism such as obesity (Body mass
index [BMI] > 35) or use of oestrogens including combined contraceptive pills

6. Clinically significant abnormalities in haematology or clinical chemistry parameters
as assessed by the investigator

7. 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 patient to undergo
study procedures (e.g., MRI), or to give informed consent

8. For women only: pregnant, breast feeding and/or for fecund women unwillingness to use
adequate contraception during the trial such as:

- Established use of oral, injected or implanted hormonal methods of contraception
that do NOT contain oestrogens

- Placement of an intrauterine device

- Barrier methods of contraception: Condom or occlusive cap (diaphragm or
cervical/vault caps) with spermicidal foam/gel/film/cream/suppository



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

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: sNN0029 infusion solution

Primary Outcome(s)

Number Patients with Adverse Events as a measure of Safety and Tolerability [Time Frame: At 24 Months]

Secondary ID(s)

2012-005034-11

sNN0029-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors