A Window of Opportunity Study to Investigate Mechanisms of Actions of Novel Therapeutic Agents in Patients With Resectable Solid Tumor Malignancies

• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (Other)
• Overall Status: Terminated
• CT.gov ID: NCT03471286
• Collaborator: Incyte Corporation (Industry)
• Enrollment: 2
• Location: 1
• Arm: 1
• Actual Duration (Months): 25.8
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

This is a single-institution, single arm, open label, window of opportunity clinical trial that will act as a Master Protocol with a primary objective to assess pharmacodynamic effects of the novel study drugs in subjects with resectable solid tumor malignancies.

Each Sub-protocol conducted under the Master Protocol will evaluate different novel therapeutic agent(s).

Estimated enrollment is 15 subjects per Sub-Protocol.

Condition or Disease	Intervention/Treatment	Phase
Solid Tumor	Drug: Epacadostat	Phase 1

Detailed Description

Patients will have a short-course (4-weeks) administration of a given compound in the timeframe between the diagnostic biopsy and the surgical resection. A pre-treatment tumor biopsy will be obtained, and blood samples will be collected before, during, and at the end of treatment.

Study Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 1b Window of Opportunity Study to Investigate Mechanisms of Actions of Novel Therapeutic Agents in Patients With Resectable Solid Tumor Malignancies
• Actual Study Start Date: Jun 6, 2019
• Actual Primary Completion Date: Jul 30, 2021
• Actual Study Completion Date: Jul 30, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Sub-Protocol A Epacadostat	Drug: Epacadostat; Drug is taken orally two times per day for 14 days, prior to surgical resection of tumor.

Outcome Measures

Primary Outcome Measures

1. Pharmacodynamic effects as measured by changes in biomarkers in pre- and post-dose tumor and blood specimens [Approximately 1 year]

   To establish baseline levels and measure pharmacodynamic biomarker changes in tumor and/or blood samples after treatment with the study drug. Sub- Protocol A will focus on changes in CD8+ T cells, CD4+Foxp3+ cells, NK cells, DCs and immune checkpoints.

Secondary Outcome Measures

1. Safety and tolerability as measured by incidence of AEs (Adverse Events) [Approximately 1 year]

   To assess the safety and tolerability of the study drug in the pre-surgical setting graded according to Common Terminology Criteria for Adverse Events (CTCAE), version 4.0.

2. Pathological Response Rate as measured by correlation of biomarkers in tumor and/or blood specimens [Approximately 1 year]

   To evaluate pathological response rate of solid tumor malignancies in subjects receiving neo-adjuvant treatment with the study drug and to assess a possible correlation with biomarkers either in the tumor specimens either in the bloods samples and the pathological response.

3. Overall Survival [Approximately 2 years]

   To determine the overall survival (OS) status of subject treated which the study drug at 6 months, 1 year and 2 year

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 100 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Must have biopsy-proven solid tumor that is eligible for elective surgical resection, disease does not require immediate therapy, and there is NO approved/ standard therapy available that is shown to prolong overall survival.

2. ECOG performance status of 0 or 1.

3. Must have a tumor lesion that is amenable to biopsy, and willing to undergo biopsy.

4. Willing to provide tissue and blood samples for research.

Exclusion Criteria:

1. The following solid tumors are NOT eligible: Primary brain tumor, Ocular melanoma, Head and neck cancer, Breast cancer, Prostate cancer, Testicular cancer, and Stage III rectal cancer.

2. Any active malignancy within 3 years prior, except: Adequately treated basal cell or squamous cell skin cancer, or early stage cancers (carcinoma in situ or stage 1) treated with curative intent.

3. Any uncontrolled intercurrent illness, including but not limited to: Symptomatic congestive heart failure, Unstable angina pectoris or coronary angioplasty or stenting within 6 months prior to enrollment, Cardiac arrhythmia, Psychiatric illness or social situations that would limit compliance with study requirements, or Ongoing or active infection.

4. Any known sensitivity to or history of allergic reaction to compounds of similar chemical or biological composition of study drug(s).

5. Unwilling or unable to follow the study schedule.

Other protocol defined inclusion/exclusion criteria may apply.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	Baltimore	Maryland	United States	21231

Sponsors and Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• Incyte Corporation

Investigators

• Principal Investigator: Nilofer Azad, MD, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• ClinicalTrials.gov Identifier: NCT03471286
• Other Study ID Numbers: J17168; IRB00156271; I-24360-17-13
• First Posted: Mar 20, 2018
• Last Update Posted: Aug 26, 2021
• Last Verified: Aug 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Neoplasms

Study Results

No Results Posted as of Aug 26, 2021