Safety and Efficacy Study of MIS416 to Treat Secondary Progressive Multiple Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02228213
• Date of registration: 21/08/2014
• Prospective Registration: Yes
• Primary sponsor: Innate Immunotherapeutics
• Public title: Safety and Efficacy Study of MIS416 to Treat Secondary Progressive Multiple Sclerosis
• Scientific title: A Phase 2B Randomised, Double-Blind, Placebo-Controlled Trial of the Efficacy and Safety of MIS416 in the Treatment of Subjects With Secondary Progressive Multiple Sclerosis
• Date of first enrolment: October 2014
• Target sample size: 93
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02228213
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, New Zealand

Contacts

• Name: Michael Silverman
• Affiliation: Innate Immunotherapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

1. A historical or current cranial MRI scan demonstrating T2-hyperintense lesions
consistent with MS.

2. Has SPMS as determined by the 2010 Update to the McDonald Criteria

3. An Expanded Disability Status Scale (EDSS) of 3.0 to 6.5 at Screening.

4. Has SPMS which, in the judgment of the investigator, has been clinically active and
functionally progressive within the 2 years prior to Screening

5. The absence of MS relapse for at least two years prior to Baseline.

6. Neurologically stable for at least four weeks prior to Screening.

7. Has the following laboratory values within three days prior to initiation of
Investigational Product:

- Absolute neutrophil count (ANC) >= 1 x 109/L;

- Platelet count >= 100 x 109/L;

- Serum creatinine =< 1.5 mg/dL;

- Aspartate aminotransferase (AST) =<2 × upper limit of normal;

- Alanine aminotransferase (ALT) =< 2 × upper limit of normal.

8. Provided written informed consent to participate.

Exclusion Criteria:

1. Has primary Progressive MS (PPMS), Relapsing Remitting (RRMS), or progressive
relapsing MS as determined by the 2010 update to the McDonald Criteria.

2. Has not completed the discontinuation period for approved and/or investigational
multiple sclerosis disease modifying therapies prior to screening.

3. Has had any other immunomodulatory drug therapy or immunosuppressive therapy within
four weeks prior to Screening, or systemic corticosteroids within the eight weeks
prior to Screening.

4. Any previous exposure to investigational MS therapeutic vaccines.

5. Any use of cell-depleting monoclonal antibodies including, but not limited to,
Rituximab, or Ocrelizumab.

6. A diagnosis or history of collagen vascular disease (including Sjögren's syndrome and
systemic lupus erythematosus), anticardiolipin antibody syndrome, cerebral autosomal
dominant arteriopathy with subcortical infarcts and leukoencephalopathy, sarcoidosis,
vasculitis, Behcet's syndrome and/or Lyme disease.

7. Contraindication to MRI (e.g., pacemaker or other contraindicated implanted metal
device, allergy to gadolinium, or unmanageable claustrophobia).

8. A history of alcohol or drug abuse (including cannabinoid use) within two years prior
to Screening.

9. Has had major surgery or radiation therapy within four weeks prior to Screening.

10. Has an active infection requiring antibiotics within two weeks prior to Screening.

11. Has had active malignancy within two years of Screening, with the exception of basal
cell carcinoma and squamous cell carcinoma of the skin.

12. Uncontrolled congestive heart failure, myocardial infarction, cerebrovascular
accident, coronary/peripheral artery bypass graft surgery, or transient ischemic
attack within twelve weeks prior to Screening.

13. Has angina, other symptomatic coronary artery disease, or known cardiomyopathy.

14. Has symptomatic cardiac dysrhythmias requiring treatment, or persistent prolongation
of the QTcF (Fredericia) interval to > 450 msec for males or > 470 msec for females.



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Secondary Progressive Multiple Sclerosis

Intervention(s)

Biological: MIS416

Drug: Saline

Primary Outcome(s)

Change from baseline of neuromuscular function at 12 months [Time Frame: Baseline, 3, 6, 9 and 12 months]

Proportion of Participants with Serious and Non-Serious Adverse Events [Time Frame: Up to 12 months]

Secondary Outcome(s)

Change from baseline of neurodegeneration by assessing changes in Magnetic Resonance Imaging (MRI) markers at 12 months [Time Frame: Baseline, 3, and 12 months]

Change from baseline of activity of immune biomarkers in serum [Time Frame: Up to 1 year]

Change from baseline of disability and health status at 12 months [Time Frame: Baseline, 3, 6, 9, and 12 months]

Change from baseline of activity of immune biomarkers in cerebrospinal fluid (CSF) [Time Frame: Up to 12 months]

Change from baseline in Peripheral Blood Mononuclear Cell (PBMC) immune biomarkers [Time Frame: Up to 12 months]

Secondary ID(s)

MIS416-202

U1111-1166-0910

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

INC Research