Study of ACE-041 in Patients With Advanced Solid Tumors or Relapsed/Refractory Multiple Myeloma

Study Overview
=================
Brief Summary
-----------------
For cancer cells to grow, they need to have nutrients supplied to them through blood vessels. The study drug, ACE-041, is designed to work by blocking the growth of those blood vessels and preventing cancer cells from growing. The purpose of this study is to establish safe dose levels of ACE-041 in patients with advanced solid tumors or relapsed/refractory multiple myeloma following multiple dose administration. This study will also evaluate if ACE-041 has an effect on tumors.

Official Title
-----------------
A Phase 1 Open-Label, Multiple Dose, Dose Escalation Study of ACE-041 in Patients With Advanced Solid Tumors or Relapsed/Refractory Multiple Myeloma

Conditions
-----------------
Advanced Solid Tumors, Multiple Myeloma

Intervention / Treatment
-----------------
* Biological: ACE-041


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Diagnosis of metastatic or unresectable advanced solid tumors (solid tumors must be measurable) or relapsed/refractory multiple myeloma for which the disease has progressed despite available standard therapies or for which no standard therapy exists. Life expectancy of at least 12 weeks. Eastern Cooperative Oncology Group (ECOG) Performance status of 0, 1, or 2 (not declining within 2 weeks prior to study day 1). Exclusion Criteria: Central nervous system (CNS) metastases. Chemotherapy or other anti-cancer therapy within 4 weeks prior to study day 1, and/or nitrosoureas within the 6 weeks prior to study day 1. Lack of recovery from all toxic effects of previous chemotherapy, radiation therapy, biologic therapy, and/or experimental therapy with the exception of alopecia. Radiation therapy within 4 weeks prior to study day 1. Clinically significant pulmonary, cardiovascular, endocrine, neurologic, gastrointestinal or genitourinary disease unrelated to underlying solid tumor or multiple myeloma Significant cardiac risk (e.g. history of myocardial infarction, unstable angina, pulmonary hypertension, clinically significant arrhythmia, congestive heart failure within 1 year prior to study day 1). Diagnosis or family history of hereditary hemorrhagic telangiectasia. Major surgery within 6 weeks prior to study day 1. Parenteral antibiotics, or any life-threatening or active infection requiring parenteral antibiotic therapy within 1 month prior to study day 1. Therapeutic anti-coagulation. Uncontrolled hypertension. Autoimmune or hereditary hemolysis. Clinically significant gastrointestinal bleeding or any other clinically significant active bleeding within 3 months prior to study day 1. Treatment with another investigational drug or device, or approved therapy for investigational use within 28 days prior to study day 1. Pregnancy or lactation for female patients.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ACE-041<br>Patients assigned to 1 of 9 possible dosing groups | Biological: ACE-041<br>* Subcutaneous dose of ACE-041 approximately once every 3 weeks for a total of 4 doses. If disease stays the same or gets better, additional study drug may be offered.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the safety and tolerability of ACE-041 in patients with advanced solid tumors or relapsed/refractory multiple myeloma by monitoring adverse events, clinical laboratory tests, vital signs, physical examinations, and anti-drug antibody tests. |  | 4 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the pharmacokinetic effect of ACE-041 through the collection of blood samples and to evaluate the preliminary anti-tumor activity of ACE-041 through tumor response evaluation, collection of blood biomarkers, and imaging techniques. |  | 4 months |