Joining a clinical trial is an important and personal decision. Only a qualified healthcare professional can determine whether you are eligible to participate in a clinical trial. However, this information may be beneficial in initiating a conversation with your physician. If you believe this clinical trial might be a good fit and are interested in participating, please take the next step and contact a research site to see if you are eligible. The BOLSTER trial may be an option if you meet the following eligibility criteria: Male or female and a minimum of 18 years of age If you are a female who can get pregnant, you must use appropriate contraception. If you are male with a female partner who can get pregnant, you must use appropriate contraception. Patients that: Have been diagnosed with metastatic bile duct cancer or bile duct cancer that cannot be surgically removed and have not received any prior anti-cancer treatment Each clinical study has its own guidelines for who can participate, called eligibility criteria. However, only the research study staff can determine if you qualify to enroll in the study. The study doctor will tell you about all the other criteria that you have to meet in order to participate in this Study.

Approximately 40 subjects will be enrolled in this study. If you are interested in participating, the clinical trial team will schedule a screening visit to determine if you are eligible to participate in the BOLSTER trial. Participants who provide informed consent will be screened for eligibility for up to 28 days. Once eligibility is confirmed, you will be randomly assigned (like flipping a coin) to receive either study drug or placebo (an inactive substance that looks the same as the study drug) in addition to standard chemotherapy. The first dose of either Certepetide or placebo will be given alone without any other medication. Three days later, the first cycle of treatment will start. Tumor scans will be performed every 8 weeks (56 days ± 7 days) while on treatment.

Is this Cholangiocarcinoma trial on ClinicalTrials.gov?

Yes. To view the BOLSTER trial page on clinicaltrials.gov, please visit https://clinicaltrials.gov/study/NCT05712356#study-overview

What are the possible side effects of Treatment with Certepetide?

As with any investigational drug, there may be side effects that are not yet known. Potential risks include: Gastrointestinal bleeding, Diarrhea, Pneumonia (lung infection), Buildup of fluid in the sac around the heart (pericardial effusion). The most common side effects that have been seen in subjects with pancreatic cancer who received Certepetide in combination with gemcitabine and nab-paclitaxel chemotherapy regardless of its dose and its causal relationship included: Low levels of white blood cells (neutropenia), Low levels of red blood cells (anemia), Loss of sensation or weakness of peripheral nerves (nerves located outside of the brain and spinal cord). Additional side effects seen in subjects treated with both Certepetide and chemotherapy (gemcitabine and nab paclitaxel) include: Fatigue, Nausea, Constipation, Diarrhea, Hair loss, Swelling (peripheral edema), Abdominal pain, Vomiting, Change in taste, Loss of appetite, Breathlessness (dyspnea), Rash, Cough, Low platelets (thrombocytopenia), Nosebleeds (epistaxis). These adverse events are commonly seen with gemcitabine and nab-paclitaxel when given without Certepetide. No new or worsened side effects were observed when Certepetide was given in combination with gemcitabine and nab-paclitaxel than those observed in patients who received chemotherapy alone. Allergies to medications like Certepetide are considered unlikely, but there is still the possibility of an allergic reaction. A severe allergic reaction could be life threatening. Please seek treatment immediately and tell the study doctor and study staff if you have any of these symptoms. An allergic reaction could include: Rash or hives, Difficulty breathing or wheezing, Sudden changes in blood pressure that may make you feel dizzy or lightheaded, Swelling around the mouth, throat, or eyes, Fast pulse, Sweating, There is a possibility that Certepetide might bind to the cells that help blood clot (platelets), although this was not seen in any studies in animals or in patients who have taken Certepetide to date. While you are taking part in this study, your blood cell counts will be monitored closely, and you will receive appropriate treatment if you have a low platelet count or are bleeding. Although very rare in subjects with cancer, when many tumor cells are killed rapidly, the blood can develop high levels of uric acid, potassium, and phosphorus, and low calcium levels, which can cause kidney damage. Symptoms include pain, discomfort, or burning when urinating, low urine output, blood in the urine, dull ache on the side of the upper back, loss of appetite, vomiting, cramps, and weakness. This possible side effect, called tumor lysis syndrome, is usually seen within a few days after starting cancer treatment. Subjects will be monitored, and supportive care provided if abnormal laboratory results or symptoms are observed. Based on animal studies, there is a possibility that Certepetide might cause small changes in the testes and/or the tubes that connect the testes to the urethra (epididymis), however this has not been seen in subjects to date. If you experience any of the above signs or symptoms, you should notify your doctor immediately, as you may need immediate treatment and hospitalization. Your study doctor may give you drugs to treat these symptoms.

How long will I be in the study?

Study treatment will continue until your cancer worsens or you have medically unacceptable side effects. Patients may also withdraw from the study at any time and for any reason without penalty or loss of benefits to which you are otherwise entitled.

Will my personal results and information be kept confidential?

By choosing to take part in this study, you are giving us permission to use your personal health information that includes health information in your medical records and information that can identify you. Lisata, the Sponsor, is responsible for looking after your personal health information, keeping it confidential, and using it properly. While every effort will be made to protect the confidentiality of your information, absolute confidentiality in clinical trials cannot be guaranteed. The Sponsor and companies working with the Sponsor will visit the research facility to monitor the clinical study. They will review your records for these visits. Your records may be reviewed by the FDA and the Institutional Review Board (IRB). The IRB is a group of scientists and non-scientists who review the ethics of clinical research. The goal of the IRB is to protect the rights and welfare of study subjects. The people above who may review your records will do so to make sure the study has been performed the right way. They also want to make sure that your health information has been collected appropriately, or for other reasons that are allowed under the law. A description of this clinical trial will be available on http://www.ClinicalTrials.gov, as required by U.S. Law. This website will not include information that can identify you. The website may include a summary of the research results. You can search this website at any time.

What if this Cholangiocarcinoma study is not right for me?

You can choose not to take part in this clinical trial. If you decide not to participate in this trial, your other choices may include the following: Getting treatment or care for your cancer without being in a study, Taking part in another cholangiocarcinoma study, Getting no treatment for cholangiocarcinoma, Getting cancer comfort care (also called palliative care) The study doctor will discuss available cancer treatment options, including their risks and benefits, with you.

FORTIFIDE: Metastatic Pancreatic Ductal Adenocarcinoma Clinical Trial

FORTIFIDE trial design

Phase 1b/2a, double-blind, placebo-controlled, three-arm, randomized study evaluating a 4-hour infusion of certepetide (LSTA1) when added to standard of care (SoC) in patients with second-line metastatic pancreatic ductal adenocarcinoma (mPDAC) who have progressed on FOLFIRINOX.

Certepetide is an experimental drug that has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. In other words, the study drug is not yet available for use outside of a clinical trial.

FORTIFIDE trial objective

The main goal of the clinical trial is to test the safety and therapeutic effect of certepetide when given as a 4-hour infusion in combination with SoC in subjects with second-line mPDAC who have progressed on FOLFIRINOX. Haystack Oncology MRD™ technology will be used to measure ctDNA for early efficacy exploration.

Locations Actively Recruiting Clinical Trial Patients

The principal investigator is the physician responsible for the scientific and technical direction of the clinical trial at each respective location.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided below. Please refer to this study as LSTA1-P06.

UNITED STATES

North Carolina, USA

FirstHealth of the Carolinas, Inc.

Pinehurst, North Carolina, United States, 28374

Contact: Lindsey Ferry | tel: 910-715-2200 | email: lferry@firsthealth.org

Principal Investigator: Charles Kuzma, MD

Who may participate in this clinical trial

Joining a clinical trial is an important and personal decision. Only a qualified healthcare professional can determine whether you are eligible to participate in a clinical trial. However, this information may be beneficial in initiating a conversation with your physician. If you believe this clinical trial might be a good fit and are interested in participating, please take the next step and contact a research site to see if you are eligible.

The FORTIFIDE trial may be an option if you meet the following eligibility criteria:

Male or female and a minimum of 18 years of age
If you are a female who can get pregnant, you must use appropriate contraception. If you are male with a female partner who can get pregnant, you must use appropriate contraception.
Patients that have been diagnosed with metastatic pancreatic ductal adenocarcinoma that cannot be surgically removed
Patients that have documented disease progression on first-line standard or modified FOLFIRINOX treatment and are eligible for second-line treatment with nab-paclitaxel and gemcitabine

Each clinical study has its own guidelines for who can participate, called eligibility criteria. However, only the research study staff can determine if you qualify to enroll in the study. The study doctor will tell you about all the other criteria that you have to meet in order to participate in this study.

What to expect in this clinical trial

SCREENING:

Potential participants will be evaluated to see if they qualify for the study for up to 28 days.
RANDOMIZATION:

Eligible participants will be randomly assigned to one of three study treatment groups.
RUN-IN PERIOD:

At the first study treatment visit, participants will receive only the certepetide or placebo component of the assigned treatment.
TREATMENT:

Treatment cycles will begin after the run-in period. Participants will visit the clinic 3 times every 28 days for treatment and tests. Participants will also have CT or MRI scans every 8 weeks while on treatment.

Important Note: This is a clinical trial, which means the purpose is to gather data about the safety and effectiveness of a new drug.
Your participation is voluntary, and you can withdraw from the study at any time.

Frequently asked questions

As with any investigational drug, there may be side effects that are not yet known. Potential risks include:

Gastrointestinal bleeding, which has been noted in subjects with pancreatic ductal adenocarcinoma

The most common side effects that have been seen in patients with pancreatic cancer who received certepetide in combination with gemcitabine and nab-paclitaxel chemotherapy regardless of its dose and its causal relationship included:

Low levels of white blood cells (neutropenia), which can make you more susceptible to infection
Low levels of red blood cells (anemia), which can make you overly tired
Loss of sensation or weakness of peripheral nerves (nerves located outside of the brain and spinal cord)

Additional side effects seen in patients treated with both certepetide and chemotherapy (gemcitabine and nab paclitaxel) include:

Fatigue (feeling tired)
Nausea (feeling sick to your stomach)
Constipation
Diarrhea
Hair loss
Swelling (peripheral edema)
Abdominal pain
Vomiting
Change in taste
Loss of appetite
Breathlessness (dyspnea)
Rash
Cough
Low platelets (thrombocytopenia), which can lead to excessive bruising
Nosebleeds (epistaxis)

These adverse events (side effects) are commonly seen with gemcitabine and nab-paclitaxel when given without certepetide. No new or worsened side effects were observed in patients when certepetide was given in combination with gemcitabine and nab-paclitaxel than those observed in patients who received chemotherapy alone.

Allergies to medications like certepetide are considered unlikely, but there is still the possibility of an allergic reaction. A severe allergic reaction could be life-threatening. Please seek treatment immediately and tell the study doctor and study staff if you have any of these symptoms. An allergic reaction could include:

Rash or hives
Difficulty breathing or wheezing when you breathe
Sudden changes in blood pressure that may make you feel dizzy or lightheaded
Swelling around the mouth, throat, or eyes
Fast pulse
Sweating

Although very rare in patients with cancer, when many tumor cells are killed rapidly, the blood can develop high levels of uric acid, potassium, and phosphorus, and low calcium levels, which can cause kidney damage. Symptoms include pain, discomfort, or burning when urinating, low urine output, blood in the urine, dull ache on the side of the upper back, loss of appetite, vomiting, cramps, and weakness. This possible side effect, called tumor lysis syndrome, is usually seen within a few days after starting cancer treatment. Subjects will be monitored, and supportive care provided if abnormal laboratory results or symptoms are observed. Tumor lysis syndrome can be life-threatening or even fatal.

Based on animal studies, there is a possibility that certepetide might cause small changes in the testes and/or the tubes that connect the testes to the urethra (epididymis), however this has not been seen in human subjects to date.

If you experience any of the above signs or symptoms, you should notify your doctor immediately, as you may need immediate treatment and hospitalization. Your study doctor may give you drugs to treat these symptoms.