Assessment of ANK-700 in Patients With Relapsing Remitting Multiple Sclerosis

Study Overview
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Brief Summary
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A safety study of ANK-700 in patients with relapsing remitting multiple sclerosis. The study has two parts: Part A - first in human study in which patients receive a single dose of ANK-700 Part B - patients will receive three doses of either ANK-700 or placebo

Detailed Description
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Study ANK-700-01 is a Phase 1, FIH study designed to evaluate the safety and tolerability of ANK-700 in patients with relapsing remitting multiple sclerosis (rrms). An overview of the two parts and proposed dose groups is given below: Part A (SAD): Patients will receive a single dose of ANK-700. Part B (MAD): Patients will receive three doses of either ANK-700 or placebo.

Official Title
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A Phase 1 Study of the Safety and Tolerability of Single and Multiple Doses of ANK-700 in Patients With Relapsing Remitting Multiple Sclerosis

Conditions
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Multiple Sclerosis (MS), Relapsing Remitting Multiple Sclerosis

Intervention / Treatment
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* Drug: ANK-700
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed with RRMS per revised McDonald criteria (2017) with an EDSS score ≤ 6.5 at screening Neurologically stable with no evidence of relapse within the 28 days before signing the informed consent form (ICF) Either not currently receiving disease modifying MS therapy, or currently using fumarate drugs (dimethyl fumarate or diroximel fumarate) Patients must use a highly effective method of birth control or are sterile or postmenopausal as confirmed by study Investigator Patient has signed and understands the ICF Exclusion Criteria: Diagnosis of primary progressive MS or secondary progressive MS Uncontrolled or significant medical conditions (including active infection or chronic hepatitis) which, in the opinion of the Investigator, preclude participation Patients treated with glatiramer acetate, parenteral steroids or adrenocorticotropic hormone, β-interferon, plasma exchange within the 3 months prior to first dose Patients treated with sphingosine-1-phospate receptor modulators such as fingolimod, ozanimod, or siponimod within 6 months prior to first dose Patients treated with cytotoxic agents (including, but not limited to, cladribine, mitoxantrone, cyclophosphamide, azathioprine, and methotrexate), laquinimod, teriflunomide, or IV gamma globulin within 12 months prior to first dose Patients treated with monoclonal antibody therapy (including natalizumab, daclizumab, rituximab, ofatumumab, and ocrelizumab) within 24 months prior to first dose Patients previously treated with alemtuzumab, total lymphoid irradiation, mesenchymal stem cell or hematopoietic stem cell transplantation, or tolerance-inducing therapies for MS Contraindication to or inability to undergo gadolinium-enhanced magnetic resonance imaging (MRI) scan Use of any investigational drug or experimental procedure within previous 6 months that would interfere with the assessment of ANK-700 Patients who are pregnant or breastfeeding Patients receiving any vaccination within 28 days prior to first dose Patient does not agree to limit alcohol intake to 2 drink equivalents or less per day during the study

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ANK-700 SAD Cohort 1, Dose A<br>All enrolled patients will receive one dose of ANK-700 Dose A | Drug: ANK-700<br>* Intravenous (IV) infusion<br>|
| Experimental: ANK-700 SAD Cohort 2, Dose B<br>All enrolled patients will receive one dose of ANK-700 Dose B | Drug: ANK-700<br>* Intravenous (IV) infusion<br>|
| Experimental: ANK-700 SAD Cohort 3 Dose C<br>All enrolled patients will receive one dose of ANK-700 Dose C | Drug: ANK-700<br>* Intravenous (IV) infusion<br>|
| Experimental: MAD Cohort 4 ANK-700 Dose A or Placebo<br>All enrolled patients will receive three doses of ANK-700 Dose A or placebo | Drug: ANK-700<br>* Intravenous (IV) infusion<br>Drug: Placebo<br>* Intravenous (IV) infusion<br>|
| Experimental: MAD Cohort 5 ANK-700 Dose B or placebo<br>All enrolled patients will receive three doses of ANK-700 Dose B or placebo | Drug: ANK-700<br>* Intravenous (IV) infusion<br>Drug: Placebo<br>* Intravenous (IV) infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and severity of treatment-emergent adverse events (TEAEs) as assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) v5.0 or higher |  | Up to 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Geometric mean of maximum plasma concentration (Cmax) |  | Up to 21 days | 
| Area under the plasma concentration-time curve from time 0 to the last measurable time point (AUC last) |  | Up to 21 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autoimmune, Multiple Sclerosis (MS), Relapsing Remitting MS