CLL Clinical Trial FAQs

Chronic Lymphocytic Leukemia (CLL) Clinical Trial FAQs

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What is the treatment under investigation in this study?

The treatment being investigated is known as MyVax® Personalized Immunotherapy. MyVax® Personalized Immunotherapy uses a person’s own immune system (thus, the term immunotherapy) in an attempt to combat disease. MyVax® Personalized Immunotherapy is called personalized immunotherapy because it is developed from an idiotype—or Id protein, which is identified from the patient’s tumor cells and is unique to that patient’s tumor cells. In fact, the Id can be thought of as a tumor cell “fingerprint.” MyVax® Personalized Immunotherapy is different from currently available therapies in that it is made individually and specifically for each patient. Because it has been custom-designed to work with each patient’s specific immune system and to target each patient’s particular tumor cells, healthy cells within the patient’s body should not be affected.

MyVax® Personalized Immunotherapy is composed of (1) the patient- and tumor-specific idiotype (Id) protein, and (2) a second protein called keyhole limpet hemocyanin (KLH). KLH comes from a giant sea snail that lives off the coast of California. This protein is highly immunogenic, meaning that when it is put into the body, the immune system responds strongly to it—and to anything attached to it. In addition, an adjuvant (a substance that enhances the immune response) is administered along with MyVax® Personalized Immunotherapy. This adjuvant is called granulocyte-macrophage colony-stimulating factor (GM-CSF).

Patient- and tumor-specific personalized immunotherapy such as MyVax® Personalized Immunotherapy may also be referred to as active idiotype immunotherapy, idiotype (Id) vaccines, or therapeutic idiotype vaccines. These are simply different names for the same therapeutic approach.

How is the treatment manufactured?

The first step in the manufacturing of MyVax® Personalized Immunotherapy is the collection of a tissue or blood sample from the patient. From that sample, the unique genetic material associated with the specific idiotype (Id) that the person’s cancer is expressing (producing) is identified and isolated. This genetic material is then used to produce a recombinant, or genetically engineered, version of the Id protein, which is then grown in the laboratory, purified, and combined with KLH.

In a small number of cases, it may not be possible to produce a personalized immunotherapy for a specific patient due to the biology of his/her cancer cells (ie, the cancer cells may not express an Id protein or may not produce enough of the Id protein).

What is the rationale for testing this treatment in patients with B-cell chronic lymphocytic leukemia (CLL)?

Idiotype vaccines were first studied in clinical trials conducted by Stanford University Medical Center and the National Cancer Institute. Genitope Corporation’s lead product candidate, MyVax® Personalized Immunotherapy, has been studied in phase 2 trials in patients with B-cell non-Hodgkin’s lymphoma (NHL) and is currently being evaluated in a phase 3 study in patients with follicular lymphoma. Encouraging efficacy results and a favorable safety profile have been seen to date in these studies. Since CLL is another type of B-cell–related malignancy, it is thought that MyVax® Personalized Immunotherapy may be helpful in this patient population.

What is this study designed to measure?

This study is a phase 1/2 clinical trial designed to evaluate both immune and clinical responses to MyVax® Personalized Immunotherapy, as well as its safety and tolerability. The study will also evaluate whether or not treatment can help avoid or delay the need for chemotherapy. In addition, lymphocyte counts in the patient’s peripheral blood will be measured.

Who is eligible for this study?

To be eligible for the study, a patient must meet all of the following criteria:

  • Diagnosis of B-cell CLL, with Rai stage 0, I, or II. Patients diagnosed with small lymphocytic lymphoma (SLL) are also eligible, as the latest World Health Organization classifications regard CLL and SLL as one disease.
  • No previous anti-leukemic or anti-lymphoma treatment(s)
  • 18 years of age or older
  • Able to “watch and wait” (that is, not receive therapy) for up to 8 months after entry into the study. (For reasons having to do with the biology of a patient’s tumor cells, it may take up to 8 months to manufacture a patient’s personalized immunotherapy)
  • No prior malignancy (except basal or squamous cell carcinoma or cervical carcinoma in situ)
  • Not pregnant or breast feeding
  • Not have an autoimmune disease (including previously treated autoimmune hemolytic anemia or immune thrombocytopenia purpura)
  • Not currently receiving any immunosuppressive therapy, steroids (topical, inhaled, or oral), or chemotherapy
  • Not currently participating in any other therapeutic clinical study
Can a patient with an advanced stage/aggressive case of CLL participate in this study?

No. A patient’s CLL must be classified as Rai stage 0, I, or II.

Where is this study being conducted?

This study is being conducted at multiple sites throughout the United States and Canada. For a current listing of participating centers, call 1.866.GENITOP (1.866.436.4867) or visit www.genitope.com.

What is the time commitment for patients participating in this study?

Once a patient is accepted into the study, there is a “watch and wait” period of approximately 8 months (this time is needed to manufacture the individual patient’s unique MyVax® Personalized Immunotherapy). After the “watch and wait” period, 16 MyVax® Personalized Immunotherapy immunizations will be administered over the course of 1 year—the first 5 at 2-week intervals, and the remaining 11 at 4-week intervals. The immunization period is followed by a 1-year follow-up period, during which study participants are seen every 3 months. The total time the participant will be in the study is approximately 2 years 8 months. After finishing the study, patients or their doctors will be contacted every 6 months to collect information about subsequent anti-leukemic therapy and survival.

What is the cost to participate in this study? What costs are covered by medical insurance?

MyVax® Personalized Immunotherapy and GM-CSF are provided at no cost to patients participating in this study. However, since other study-related visits and procedures are considered standard of care, these medical costs will be billed to the patient’s insurance. Some study sites provide study participants with reimbursement for travel-related costs, and study participants may want to check with their study site to see if reimbursement is available.

Does everyone who participates in this study receive the treatment?

Yes, all eligible study participants will receive MyVax® Personalized Immunotherapy. In other words, there is no placebo group in this study.

What happens if a patient’s cancer progresses (gets worse) before he/she begins treatment (that is, during the “watch and wait” period)?

If during the “watch and wait” period a patient’s cancer progresses to the point where treatment is required to control the disease, that patient will no longer be eligible for participation in this study and will not be able to receive MyVax® Personalized Immunotherapy.

What happens if a patient’s cancer progresses (gets worse) during the immunization period?

As long as other treatments are not required to control the disease, the patient can continue to participate in this study. However, if a patient’s physician determines that other treatment(s) is necessary, that patient will no longer be eligible for participation in this study and will not be able to receive any further immunizations of MyVax® Personalized Immunotherapy.

Can study participants receive other immunizations, such as flu shots, during the study?

If the physician responsible for the study determines that such immunizations are medically necessary for a patient, it may be possible for patients to receive them.

Can study participants receive supportive therapies such as Procrit® (epoetin alfa) or Neumega® (oprelvekin) during the study?

While the use of Procrit or Neumega is not specifically prohibited, if a patient were to require such supportive therapy, his/her cancer would probably have progressed to the point where other anti-leukemic therapy(ies) would be necessary. In this case, he/she would no longer be eligible for participation in this study and would not be able to receive any further immunizations of MyVax® Personalized Immunotherapy.

How is the treatment administered?

MyVax® Personalized Immunotherapy is given as a series of injections just under the skin (subcutaneously) via a small needle. Each MyVax® Personalized Immunotherapy injection is given along with an injection of GM-CSF. This is followed by a 3-day series of once-daily GM-CSF injections for the first 8 doses of MyVax® Personalized Immunotherapy. The GM-CSF can be self-administered at home.

What physical reactions should patients expect as a result of the treatment?

Adverse events that patients receiving MyVax® Personalized Immunotherapy have reported include injection-site reactions, such as redness, swelling, bruising, itching, soreness, and/or pain at the site of the injection. Some patients also experienced flu-like symptoms, including fever, chills, nausea, and muscle soreness. These adverse events generally occur in the first few days after the injection, but they may last longer.

Side effects associated with the adjuvant (GM-CSF) include fever, bone or joint pain, flu-like symptoms (nausea, headache, tiredness), and mild skin reactions at the injection site. More information about GM-CSF side effects can be found at www.leukine.com.

As with any medication, there is a possibility of allergic reactions with MyVax® Personalized Immunotherapy. Allergic reactions have been seen in only a small number of patients, and patients are monitored closely after each injection for signs of such reactions. There are effective medications available to counteract allergic reactions, should they be required.

Is there a risk of death, secondary cancers, or autoimmune disorders associated with the treatment?

In studies where MyVax® Personalized Immunotherapy has been used to treat patients with NHL, there have been no treatment-related deaths. There have been a few reports of new cancers or autoimmune disorders, but it is difficult to determine if these were related to the use of MyVax® Personalized Immunotherapy, because these patients also received other medications, such as chemotherapy. Monitoring for these risks in patients in the NHL study is ongoing, and these types of risks will also be monitored in this study.

Will bone marrow biopsies be required during the study? How about CT scans?

Bone marrow biopsies are required prior to receiving MyVax® Personalized Immunotherapy. In patients with a complete response to MyVax® Personalized Immunotherapy, a bone marrow biopsy will be repeated at 3, 6, and/or 12 months after all immunizations have been received. CT scans are required prior to receiving MyVax® Personalized Immunotherapy, and at 3, 6, and 12 months after all immunizations have been received.

How many patients will be treated in this study?

It is anticipated that 40 patients will receive MyVax® Personalized Immunotherapy.

Will the results of this study be published?

Yes, study results will be published once the trial is completed.