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A Phase I Open-Label Dose Escalation Study to Assess the Safety and Tolerability of the BikDD Nanoparticle in Patients With Advanced Pancreatic Cancer


Phase 1
18 Years
N/A
Not Enrolling
Both
Pancreatic Cancer

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Trial Information

A Phase I Open-Label Dose Escalation Study to Assess the Safety and Tolerability of the BikDD Nanoparticle in Patients With Advanced Pancreatic Cancer


The Study Drug:

BikDD is a modified gene product that is designed to make cancer cells destroy themselves.
Delivering BikDD in a fat molecule (a BikDD nanoparticle) may improve the delivery of the
gene product into the cells. This may help to stop or slow the growth of pancreatic cancer.
This is the first study using BikDD nanoparticle in humans.

Study Drug Dose Level:

If you are found to be eligible to take part in this study, you will be assigned to a dose
level of BikDD nanoparticle based on when you joined this study. Different dose levels of
BikDD nanoparticle will be tested. Three (3) participants will be enrolled at each dose
level. The first group of participants will receive a low dose level. Each new group will
receive a higher dose level, if no intolerable side effects were seen. If intolerable side
effects were seen, then the next group of participants will receive a lower dose. This will
continue until the highest tolerable dose of BikDD nanoparticle is found.

The dose of study drug that you receive will never be increased. However, if you have side
effects, your dose of study drug may be lowered. Your doctor will discuss with you which
dose you will receive.

Study Drug Administration:

There will be 28 days in each cycle. The study visit schedule is described below. In
certain circumstances, with the permission of your doctor, the study visits may occur up to
2 or 3 days earlier or later than described below.

On Days 1, 8, 15, 22 of each cycle, you will receive BikDD nanoparticle through a needle in
your vein over about 10 minutes.

Study Visits:

Liver Tumor Biopsy:

Before you receive the study drug, you will have a biopsy of the liver tumor. Researchers
will compare this biopsy with a later biopsy to learn if the BikDD nanoparticle will be able
to reach the tumor and what effect it is having on the cancer.

You will be awake during the biopsy. A radiologist will find the tumor with the help of a
scan such as an ultrasound or CT scan. You will receive a local anesthetic. A needle will
be inserted through the skin into the tumor, and tissue sample(s) will be taken. You will
receive fluids and drugs for relaxation and/or pain as needed through a needle in your arm
or hand.

On Days 1, 8, 15, 22 of Cycle 1, the following tests and procedures will be performed:

- You will have a physical exam, including measurement of your weight.

- Your vital signs will be measured 5 times.

- Your performance status will be recorded.

- You will be asked about any drugs and/or supplements you are taking or have taken since
your last visit.

- You will be asked about any side effects you may be experiencing.

- Blood (about 2-3 tablespoons) will be drawn for routine tests.

- On Day 1 only, blood (about 2 teaspoons each time) will be drawn 9 times for
pharmacokinetic (PK) testing. PK testing measures the amount of study drug in the body
at different time points.

On Day 2 of Cycle 1, blood (about 2 teaspoons) will be drawn 1 time for PK testing.

On Days 4, 11, 18, and 25 of Cycle 1, the following tests and procedures will be performed:

- Blood (about 2-3 tablespoons) will be drawn for routine tests.

- You will be asked about any side effects you may be having.

On Day 24 of Cycle 1, you will have a biopsy of the liver tumor to learn if BikDD
nanoparticle is reaching the tumor and its effect on cancer. If for any reason the biopsy
can't be performed then, you may be asked to have the biopsy 6-8 days later.

On Day 1 of Cycle 2, the following tests and procedures will be performed:

- Your medical history will be recorded, including any drugs and/or supplements you are
taking or have taken since your last visit.

- You will have a physical exam, including measurement of your weight.

- Your vital signs will be measured 5 times.

- Your performance status will be recorded.

- You will be asked about any side effects you may be experiencing.

- Blood (about 2-3 tablespoons) and urine will be collected for routine tests.

- You will have an ECG.

On Days 8, 15, and 22 of Cycle 2, the following tests and procedures will be performed:

- Blood (about 1 tablespoon) will be drawn for routine tests.

- Your vital signs will be measured 5 times.

- You will be asked about any side effects you may be experiencing.

At the end of Cycle 2 and every other cycle (Cycle 4, 6, 8, and so on), you will have a CT
scan or MRI of the chest, abdomen, and pelvis to check the status of the disease. Blood
(about 1 teaspoon) will be drawn to measure your CA 19-9 level. Because it may take some
time for the effects BikDD may have on the tumor to be known, the study doctor may decide to
continue the BikDD even if the scan/MRI shows the tumor is worse. In this case, you will
have another CT scan or MRI of the chest, abdomen, and pelvis 4 to 6 weeks later. If the
cancer is worse, you will stop BikDD.

On Day 1 of Cycles 3 and beyond, the following tests and procedures will be performed:

- Your medical history will be recorded, including any drugs and/or supplements you are
taking or have taken since your last visit.

- You will have a physical exam, including measurement of your weight.

- Your vital signs will be measured 5 times.

- Your performance status will be recorded.

- You will be asked about any side effects you may be experiencing.

- You will have an ECG.

- Blood (about 2-3 tablespoons) and urine will be collected for routine tests.

On Days 8, 15, and 22 of Cycles 3 and beyond, the following tests and procedures will be
performed:

- Your vital signs will be measured 5 times.

- You will be asked about any side effects you may be experiencing.

- Blood (about 2-3 tablespoons) will be drawn for routine tests.

Length of Study:

You may continue to receive the study drug as long as your doctor thinks it is in your best
interest. If all of the cancer disappears, you will receive 2 extra cycles of study drug and
then stop study drug. You will be taken off study if the disease gets worse, your doctor
decides that it is in your best interest to stop the drug, or if you experience intolerable
side effects.

End-of-Study Visit:

Once you stop the study drug for any reason, you will have an end-of-study visit. At this
visit, the following tests and procedures will be performed.

- Your medical history will be recorded, including any drugs and/or supplements you are
taking or have taken since your last visit.

- You will have a physical exam, including measurement of your weight and vital signs

- Your performance status will be recorded.

- You will be asked about any side effects you may be experiencing.

- You will have an ECG.

- Blood (about 2-3 tablespoons) and urine will be collected for routine tests and to
measure your CA 19-9 level.

- If you have not had one within the last 4 weeks, you will have a CT scan or MRI of the
chest, abdomen, and pelvis to check the status of the disease

Once a week for at least 30 days after your last dose of study drug, you will be asked if
you are experiencing any side effects. If you do not have a clinic visit already scheduled,
you will be called. If you are called, it will take about 5-10 minutes.

If you experience any side effects, you may continue follow-up visits until the medical
problem has gone away or stabilized.

Follow-Up:

Every 2 months from then on, the study staff will continue to see how you are doing, either
at a routine clinic visit or by phone. If you are called, it will last about 5-10 minutes.
If you move, you will also be asked to provide your new address and telephone number to your
doctor.

This is an investigational study. BikDD nanoparticle is not FDA approved or commercially
available. At this time, BikDD nanoparticle is only being used in research.

Up to 30 patients will take part in this study. All will be enrolled at M D Anderson.


Inclusion Criteria:



1. Patients must have histologically or cytologically confirmed advanced pancreatic
adenocarcinoma that is unresectable or metastatic.

2. Patient must have received prior gemcitabine or a regimen containing oxaliplatin,
irinotecan, and 5-FU with or without leucovorin for treatment of advanced or
metastatic disease, unless the patient refused such treatment. Up to two prior
chemotherapeutic regimens are permitted.

3. Patients must have measurable disease including liver metastases >/= 2.0 cm amenable
to percutaneous CT or U/S guided biopsy and must agree to undergo two liver biopsies.

4. Minimum of three weeks since any major surgery, radiation, or systemic anticancer
therapy.

5. Any clinically significant residual adverse events from any prior anticancer therapy
must have resolved to grade Criteria for Adverse Events (CTCAE) v4.0.

6. Age >/=18 years. Because no dosing or adverse event data are currently available on
the use of gene therapy in patients < 18 years of age, children are excluded from
this study.

7. ECOG performance status 0 or 1.

8. Adequate hematologic, hepatic and renal parameters: leukocytes >/= 3,000/microliter,
absolute neutrophil count >/= 1,500/microliter, platelets >/= 100,000/microliter,
hemoglobin >/= 9g/dL, total bilirubin of normal (ULN) for subjects with documented liver metastases; AST and ALT ULN for subjects without evidence of liver metastases, creatinine calculated creatinine clearance of >/= 60 mL/min.

9. PT/PTT are within normal limits.

10. Women of childbearing potential must have a negative pregnancy test (serum or urine)
within 14 days prior to treatment. Women must be surgically sterile or have been
amenorrheic for at least 12 months to be considered of non-childbearing potential.

11. Women of childbearing potential and men must agree to use double barrier
contraception prior to study entry and continuing for 30 days after the last dose of
study drug.

12. Signed written informed consent/authorization form.

Exclusion Criteria:

1. Prior treatment with any investigational drug within the preceding 3 weeks.

2. Uncontrolled brain or leptomeningeal metastases, including patients who continue to
require glucocorticoids for brain or leptomeningeal metastases (Brain imaging studies
are not required if the patient does not have a history of brain metastases and has
no neurological signs or symptoms).

3. Other malignancies within the past 3 years except for adequately treated carcinoma of
the cervix or basal or squamous cell carcinomas of the skin.

4. Patients who have any known severe and/or uncontrolled medical conditions or other
conditions that could affect their participation in the study.

5. A known history of HIV seropositivity.

6. Women who are pregnant or breast feeding.

7. History of MI within 6 months, angina, history of arrhythmias on active therapy,
patients with LV ejection fraction

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Maximum Tolerated Dose (MTD)

Outcome Time Frame:

Weekly during 28 day cycles

Safety Issue:

Yes

Principal Investigator

Milind Javle, MD

Investigator Role:

Study Chair

Investigator Affiliation:

UT MD Anderson Cancer Center

Authority:

United States: Institutional Review Board

Study ID:

2007-0762

NCT ID:

NCT00968604

Start Date:

June 2013

Completion Date:

Related Keywords:

  • Pancreatic Cancer
  • Pancreatic Cancer
  • Pancreas
  • Liver
  • Liver Tumor Biopsy
  • BikDD Nanoparticle
  • C-VISA BikDD
  • Pancreatic Neoplasms

Name

Location

UT MD Anderson Cancer CenterHouston, Texas  77030