Solid TumorsPhase 1/2NCT02587962

Dose-escalation Study of Birinapant and Pembrolizumab in Solid Tumors

A Phase 1/2 study testing Birinapant and Pembrolizumab in people with Solid Tumors, run by Medivir at 9 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

An ascending dose study in patients with solid tumors to evaluate the safety, tolerability, pharmacodynamics and efficacy of birinapant when given in combination with pembrolizumab. A dose expansion phase of 4 cohorts will also be included. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Solid Tumors
What is being tested?: Birinapant and Pembrolizumab
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 34 participants
Where: 9 sitesUnited States
Study dates: 4 Aug 2017to 17 Feb 2020
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Histologically confirmed solid malignancy that is metastatic or unresectable for which standard curative or palliative measures do not exist or are no longer effective (Dose Escalation phase only)
Measurable disease according to response evaluation criteria in solid tumors (RECIST) v 1.1
Eastern Cooperative Oncology Group (ECOG) score of 0 or 1
Normal organ and marrow function
Dose Expansion phase specific additional inclusion criteria:
Patients with metastatic colorectal cancer with no available therapy options that are known to provide clinical benefit per institutional standard of care (colorectal cancer cohort only)

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Patients must have a histologically confirmed epithelial ovarian cancer, primary peritoneal cancer or fallopian tube solid tumor cancer that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care (ovarian cancer cohort only)
Patients must have histologically or cytologically confirmed cervical squamous cell carcinoma that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care (cervical cancer cohort only)
Patients must have histologically or cytologically confirmed head and neck squamous cell carcinoma that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care. (various solid tumors cohort: head and neck squamous cell carcinoma groups only).
Patients must have received prior therapy with an anti-programmed death protein (PD-1) or anti-PD-ligand 1(L1) antibody, or previously participated in Merck MK 3475 clinical trials. Patients must have experienced documented, confirmed radiographic progression of disease by immune RECIST (iRECIST), or by RECIST v1.1 (various solid tumors cohort head and neck squamous cell carcinoma, Check point inhibitor experienced group only).
Patients must have histologically or cytologically confirmed small cell lung carcinoma (SCLC) that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care (various solid tumors cohort, SCLC group only).
Patients must have histologically or cytologically confirmed cholangiocarcinoma that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care (various solid tumors cohort, cholangiocarcinoma group only).
Patients must have histologically or cytologically confirmed mesothelioma that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care (various solid tumors cohort, mesothelioma group only).
Patients must have histologically or cytologically confirmed carcinoma of the esophagus including the gastroesophageal junction that is locally advanced or metastatic with no available therapy options that are known to provide clinical benefit per institutional standard of care (various solid tumors cohort, gastroesophageal carcinoma group only).

Who cannot join

Exclusion criteria apply to all phases and cohorts in the study unless otherwise stated
Prior monoclonal antibody, within 4 weeks prior to first dose of study drug.
Prior chemotherapy, targeted small molecule therapy or radiotherapy within 2 weeks prior to first dose of study drug.
Patients who have received any other investigational agents within 4 weeks of first dose of study drug.
Prior therapy with an anti-PD-1, anti-PD-L1, anti-PD-ligand 2(L2), anti-cluster of differentiation 137 (anti-CD137), or anti-Cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4) antibody. (Not applicable for various solid tumors cohort, head and neck squamous cell carcinoma check-point inhibitor experienced group)
History of allergic reactions attributed to compounds of similar chemical or biologic composition to birinapant or pembrolizumab or their constituents.

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Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, hypertension, unstable angina pectoris, cardiac arrhythmia, autoimmune disease or inflammatory diseases, or psychiatric illness/social situations that would limit compliance with study requirements.
Evidence of active, non-infectious pneumonitis or a history of interstitial lung disease.
Known history of Human Immunodeficiency Virus (HIV) (HIV1/2 antibodies), or Active Hepatitis B (HBsAg reactive). Patients with active Hepatitis C (HCV-RNA qualitative).
Currently breast feeding, pregnant or planning to conceive or father Children from screening through 120 Days after last dose of study drug.
Patients who have received anti-PD-L2, anti-CD137, or anti-Cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4) antibody (including ipilimumab or any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathways) (Various solid tumor cohort, head and neck squamous cell carcinoma check point inhibitor experienced group only)

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

United States9 sites

The Sidney Kimmel Comprehensive Cancer Center at John Hopkins
Baltimore, Maryland, 21287
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Mary Crowley Cancer Research
Dallas, Texas, 75251
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234
MD Anderson Cancer Center, The University of Texas
Houston, Texas, 77030
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Mid Florida Hematology and Oncology Center
Orange City, Florida, 32763
Thomas Jefferson University Sidney Kimmel Cancer Center
Philadelphia, Pennsylvania, 19107
UCLA Dept of Medicine-Hematology/Oncology
Santa Monica, California, 90404

Other trials in United States

About this study

Official title: A Phase 1/2 Multicenter, Single-Arm, Open-Label, Dose-Escalation Study of Birinapant in Combination With Pembrolizumab (KEYTRUDA®) in Patients With Relapsed or Refractory Solid Tumors

Read the detailed description

This study will be conducted in two phases. The Phase 1 portion of the study will employ a sequential group dose-escalation design to determine the dose-limiting toxicity (DLT) and recommended Phase 2 dose (RP2D) of birinapant administered in combination with 200 mg pembrolizumab, both administered as a 30-minute intravenous (IV) infusion. The following proposed doses of birinapant are to be evaluated: 5.6, 11, 17, and 22 mg/m2.

The Phase 2 portion, the dose expansion phase, will compromise 4 cohorts of 26-30 patients.

The 4 cohorts will include the following:

Colorectal cancer
Ovarian Cancer
Cervical cancer
Various solid tumors (30 patients, including 5 patients with each of the following 6 tumor types: Head and Neck Squamous Cell Carcinoma (HNSCC)-checkpoint-inhibitor naïve; and HNSCC checkpoint-inhibitor experienced; Gastroesophageal carcinoma; Mesothelioma; Small cell lung cancer (SCLC); Cholangiocarcinoma

A Simon's 2-stage design will be used for each of the cohorts in colorectal cancer, ovarian cancer and cervical cancer. A predefined interim analysis allowing stopping each of these cohorts for futility and safety will be conducted in the first stage to limit undue exposure before further inclusion into a given cohort. The design of the various solid tumors cohort will limit undue exposure in any of the selected tumor types by limiting the number of enrolled patient to five in each tumor type.

Conditions: Solid Tumors
Being tested: Birinapant, Pembrolizumab

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BirinapantDrug
Birinapant intravenous (IV) on Days 1 and 8 of each 21-Day Cycle. The following escalating doses of birinapant to be studied: 5.6, 11, 17, and 22 mg/m2. In the expansion phase the assigned recommended phase two dose will be administered in all cohorts
PembrolizumabDrug
Also called KEYTRUDA, lambrolizumab, MK-3475, SCH 9000475
200 mg pembrolizumab IV on Day 1 of each 21-Day Cycle

Study groups in NCT02587962
Study group	Role	What they receive
Birinapant in combination with pembrolizumab	Experimental	Birinapant in combination with pembrolizumab Receives: Drug: Birinapant; Drug: Pembrolizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

Blood Pressure (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through blood pressure.
Measured: Baseline and up to 2 yrs (follow-up)
Electrocardiogram: QT Interval (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through electrocardiogram.
Measured: Baseline and up to 2 yrs (follow-up)
Amylase and Lipase (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through amylase and lipase.
Measured: Baseline and up to 2 yrs (follow-up)
Thyroxine Free (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through thyroxine free.
Measured: Baseline and up to 2 yrs (follow-up)
Thyrotropin (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through Thyrotropin.
Measured: Baseline and up to 2 yrs (follow-up)
Hemoglobin (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through hemoglobin.
Measured: Baseline and up to 2 yrs (follow-up)
Physical Exam (Safety and Tolerability in the Dose Escalation Phase)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through physical exam.
Measured: Baseline and up to 2 yrs (follow-up)
Overall Response (Applicable for: Dose Escalation Phase and Dose Expansion Phase in Cohorts of Colorectal Cancer, Ovarian Cancer and Cervical Cancer)
Evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1. A responder was a patient who showed best overall response of complete response (CR, disappearance of all target lesions) or partial response (PR, ≥30% decrease in the sum of the longest diameter of target lesions), which was confirmed again at least 4 weeks after the initial assessment.
Measured: Baseline and up to 2 yrs (follow-up)

Secondary outcomes (8)

Tumor Response Evaluated Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1:
Analysis of progression-free survival (PFS); time to progression, where progressive disease (PD) corresponds to ≥20% increase in the sum of the longest diameter of target lesions.
Measured: Every 9 weeks; up to 2 yrs
Blood Pressure (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of birinapant at the recommended phase 2 dose (RP2D) when given in combination with pembrolizumab assessed through blood pressure.
Measured: Baseline and up to 2 yrs (follow-up)
Electrocardiogram: QT Interval (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of birinapant at the recommended phase 2 dose (RP2D) when given in combination with pembrolizumab assessed through electrocardiogram.
Measured: Baseline and up to 2 yrs (follow-up)
Amylase and Lipase (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of birinapant at the recommended phase 2 dose (RP2D) when given in combination with pembrolizumab assessed through amylase and lipase.
Measured: Baseline and up to 2 yrs (follow-up)
Thyroxine Free (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of birinapant at the recommended phase 2 dose (RP2D) when given in combination with pembrolizumab assessed through thyroxine free.
Measured: Baseline and up to 2 yrs (follow-up)
Thyrotropin (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of birinapant at the recommended phase 2 dose (RP2D) when given in combination with pembrolizumab assessed through thyrotropin.
Measured: Baseline and up to 2 yrs (follow-up)
Hemoglobin (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of birinapant at the recommended phase 2 dose (RP2D) when given in combination with pembrolizumab assessed through hemoglobin.
Measured: Baseline and up to 2 yrs (follow-up)
Physical Exam (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Evaluation of the safety and tolerability of the RP2D of birinapant when given in combination with pembrolizumab assessed through physical exam.
Measured: Baseline and up to 2 yrs (follow-up)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

4 Aug 201717 Feb 2020

First posted27 Oct 2015 (estimated)
Study start4 Aug 2017
Primary completion17 Feb 2020
Study completion17 Feb 2020

Why it stopped: Based on a futility analysis, the data monitoring committee recommended to stop enrollment.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Solid TumorsStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 34 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02587962
Sponsor's protocol ID: BPT-201
Other IDs: MK3475 KEYNOTE KN163
Status last verified: Dec 2020
First submitted: 21 Oct 2015
Last updated: 14 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
Active, not recruitingPhase 1
A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK043 in Patients With Advanced Solid Tumor
Neoplasms
Active, not recruitingPhase 3
A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial
Solid Tumors
RecruitingPhase 1
IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Relapsed Solid Tumor Malignancies, Refractory Solid Tumor Malignancies
RecruitingObservational study
Immune Checkpoint Inhibitor Toxicity Risk Prediction in Solid Tumors
Malignant Solid Neoplasm

All Solid Tumors trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2 Multicenter, Single-Arm, Open-Label, Dose-Escalation Study of Birinapant in Combination With Pembrolizumab (KEYTRUDA®) in Patients With Relapsed or Refractory Solid Tumors
ClinicalTrials.gov ID: NCT02587962
Acronym / sponsor ID: BPT-201
Conditions: Solid Tumors
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Based on a futility analysis, the data monitoring committee recommended to stop enrollment.
Enrolment: 34 participants (actual)
Lead sponsor: MedivirIndustry (pharma, biotech, devices)
Collaborators: Merck Sharp & Dohme LLC
Study start: 4 Aug 2017 (actual)
Primary completion: 17 Feb 2020 (actual)
Study completion: 17 Feb 2020 (actual)
First posted: 27 Oct 2015 (estimated)
Results: Posted 14 Jan 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

The Phase 2 portion, the dose expansion phase, will compromise 4 cohorts of 26-30 patients.

The 4 cohorts will include the following:

Colorectal cancer
Ovarian Cancer
Cervical cancer
Various solid tumors (30 patients, including 5 patients with each of the following 6 tumor types: Head and Neck Squamous Cell Carcinoma (HNSCC)-checkpoint-inhibitor naïve; and HNSCC checkpoint-inhibitor experienced; Gastroesophageal carcinoma; Mesothelioma; Small cell lung cancer (SCLC); Cholangiocarcinoma

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Birinapant in combination with pembrolizumab	Experimental	Drug: Birinapant, Drug: PembrolizumabBirinapant in combination with pembrolizumab

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (14 listed)

Histologically confirmed solid malignancy that is metastatic or unresectable for which standard curative or palliative measures do not exist or are no longer effective (Dose Escalation phase only)
Measurable disease according to response evaluation criteria in solid tumors (RECIST) v 1.1
Eastern Cooperative Oncology Group (ECOG) score of 0 or 1
Normal organ and marrow function
Patients with metastatic colorectal cancer with no available therapy options that are known to provide clinical benefit per institutional standard of care (colorectal cancer cohort only)

Key exclusion criteria (11 listed)

Exclusion criteria apply to all phases and cohorts in the study unless otherwise stated
Prior monoclonal antibody, within 4 weeks prior to first dose of study drug.
Prior chemotherapy, targeted small molecule therapy or radiotherapy within 2 weeks prior to first dose of study drug.
Patients who have received any other investigational agents within 4 weeks of first dose of study drug.
Prior therapy with an anti-PD-1, anti-PD-L1, anti-PD-ligand 2(L2), anti-cluster of differentiation 137 (anti-CD137), or anti-Cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4) antibody. (Not applicable for various solid tumors cohort, head and neck squamous cell carcinoma check-point inhibitor experienced group)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BirinapantBirinapant intravenous (IV) on Days 1 and 8 of each 21-Day Cycle. The following escalating doses of birinapant to be studied: 5.6, 11, 17, and 22 mg/m2. In the expansion phase the assigned recommended phase two dose will be administered in all cohorts	Drug	Birinapant in combination with pembrolizumab
PembrolizumabAlso: KEYTRUDA, lambrolizumab, MK-3475, SCH 9000475200 mg pembrolizumab IV on Day 1 of each 21-Day Cycle	Drug	Birinapant in combination with pembrolizumab

Primary outcome

Blood Pressure (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through blood pressure.
Electrocardiogram: QT Interval (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through electrocardiogram.
Amylase and Lipase (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through amylase and lipase.
Thyroxine Free (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through thyroxine free.
Thyrotropin (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through Thyrotropin.
Hemoglobin (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through hemoglobin.
Physical Exam (Safety and Tolerability in the Dose Escalation Phase)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluation of the safety and tolerability of birinapant when given in combination with pembrolizumab assessed through physical exam.
Overall Response (Applicable for: Dose Escalation Phase and Dose Expansion Phase in Cohorts of Colorectal Cancer, Ovarian Cancer and Cervical Cancer)
Time frame: Baseline and up to 2 yrs (follow-up)
Evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1. A responder was a patient who showed best overall response of complete response (CR, disappearance of all target lesions) or partial response (PR, ≥30% decrease in the sum of the longest diameter of target lesions), which was confirmed again at least 4 weeks after the initial assessment.

8 secondary outcomes

Tumor Response Evaluated Using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1:
Time frame: Every 9 weeks; up to 2 yrs
Blood Pressure (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)
Electrocardiogram: QT Interval (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)
Amylase and Lipase (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)
Thyroxine Free (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)
Thyrotropin (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)
Hemoglobin (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)
Physical Exam (Safety and Tolerability in the Dose Expansion Phase - Colorectal Cancer Cohort)
Time frame: Baseline and up to 2 yrs (follow-up)

Results and safety

Results posted to the registry on 14 Jan 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Phase 1 - 5.6 mg	3	1
Phase 1 - 11 mg	3	0
Phase 1 - 17 mg	6	0
Phase 1 - 22 mg	7	1
Phase 2 - 22 mg	15	0

Primary outcome: Blood Pressure (Safety and Tolerability in the Dose Escalation Phase)

Time frame: Baseline and up to 2 yrs (follow-up). Mean (Standard Deviation). Unit: mmHg

Measure	Phase 1 - 5.6 mg n = 3	Phase 1 - 11 mg n = 3	Phase 1 - 17 mg n = 6	Phase 1 - 22 mg n = 7
Systolic blood pressure at baseline	137.3 (18.6)	143.3 (14.5)	134.0 (22.1)	118.6 (11.4)
Systolic blood pressure at follow-up	120.0 (NA)	107.0 (1.4)	119.7 (30.2)	120.0 (NA)
Diastolic blood pressure at baseline	78.0 (2.0)	77.0 (15.5)	74.3 (9.3)	66.6 (7.9)
Diastolic blood pressure at follow-up	84.0 (NA)	66.0 (8.5)	71.3 (11.4)	75.0 (NA)

Population analysed: As the population of participants were very ill, assessments outside clinical praxis were sometimes not performed.

Primary outcome: Electrocardiogram: QT Interval (Safety and Tolerability in the Dose Escalation Phase)

Time frame: Baseline and up to 2 yrs (follow-up). Mean (Standard Deviation). Unit: ms

Measure	Phase 1 - 5.6 mg n = 3	Phase 1 - 11 mg n = 3	Phase 1 - 17 mg n = 6	Phase 1 - 22 mg n = 7
Electrocardiogram QT interval at baseline	368.7 (30.6)	388.3 (11.2)	386.3 (32.9)	365.9 (32.7)
Electrocardiogram QT interval at follow-up	392.0 (NA)	–	380.0 (NA)	364.0 (NA)

Population analysed: As the population of participants were very ill, assessments outside clinical praxis were sometimes not performed.

Primary outcome: Amylase and Lipase (Safety and Tolerability in the Dose Escalation Phase)

Time frame: Baseline and up to 2 yrs (follow-up). Mean (Standard Deviation). Unit: U/L

Measure	Phase 1 - 5.6 mg n = 3	Phase 1 - 11 mg n = 3	Phase 1 - 17 mg n = 6	Phase 1 - 22 mg n = 7
Amylase at baseline	33.7 (32.5)	38.3 (9.0)	64.3 (8.0)	40.6 (27.4)
Amylase at follow-up	27.0 (NA)	49.0 (41.0)	56.5 (38.9)	60.0 (NA)
Lipase at baseline	17.0 (12.2)	24.0 (21.0)	32.2 (21.4)	49.9 (63.3)
Lipase at follow-up	12.0 (NA)	15.5 (10.6)	16.0 (2.8)	18.0 (NA)

Population analysed: As the population of participants were very ill, assessments outside clinical praxis were sometimes not performed.

Primary outcome: Thyroxine Free (Safety and Tolerability in the Dose Escalation Phase)

Time frame: Baseline and up to 2 yrs (follow-up). Mean (Standard Deviation). Unit: nmol/L

Measure	Phase 1 - 5.6 mg n = 3	Phase 1 - 11 mg n = 3	Phase 1 - 17 mg n = 6	Phase 1 - 22 mg n = 7
Thyroxine free at baseline	16.8 (5.0)	15.0 (3.0)	15.0 (3.3)	18.7 (1.6)
Thyroxine free at last assessment	25.2 (8.0)	16.7 (5.4)	16.9 (4.1)	16.5 (3.6)

Population analysed: As the population of participants were very ill, assessments outside clinical praxis were sometimes not performed.

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 2 years. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase 1 - 5.6 mg	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3
Phase 1 - 11 mg	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Phase 1 - 17 mg	4 of 6 (66.7%)	6 of 6 (100%)	1 of 6
Phase 1 - 22 mg	3 of 7 (42.9%)	7 of 7 (100%)	0 of 7
Phase 2 CRC - 22 mg	5 of 15 (33.3%)	15 of 15 (100%)	1 of 15

Limitations and caveats (from the sponsor): The dose escalation (Phase 1) part was analyzed per the protocol. Based on data from the interim analysis of the colorectal cancer cohort (dose expansion \[Phase 2\] part), the study was closed early due to futility on recommendation by the data monitoring committee (DMC).

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Solid Tumors

RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
Active, not recruitingPhase 1
A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK043 in Patients With Advanced Solid Tumor
Neoplasms
Active, not recruitingPhase 3
A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial
Solid Tumors
RecruitingPhase 1
IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
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RecruitingObservational study
Immune Checkpoint Inhibitor Toxicity Risk Prediction in Solid Tumors
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

9 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Birinapant and Pembrolizumab in people with Solid Tumors, run by Medivir at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 14 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Blood Pressure (Safety and Tolerability in the Dose Escalation Phase) (time frame: Baseline and up to 2 yrs (follow-up)). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02587962.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2021.