A Study to Determine the Efficacy, Safety and Pharmacokinetics of GMI-1271 as Adjunct to Standard of Care for the Treatment of Multiple Myeloma

Study Overview
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Brief Summary
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This study will evaluate GMI-1271, a specific E-selectin antagonist, in multiple myeloma as adjunct to standard of care chemotherapy used to treat this disease.

Official Title
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A Phase I Open-Label Dose Escalation Study to Determine the Efficacy, Safety and Pharmacokinetics of GMI-1271 as Adjunct to Standard of Care Chemotherapy for the Treatment of Multiple Myeloma

Conditions
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Multiple Myeloma

Intervention / Treatment
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* Drug: GMI-1271


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and females ≥ 18 years of age Confirmed diagnosis of Multiple Myeloma Subjects with progressive MM who have received at least 1 prior therapy and who have already undergone or are not expected to undergo hematopoietic stem cell transplantation Subjects previously treated with an IMiD-based regimen or ineligible for an IMiD-based treatment regimen Subjects must be receiving treatment with a proteasome inhibitor-based regimen (bortezomib-based or carfilzomib-based) Adequate hepatic, renal, and cardiac function Exclusion Criteria: Intolerant to bortezomib or carfilzomib Progressing evidence of end organ damage attributed to the underlying disease Plasma cell leukemia Congestive heart failure Acute active infection Non-hematologic malignancy within the past 3 years except: a) adequately treated basal cell or squamous cell skin cancer, b) carcinoma in situ of the cervix, or c) prostate cancer with stable PSA Significant peripheral neuropathy

Ages Eligible for Study
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Minimum Age: 18 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: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Dose 1<br> | Drug: GMI-1271<br> <br> |
| Experimental: Dose 2<br> | Drug: GMI-1271<br> <br> |
| Experimental: Dose 3<br> | Drug: GMI-1271<br> <br> |
| Experimental: Dose 4<br> | Drug: GMI-1271<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety assessed by frequency, severity and relatedness of AEs | Assessed by frequency, severity and relatedness of AEs | up to 6 months | 
| Overall Response Rate | Overall Response Rate = Stringent Complete Response + Complete Response + Very Good Partial Response + Partial Response | up to 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical benefit rate = Stringent Complete Response + Complete Response + Very Good Partial Response + Partial Response + Minimal Response + Stable Disease |  | 18 months | 
| Time to response |  | 18 months | 
| Time to progression |  | 18 months | 
| Duration of response |  | 18 months | 
| Progression free survival |  | 18 months | 
| Overall survival |  | 18 months | 
| Time versus plasma concentration profile of GMI-1271 |  | up to Day 23 of Cycle 1 (each cycle is 21 or 28 days) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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GMI-1271, bortezomib, carfilzomib, multiple myeloma