An Open-Label Study of Intravenous BAL101553 in Adult Patients With Solid Tumors

Study Overview
=================
Brief Summary
-----------------
First in human, open-label, sequential dose escalation and expansion study of intravenous BAL101553 in adult patients with advanced solid tumors.

Official Title
-----------------
An Open-Label Phase I/IIa Study of Intravenous BAL101553 in Adult Patients With Advanced Solid Tumors

Conditions
-----------------
Solid Organ Tumors

Intervention / Treatment
-----------------
* Drug: BAL101553


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Age ≥ 18 years Patients with one of the following advanced or recurrent solid tumor types, who failed standard therapy or for whom no effective standard therapy is available:colorectal; gastric or cancers of the gastro-esophageal junction; non-small cell lung cancer; ovarian (or primary peritoneal); pancreatic (including ampullary); triple-negative breast Measurable tumor disease (or non-measurable ovarian cancer that can be followed by CA-125) Life expectancy ≥ 12 weeks Acceptable organ and marrow function at baseline (protocol defined laboratory parameters) Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1 Other protocol-defined inclusion criteria may apply. Exclusion Criteria: Patients who have received chemotherapy, radiotherapy, immunotherapy, or investigational agents within 4 weeks prior to starting study drug or who have not recovered from side effects of prior therapies Symptomatic brain metastases (including leptomeningeal disease) indicative of active disease Peripheral neuropathy ≥ CTCAE v4 grade 2 Uncontrolled intercurrent illness that would unduly increase the risk of toxicity or limit compliance with study requirements Women who are pregnant or breast-feeding. Men or women of reproductive potential who are not willing to apply effective birth control Systolic blood pressure ≥ 140 mmHg and/or diastolic blood pressure ≥ 90 mmHg observed as part of the screening examination. Patients treated with a calcium channel blocker or who require combination of more than 2 antihypertensives to control blood pressure. Other protocol-defined exclusion criteria may apply.

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Drug: BAL101553 at MTD<br> | Drug: BAL101553<br>* Intravenous administration<br>|
| Experimental: Drug: BAL101553 at 50% of MTD<br> | Drug: BAL101553<br>* Intravenous administration<br>|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To determine the maximum tolerated dose and characterize dose limiting toxicities of BAL101553 | First-cycle dose limiting toxicities (DLT) | 28 day cycles | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate safety and tolerability of BAL101553 treatment | Incidence of adverse events, laboratory abnormalities, clinically significant changes in vital signs or ECG assessments | 28 day cycles | 
| To evaluate BAL101553 pharmacokinetics | BAL101553 and BAL27862 PK parameters including (but not limited to): Cmax (maximum observed plasma concentration), AUC (area under the concentration time curve), half-life, volume of distribution | 28 day cycles | 
| To assess anti-tumor activity of BAL101553 | Response rate per RECIST guidelines | 28 day cycles | 
| To explore the use of biomarkers and to characterize pharmacodynamic effects of BAL101553 | Exploratory assessment of baseline levels and change from baseline in the number of circulating tumor cells and other biomarkers | 28 day cycles |