A Pilot Study of a Personalized Neoantigen Cancer Vaccine With and Without Low-Dose Cyclophosphamide in Treatment Naïve, Asymptomatic Patients With IGHV Unmutated Chronic Lymphocytic Leukemia.
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|ClinicalTrials.gov Identifier: NCT03219450|
Recruitment Status : Not yet recruiting
First Posted : July 17, 2017
Last Update Posted : June 3, 2019
This research study is studying a novel type of CLL vaccine as a possible treatment for chronic lymphocytic leukemia (CLL)
The names of the study interventions involved in this study are:
- Personalized NeoAntigen Vaccine
|Condition or disease||Intervention/treatment||Phase|
|Lymphocytic Leukemia||Drug: NeoVax Drug: Cyclophosphamide||Phase 1|
This research study evaluates the feasibility and tolerability of an investigational intervention and also tries to define the appropriate dose of the investigational intervention to use for further studies. "Investigational" means that the intervention is being studied and that research doctors are trying to find more about it. It also means that the FDA (US Food and Drug Administration) has not approved the Personalized Neoantigen Cancer Vaccine for any use in patients, including people with CLL.
This is the first time NeoVax vaccine will be given in combination with cyclophosphamide in humans.
The purpose of this study is to determine if it is possible to make and safely administer a vaccine against CLL. The investigators plan to analyze the specific genetic characteristics (mutations) of the participant's own CLL and use that information to produce proteins that may help the immune system recognize and fight CLL cells.
This vaccine is also being tested in clinical trials in patients with advanced melanoma (a type of skin cancer) or glioblastoma (a type of brain cancer). The current study will examine the ability of the vaccine to stimulate the immune system when given at several different timepoints, and will examine the participant blood cells for signs that the CLL has changed or decreased.
CLL cells will be obtained from bone marrow biopsy and blood draws. The genetic material contained in the CLL cells will be examined for the presence of tumor-specific mutations. This information will be used to prepare small protein fragments, which are called "peptides." The vaccine will consist of up to 20 of these peptides as well as a drug called Poly-ICLC. A peptide from the tetanus vaccine will also be included to boost the immune response. Half of the patients, enrolled in the second half of this trial, will also receive low doses of a chemotherapy drug called cyclophosphamide.
Poly-ICLC (also called Hiltonol) is an experimental "viral mimic" and an activator of immunity. Poly-ICLC binds proteins on the surface of certain immune cells to make it appear as if a virus is present. When the cells detect the vaccine, they think it is a virus and turn on the immune system. Poly-ICLC will be mixed with NeoAntigen peptides and administered as an injection given underneath the skin. Poly-ICLC is an investigational drug, meaning the FDA has not approved it as a treatment for any disease.
Cyclophosphamide (also called Cytoxan) is a chemotherapy drug used to treat many cancers, including CLL. At the much lower dose used in this study, it is an investigational drug to help the immune cells to be better at attacking cancer cells.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of a Personalized Neoantigen Cancer Vaccine With and Without Low-Dose Cyclophosphamide in Treatment Naïve, Asymptomatic Patients With IGHV Unmutated Chronic Lymphocytic Leukemia.|
|Estimated Study Start Date :||September 1, 2019|
|Estimated Primary Completion Date :||September 30, 2020|
|Estimated Study Completion Date :||September 30, 2023|
It stimulates the immune system to attack cancer cells.
Experimental: Neovax + Low-dose cyclophosphamide
It stimulates the immune system to attack cancer cells.
It is a chemotherapy drug used to treat many cancers. At low doses, it is an investigational drug to help the immune cells to be better at attacking cancer cells while avoiding chemotherapy toxicity.
Other Name: Cytoxan
- The proportion of all enrolled patients for whom sequencing and analysis leads to identification of at least 7 actionable peptides to initiate vaccine production [ Time Frame: 2 years ]
- The proportion for whom the time from sample collection to vaccine availability is less than 12 weeks [ Time Frame: 12 weeks ]
- The number of patients with treatment-limiting toxicities [ Time Frame: 2years ]
- The proportion of participants who achieve an IFN-γ T cell response to a peptide pool [ Time Frame: 2 years ]
- The proportion of participants who meet 2008 IW-CLL criteria for clinical response at 2 months and/or have not required therapy by 24 months after vaccine administration [ Time Frame: 2 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03219450
|Contact: Pavan Bachireddy, MDfirstname.lastname@example.org|
|United States, Massachusetts|
|Dana Farber Cancer Institute|
|Boston, Massachusetts, United States, 02215|
|Contact: Pavan Bachireddy, MD 617-632-6091 email@example.com|
|Principal Investigator: Pavan Bachireddy, MD|
|Principal Investigator:||Pavan Bachireddy, MD||Dana-Farber Cancer Institute|