Intracystic Injection of NanoPac® in Subjects With Mucinous Cystic Pancreatic Neoplasms

NanOlogy

Status and phase: Completed; Phase 2

Conditions: Pancreatic Mucinous-Cystic Neoplasm

Treatments: Drug: NanoPac®

Study type: Interventional

Funder types: Industry

Identifiers

NCT03188991

NANOPAC-2017-01

Details and patient eligibility

About

This study will evaluate intracystic NanoPac® (Sterile Nanoparticulate Paclitaxel) administered via endoscopic ultrasound-guided fine needle injection (EUS-FNI) in subjects with mucinous cystic pancreatic neoplasms.

Full description

In this open-label, dose rising trial, subjects with mucinous cystic pancreatic neoplasms will receive intracystic NanoPac® via endoscopic ultrasound-guided fine needle injection (EUS-FNI).

In the dose escalation phase, subjects will be enrolled in sequential cohorts of NanoPac® at 6, 10, and 15 mg/mL at volumes sufficient to fill the cyst, at least equal to the amount of cyst fluid aspirated. Each cohort will have three subjects, with cohorts enrolled sequentially starting at the lowest concentration. Following Data Safety Monitoring Board (DSMB) review of the cohort data, the next cohort may begin enrolling, an additional three at the current dose may be enrolled, or if the first dose does not provide adequate safety and tolerability the study may be halted. The dose determined to be the most suitable for further evaluation, defined as the highest dose with an acceptable safety and tolerability profile (as determined by the DSMB), will be the dose used in the second phase of the study which will enroll 9 additional subjects. Subjects enrolled in the second phase of the study will also receive a second injection of NanoPac at the same dose 12 weeks after the first NanoPac injection..

Plasma samples will be taken on the day(s) of NanoPac® injection at 1 and 2 hours after injection, as well as at each of the subsequent study visits, to characterize the pharmacokinetics (PK) of intracystic NanoPac®.

Subjects will be followed for 6 months after the first NanoPac® injection for safety, tolerability and cyst response to therapy (as shown by imaging).

Enrollment: 20 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Signed informed consent;

• Patients over the age of 18;

• Recently confirmed mucinous cystic pancreatic neoplasm; may be confirmed by presence of mucin, cyst fluid carcinoembryonic antigen (CEA) above 192 U/L, or other reliable diagnostic means such as endomicroscopy; KRAS analysis may also be performed at the discretion of the Investigator;

• Unilocular cyst with diameter of at least 1.5 cm but no more than 4 cm;

• Normal hematologic, hepatic, and renal function at study entry;

• Appropriate steps taken to minimize or avoid the potential for pregnancy for subjects of child-bearing potential.*

  • Note: A female patient is considered to be of childbearing potential unless she has had a hysterectomy, is at least one year postmenopausal or has undergone tubal ligation. For the purposes of this study, adequate birth control includes at least one medically approved and highly effective method of birth control, defined as those which result in a low failure rate (i.e., < 1% per year) when used consistently and correctly, such as implants, injectables and oral contraceptives combined with the use of condoms. Only male patients whose vasectomy has been confirmed by semen analysis at least 3 months after the vasectomy are allowed not to use acceptable contraceptive methods.

Exclusion criteria

• Positive cytology indicating malignancy;
• Thrombotic or embolic events;
• Known hypersensitivity to study agent;
• Known drug or alcohol abuse;
• Pregnant or breastfeeding women.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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20 participants in 4 patient groups

Dose Escalation: NanoPac® 6 mg/mL

Experimental group

Description:

Single intracystic injection of NanoPac® at a dose of 6 mg/mL in a volume sufficient to fill the cyst, at least equal to the amount of cyst fluid aspirated

Treatment:

Drug: NanoPac®

Dose Escalation: NanoPac® 10 mg/mL

Experimental group

Description:

Single intracystic injection of NanoPac® at a dose of 10 mg/mL in a volume sufficient to fill the cyst, at least equal to the amount of cyst fluid aspirated

Treatment:

Drug: NanoPac®

Dose Escalation: NanoPac® 15 mg/mL

Experimental group

Description:

Single intracystic injection of NanoPac® at a dose of 15 mg/mL in a volume sufficient to fill the cyst, at least equal to the amount of cyst fluid aspirated

Treatment:

Drug: NanoPac®

Second Phase: NanoPac® at Best Dose

Experimental group

Description:

Intracystic injection of NanoPac®. The dose administered in the second phase will be determined during the dose escalation phase. Subjects will receive two NanoPac® injections, with the second injection administered 12 weeks after the first injection.

Treatment:

Drug: NanoPac®