Phase 1 Study of ATR-101 in Subjects With Advanced Adrenocortical Carcinoma

Study Overview
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Brief Summary
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This first-in-human study is designed to establish the safety and tolerability of ATR-101 in patients with advanced adrenocortical carcinoma whose tumor has progressed on standard therapy. Information will also be collected to determine how long ATR-101 stays in the blood, and if any effect on tumor progression is seen. Biomarkers (blood and urine tests) will determine if any effects on production of steroid hormones (cortisol, aldosterone, estrogen and testosterone) are seen.

Official Title
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Phase 1 Study of the Safety and Tolerability of ATR-101 in Adrenocortical Carcinoma

Conditions
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Adrenocortical Carcinoma, Adrenal Cancer, ACC

Intervention / Treatment
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* Drug: ATR-101


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 years; Clinical confirmation of adrenocortical carcinoma that is locally advanced or metastatic and not amendable to surgical resection; Failed or declined mitotane (adjuvant or therapeutic) therapy or a platinum-based chemotherapy regimen; Able to understand and comply with the protocol requirements; Willing and able to provide informed consent. Exclusion Criteria: Mitotane level > 5 Use of contraindicated concomitant medications Unstable medical condition that, in the judgment of the investigator, could interfere with study procedures or data interpretation.

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ATR-101<br>ATR-101 will be administered as capsules or tablets by mouth once or twice per day to ascending dose cohorts | Drug: ATR-101<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of dose-limiting toxicity and determination of maximum tolerated dose | Adverse events will be recorded and tabulated by grade and system organ class according to CTCAE v 4.03. Laboratory measures and ECGs will be assessed. | Occurrence of DLT at 28 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Area under the plasma concentration versus time curve (AUC) of ATR-101 | Plasma levels of ATR-101 will be assessed after daily oral dosing and pharmacokinetic parameters will be calculated. | Day 1 and Day 22 | 
| Change in plasma cortisol levels |  | Baseline and day 22 | 
| Change in objective measurement of tumor size | CT or MRI scans will be read according to RECIST 1.1 | Baseline and 8 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ACC, Adrenocortical carcinoma, Adrenal cancer