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Protocol Details

A Phase 1/2a, Open-Label, Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Anti-Tumor Activity of PEN-866 in Advanced Solid Malignancies

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Cancer Institute (NCI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 18 Years
Max Age: N/A

Referral Letter Required


Population Exclusion(s)

Adults who are or may become unable to consent;
Pregnant Women;


Hsp90 Inhibitor;
SN-38 Inhibitor;
Advanced Solid Malignancies;
Maximum Tolerated Dose;
Recommended Phase 2 Dose

Recruitment Keyword(s)



Small Cell Lung Carcinoma;
Triple Negative Breast Neoplasms;
Colorectal Neoplasms;
Stomach Neoplasms

Investigational Drug(s)

Folinic acid

Investigational Device(s)



Drug: PEN-866
Drug: folinic acid
Drug: fluorouracil

Supporting Site

National Cancer Institute


There are not many treatment options for people with advanced tumors whose cancer has progressed during or after standard treatments. Researchers want to find new therapies to improve outcomes for people with these cancers. PEN-866 is a new drug designed to target certain tumor cells.


To learn how PEN-866 affects the body and whether it makes tumors smaller or disappear.


Adults ages 18 and older with an advanced cancer that has not been controlled well enough by available drugs or other methods


Participants will be screened with

--Medical history

--Physical exam

--Blood and urine tests

--Tumor measurement by computerized x-ray or by magnetic resonance imaging. Participants may have to drink or have an injection of a material that makes the images clearer.

--Eye exam. Eye drops may be used for this exam.

--Electrocardiogram. Sticky pads on the arms, legs, and chest will measure heart electrical activity.

A central venous access line will be inserted if patient does not have a port. This is a tube in the chest, neck, or arm to give anti-cancer drugs.

The study drug will be given into the central venous line or port by needle.

The study will be in 4-week cycles. Participants will get PEN-866 about once a week for 3 weeks. Then they will have a week without it.

For some PEN-866 visits, participants will stay at the clinic overnight.

During the study, participants will repeat the screening tests. They may have a small sample of tumor removed. They may have hair plucked.

After finishing treatment, participants will return for 2 follow-up tests in the first month, then about every 8 weeks. They will repeat some screening tests.

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Both phase I and phase II:

-Male or female aged greater than or equal to 18 years

-Adequate organ functions

-ECOG PS of 0 - 1

-Provision and understanding of signed and dated, written informed consent prior to any mandatory study-specific procedures, sampling, analysis.

Phase Ia only:

-Histologically- or cytologically-confirmed advanced solid malignancy based on archival tumor sample having progressed after treatment with at least one prior line of anticancer therapy. In addition, participants must have no other standard of care therapies that are deemed appropriate for the treatment of their malignancy.

Phase Ib receiving PEN-866 in combination with fluorouracil and folinic acid:

-Histologically- or cytologically-confirmed participants with metastatic PDAC who have progressed after having received one or more prior lines of chemotherapy, including those whose disease has progressed within 6 months of postoperative adjuvant chemotherapy,

and have documented disease progression during or after their most recent line of anticancer therapy.

Phase IIa only:

-Measurable disease per RECIST 1.1 (i.e., at least 1measurable lesion greater than or equal to 20 mm by conventional techniques or greater than or equal to 10 mm by spiral CT scan or MRI), with the last imaging performed within 28 days before C1D1, and documented disease progression during or after their most recent line of anticancer therapy.

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Anish Thomas, M.D.
National Cancer Institute (NCI)
NIHBC 10 - CRC BG RM 4-5330
(240) 760-7343

Rasa Vilimas, R.N.
National Cancer Institute (NCI)
National Institutes of Health
Building 10
Room 8D44
10 Center Drive
Bethesda, Maryland 20892
(240) 858-3158

NCI Referral Office
National Institute of Health Clinical Center (CC), 9000 Rockville Pike, Bethesda, Maryland 20892, United States: NCI Clinical Trials Referral Office

Clinical Trials Number:


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