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Renal Cell CarcinomaPhase 1NCT02795819

Study of the Pan-DAC Inhibitor AR-42 and Pazopanib in Advanced Sarcoma and Kidney Cancer

A Phase 1 study testing AR-42 and Pazopanib in people with Renal Cell Carcinoma and Soft Tissue Sarcoma, run by Virginia Commonwealth University at 1 site 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

This phase 1 study was developed to identify recommended phase 2 doses (RP2Ds) of AR-42 and pazopanib when given in combination for subsequent clinical trials and may have potentially identified candidate pharmacodynamic and predictive biomarkers. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
AR-42 and Pazopanib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
8 Jul 2016to 14 Mar 2019
Phase
Phase 1
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

  • Recurrent, unresectable, or metastatic Renal Cell Carcinoma (RCC) or Soft Tissue Sarcoma (STS) (any histologic type) for which pazopanib was an appropriate therapy
  • Measurable or evaluable disease by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
  • Age ≥ 18 years
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
  • Adequate bone marrow function as defined below:
  • Absolute neutrophil count (ANC) ≥ 1200/mm3
Show all 22Show fewer
  • Platelets ≥ 120,000/mm3
  • Hemoglobin ≥ 9.5 g/dL
  • Adequate renal function as defined below:
  • Creatinine ≤ 1.5 x upper limit of normal (ULN) for the laboratory or calculated or actual creatinine clearance ≥ 60 mL/min
  • Proteinuria ≤ 2+ [100 mg/dL] (using a random urine sample or < 3.0 gm using a 24-hour sample) (Note: If urine sample indicates ≥ 2+ [100 mg/dL]), a 24-hour urine sample must be collected and tested; urine protein in the 24-hour sample must be < 3.0 gm/24 hours.)
  • Adequate hepatic function as defined below:
  • Total bilirubin ≤ 1.5 x ULN for the laboratory (Note: Patients with known Gilbert's Syndrome were not eligible for this study)
  • Aspartate aminotransferase (AST) ≤ 2.5 x ULN for the laboratory
  • Alanine aminotransferase (ALT) ≤ 2.5 x ULN for the laboratory
  • Non-hematologic toxicities from previous cancer therapies resolved to ≤ grade 1
  • International normalized ratio (INR) ≤ 1.5
  • Activated partial thromboplastin time (aPTT) ≤ 1.5 x ULN for the laboratory
  • Left ventricular ejection fraction (LVEF) assessment (eg, echocardiogram, MUGA scan, first-pass technique) performed within 3 months prior to initiation of study treatment indicates an LVEF of ≥ 50%
  • A woman of childbearing potential (WCBP), defined as a woman who was < 60 years of age and had not had a hysterectomy, must have had a documented negative serum pregnancy test within 7 days prior to initiating study treatment
  • A WCBP and a male patient with a partner who was a WCBP must have agreed to use a medically accepted method for preventing pregnancy for the duration of study treatment and for 2 months following completion of study treatment
  • Ability to have understood and willingness to have signed the consent form

Who cannot join

  • Symptomatic or untreated brain metastasis
  • Leptomeningeal metastasis
  • Any investigational agent within 4 weeks prior to initiating study treatment
  • Previous therapy with pazopanib
  • Inability to swallow medication
  • Known or suspected malabsorption condition or obstruction
Show all 38Show fewer
  • Contraindication to antiangiogenic agents, including:
  • Serious non-healing wound, non-healing ulcer, or bone fracture
  • Major surgical procedure or significant traumatic injury within 4 weeks prior to initiating study treatment; other surgical procedures within 2 weeks prior to initiating study treatment
  • Pulmonary hemorrhage/bleeding event ≥ grade 2 within 12 weeks prior to initiating study treatment
  • Any other hemorrhage/bleeding event ≥ grade 3 within 12 weeks prior to initiating study treatment
  • History of organ allograft including corneal transplant
  • Evidence of bleeding diathesis or coagulopathy
  • Documented Gilbert's Syndrome
  • Resting systolic blood pressure (BP) < 100 mmHg
  • Hypertension defined as systolic BP ≥ 140 mmHg or diastolic BP ≥ 90 mmHg despite optimal medical management
  • QTc interval > 450 ms on screening 12-lead electrocardiogram (ECG)
  • If baseline QTc on screening ECG met exclusion criteria:
  • Check calcium, potassium, and magnesium serum levels.
  • Correct any identified hypocalcemia, hypokalemia, and/or hypomagnesemia and repeat ECG to reconfirm exclusion of patient due to prolonged QTc interval.
  • For patients with heart rate (HR) 60-100 bpm, manual read of QTc was not required.
  • For patients with a baseline HR < 60 bpm or > 100 bpm, manual read of the QT interval by a cardiologist was required, with Fridericia correction applied to determine QTc (ie, QTcF).
  • Active or clinically significant cardiac disease including any of the following:
  • Unstable angina (eg, anginal symptoms at rest) or onset of angina within 3 months prior to initiating study treatment
  • Myocardial infarction within 6 months prior to initiating study treatment
  • Cardiac arrhythmias currently requiring anti-arrhythmic therapy other than beta blockers
  • Any documented history of clinically significant thrombotic, embolic, venous, or arterial events, such as cerebrovascular accident, transient ischemic attack, deep vein thrombosis, or pulmonary embolism necessitating therapeutic anticoagulation within 6 months prior to initiating study treatment (Note: Patients with a tumor-associated thrombus of locally-involved vessels were not excluded from participating in the study.)
  • Active infection requiring treatment or chronic infection requiring suppressive therapy
  • Chronic or active hepatitis B or C infection requiring treatment with antiviral therapy
  • Pleural effusion or ascites that caused respiratory compromise (eg, ≥ grade 2 dyspnea)
  • Required ongoing treatment with other drugs thought to potentially have adverse interactions with either of the medications included in the study treatment; if such medications have been used, patients must have discontinued these agents at least 1 week prior to initiating study treatment. Examples include:
  • Strong CYP3A4 inhibitors and/or strong CYP3A4 inducers; the reference list of CYP isozymes and classification of strong, moderate, and weak interactions are available through the Food and Drug Administration (FDA website.
  • Strong inhibitors of P-glycoprotein (P-gp) and/or breast cancer resistance protein (BCRP); the reference list of strong inhibitors of P-gp and BCRP.
  • Simvastatin and other HMG-CoA reductase inhibitors (ie, statins)
  • Drugs that raise gastric pH including proton pump inhibitors and H2-blockers (Note: Short-acting antacids, in place of PPIs and H2-blockers, are permitted.)
  • HDAC inhibitors
  • Pregnancy or breastfeeding
  • Medical, psychological, or social condition that, in the opinion of the investigator, may have increased the patient's risk or limit the patient's adherence with study requirement

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Virginia Commonwealth University/Massey Cancer Center

    Richmond, Virginia, 23298

Other trials in United States

About this study

Official title: Phase 1 Study of the Pan-DAC Inhibitor AR-42 and Pazopanib in Advanced Soft Tissue Sarcoma and Renal Cell Carcinoma

This phase 1 study was developed to identify recommended phase 2 doses (RP2Ds) of AR-42 and pazopanib when given in combination for subsequent clinical trials and may have potentially identified candidate pharmacodynamic and predictive biomarkers.

Read the detailed description

This study was a single-arm, open-label, dose escalation phase 1 trial to determine the RP2Ds of AR-42 and pazopanib when given in combination to patients with advanced Renal Cell Carcinoma (RCC) or Soft Tissue Sarcoma (STS). Eligible patients had recurrent, unresectable, or metastatic RCC or STS for which pazopanib was an appropriate therapy.

AR-42 was taken orally once per day on 3 non-consecutive days each week during the first 3 weeks of each 4-week cycle. Pazopanib was taken by mouth once daily continuously during each cycle.

A modified 3+3 dose-escalation design was to be followed until the maximum tolerated doses (MTDs) were determined. The initial cohort of patients were assigned to an AR-42 dose of 20 mg taken once per day on 3 non-consecutive days during the first 3 weeks of each 4-week cycle and a pazopanib dose of 600 mg once daily continuously. Additional patients would be enrolled until a total of 12 patients were treated at the MTDs.

The protocol specifies a dose escalation plan that initially impacts only the pazopanib dose. The starting dose of pazopanib (600 mg) was planned to be escalated early so that the FDA-approved full dose of pazopanib (800 mg) would be reached before escalating the AR-42 dose. In subsequent dose-escalation steps, the dose of AR-42 was planned to be increased in 10-mg increments from a starting dose of 20 mg to a maximum dose of 40 mg.

If the number of Dose-Limiting Toxicities (DLT) showed that the MTD was exceeded with a pazopanib dose of 800 mg , the pazopanib dose was planned to be decreased to 600 mg and the AR-42 dose increased in 10-mg increments regardless of relationship of the DLT to one or both study medications.

Toxicities were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4.0). The DLT evaluation period was the first treatment cycle. A minimum of 6 doses of AR-42 and 21 doses of pazopanib were required for a patient to be DLT-evaluable (if DLT was not observed with the delivered dose). DLT was defined as any ≥ grade 3 toxicity occurring during the DLT evaluation period and attributed to one or both of the study medications EXCEPT the following:

  • The first incidence of grade 3 PPE at any dose level (subsequent incidences of grade 3 PPE will be considered DLT)
  • Grade 3 nausea and vomiting in the absence of adequate prophylaxis and/or responsive to medical management within 48 hours
  • Grade 3 diarrhea in the absence of adequate prophylaxis and/or responsive to medical management within 48 hours
  • Grade 3 fatigue responsive to medical management • Grade 3 asymptomatic hypertension lasting ≤ 7 days
  • Grade 3 electrolyte abnormalities that are corrected within 48 hours and, when corrected, can be maintained
  • Grade 3 asymptomatic lipase elevation lasting ≤ 7 days
  • Grade 3 decrease in platelet count if no clinically significant hemorrhage has occurred
  • Grades 3 and 4 decrease in white blood cell count
  • Grade 3 decrease in neutrophil count
  • Grade 4 decrease in neutrophil count lasting ≤ 7 days
  • Grades 3 and 4 decrease in lymphocyte count

Dose escalation was planned to continue until the maximum-tolerated dose (MTD), defined as one dose level below the dose in which dose-limiting toxicities (DLTs) are observed in >1 of the participants (e.g., in at least 2 participants in a cohort of at least 3). In the cohort of 12 patients at the MTD, no more than 3 DLTs were allowed to confirm the MTD. The RP2D was defined as equal to or less than the MTD, with an allowance to consider an RP2D below the MTD given the overall tolerability of the regimen including the frequency of AEs that were not DLTs and the frequency of dose modifications.

Being tested
AR-42, Pazopanib
Medical subject headings
Carcinoma, Renal Cell, Sarcoma, Neoplasm Metastasis

What participants receive

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

  • AR-42Drug

    Also called HDAC-42, OSU-HDAC42

    AR-42 tablets were taken orally once per day on 3 non-consecutive days during the first 3 weeks of each 4-week cycle. If treatment was interrupted during the cycle, the cycle was to be extended

  • PazopanibDrug

    Also called Votrient

    Pazopanib tablets were taken orally once daily continuously during each 4-week treatment cycle. There were no scheduled breaks in pazopanib therapy

Study groups in NCT02795819
Study groupRoleWhat they receive
Cohort minus 1 (-1)ExperimentalParticipants take 10 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 600mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib
Cohort 1ExperimentalParticipants take 20 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 600mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib
Cohort 2ExperimentalParticipants take 20 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 800mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib
Cohort 3AExperimentalParticipants take 30 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 800mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib
Cohort 3BExperimentalParticipants take 30 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 600mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib
Cohort 4AExperimentalParticipants take 40 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 800mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib
Cohort 4BExperimentalParticipants take 40 mg AR-42 orally per day on 3 non-consecutive days during the 1st 3 weeks of each 4-week cycle and pazopanib 600mg orally daily continuously
Receives: Drug: AR-42; Drug: Pazopanib

What is being measured

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

  1. The Recommended Phase 2 Doses (RP2Ds) of AR-42 and Pazopanib When Given in Combination.

    To determine the recommended phase 2 doses (RP2Ds) for AR-42 and pazopanib that are the same as or less than the maximum tolerated doses (MTDs). The dose-limiting toxicity (DLT) evaluation period will be the first treatment cycle. Patients must have taken a minimum of 6 AR-42 doses and a minimum of 21 pazopanib doses during cycle 1 to be considered evaluable for DLT, if DLT has not been observed at the delivered dose. Patients' treatment dose level, dose modification, evaluability for DLT, and DLTs will be listed and summarized by basic descriptive statistics such as frequency and proportion. The maximum tolerated dose(MTDs)/recommended phase 2 doses (RP2Ds) will be found based on the criteria in the protocol.

    Measured: 1 month

Secondary outcomes (2)
  1. Number of Participants With Adverse Events That Were Related to Treatment, AR-42 and Pazopanib Combination.

    The safety and toxicity of AR-42 and pazopanib when given in combination. Adverse Events (AEs) were characterized and graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4.0) to determine the safety and toxicity of the combination of AR-42 and pazopanib. All AEs regardless of grade were recorded from the beginning of the study procedures through 30 days following the end of study treatment.

    Measured: 5 months

  2. The Antitumor Effects of the AR-42 and Pazopanib Treatment Regimen in Patients With Advanced Renal Cell Carcinoma (RCC) or Soft Tissue Sarcoma (STS).

    Per Response Evaluation Criteria in Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI:

    Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm.

    Partial Response (PR): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.

    Progressive Disease (PD): At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. (Note: the appearance of one or more new lesions is also considered progression).

    Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

    Measured: 5 months

Timeline

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

  1. First posted10 Jun 2016 (estimated)
  2. Study start8 Jul 2016
  3. Primary completion24 Nov 2016
  4. Study completion14 Mar 2019

Why it stopped: Study drug no longer being clinically developed by manufacturer

Resources

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02795819
Sponsor's protocol ID
MCC-14-10774
Other IDs
NCI-2016-00851, P30CA016059
Status last verified
Oct 2019
First submitted
1 Jun 2016
Last updated
24 Oct 2019
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing AR-42 and Pazopanib in people with Renal Cell Carcinoma and Soft Tissue Sarcoma, run by Virginia Commonwealth University at 1 site 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 22 inclusion and 38 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 24 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 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.

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 24 Oct 2019). 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?

The Recommended Phase 2 Doses (RP2Ds) of AR-42 and Pazopanib When Given in Combination. (time frame: 1 month). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 24 Oct 2019.

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