Clinical trial: NCT01967576
Section: Alternatives
Subsections: Alternative Approaches Or Treatments What Other Choices Do I Have If I Do Not Take Part In This Study?

Instead of being in this study, you have these options Getting treatment or care for your cancer without being on a study Taking part in another study Getting no treatment at all Getting comfort care, also called palliative care. This type of care helps reduce pain, tiredness, appetite problems and other problems caused by the cancer. It does not treat the cancer directly. Instead, it tries to improve how you feel. Comfort care tries to keep you as active and comfortable as possible.
Please talk to your doctor about these and other options.
Clinical trial: NCT01967576
Section: Confidentiality
Subsections: Will Your Medical Information Be Kept Private?|Certificate Of Confidentiality

We will do our best to make sure that the personal information in your medical record will be kept private. However, we cannot guarantee total privacy. Organizations that may look at and/or copy your medical records for research, quality assurance, and data analysis include:  
The National Cancer Institute (NCI) and other government agencies, like the Food and Drug Administration (FDA), which are involved in keeping research safe for people. National Cancer Institute Institutional Review Board The study Sponsor, Dr. Tito Fojo or his representatives  
PATIENT IDENTIFICATION **CONTINUATION SHEET for either:**  
NIH-2514-1 (07-09)  
NIH-2514-2 (10-84)  
P.A.: 09-25-0099  
File in Section 4: Protocol Consent  
STUDY NUMBER: 14-C-0001 CONTINUATION: page 7 of 11 pages  
Qualified representatives from Pfizer, the pharmaceutical company who produces axitinib.
A description of this clinical trial will be available on http://www.Clinicaltrials.gov, as required by U.S. Law. This Web site will not include information that can identify you. At most the Web site will include a summary of the results. You can search this Web site at any time.  
Stopping Therapy Your doctor may decide to stop your therapy for the following reasons:
if he/she believes that it is in your best interest if the cancer grows or becomes worse during treatment if you have side effects from the treatment that your doctor thinks are too severe if new information shows that another treatment would be better for you In this case, you will be informed of the reason therapy is being stopped.
You can stop taking part in the study at any time. However, if you decide to stop taking part in the study, we would like you to talk to the study doctor and your regular doctor first. If you decide at any time to withdraw your consent to participate in the trial, we will not collect any additional medical information about you. However, according to FDA guidelines, information collected on you up to that point may still be provided to Pfizer or designated representatives. If you withdraw your consent and leave the trial, any samples of yours that have been obtained for the study and stored at the NCI can be destroyed upon request. However, any samples and data generated from the samples that have already been distributed to other researchers or placed in the research databases cannot **be recalled and destroyed.**

To help us protect your privacy, we have obtained a Certificate of Confidentiality. The researchers can use this Certificate to legally refuse to disclose information that may identify you in any federal, state, or local civil, criminal, administrative, legislative, or other proceedings, for example, if there is a court subpoena. The researchers will use the Certificate to resist any demands for information that would identify you, except as explained below. You should also know that there are several circumstances in which the Certificate does not provide coverage. These include when information:  
will be used for auditing or program evaluation internally by the NIH; or must be disclosed to meet the legal requirements of the federal Food and Drug Administration (FDA). is necessary for your medical treatment and you have consented to this disclosure; is for other research.  
PATIENT IDENTIFICATION **CONTINUATION SHEET for either:**  
NIH-2514-1 (07-09)  
NIH-2514-2 (10-84)  
P.A.: 09-25-0099  
File in Section 4: Protocol Consent  
STUDY NUMBER: 14-C-0001 CONTINUATION: page 8 of 11 pages In addition, identifiable, sensitive information protected by this Certificate cannot be admissible as evidence or used for any purpose in any action, suit, or proceeding without your consent. You should understand that a Certificate of Confidentiality does not prevent you or a member of your family from voluntarily releasing information about yourself or your involvement in this research. If an insurer, employer, or other person obtains your written consent to receive research information, then the researchers will not use the Certificate to withhold that information. The Certificate of Confidentiality will not protect against the required reporting by hospital staff of information on suspected child abuse, reportable communicable diseases, and/or possible threat of harm to self or others.
Clinical trial: NCT01967576
Section: Duration of Study Involvement
Subsections: When You Are Finished Taking The Drugs

You will be invited to clinical center approximately 4 weeks following the last dose of study drug. The following assessments will be performed at this visit:  
Medical history and physical exam Routine blood and urine tests 24-hour urine test Review of your side effects
If you are unable to travel to the NIH, we will contact you by telephone and ask you about any side effects you might have and any new treatments you may have started.
Clinical trial: NCT01967576
Section: Participant's Rights
Subsections: Research Subject'S Rights What Are The Costs Of Taking Part In This Study?

If you choose to take part in the study, the following will apply, in keeping with the NIH policy:
You will receive study treatment at no charge to you. This may include surgery, medicines, laboratory testing, x-rays or scans done at the Clinical Center, National Institutes of Health (NIH), or arranged for you by the research team to be done outside the Clinical Center, NIH if the study related treatment is not available at the NIH.  
There are limited funds available to cover the cost of some tests and procedures performed outside the Clinical Center, NIH. You may have to pay for these costs if they are not covered by your insurance company. Medicines that are not part of the study treatment will not be provided or paid for by the Clinical Center, NIH.  
Once you have completed taking part in the study, medical care will no longer be provided by the Clinical Center, NIH.
Clinical trial: NCT01967576
Section: Possible Risks, Discomforts, and Inconveniences
Subsections: Risks Or Discomforts Of Participation What Side Effects Or Risks Can I Expect From Being In This Study?|Potential Benefits Of Participation Are There Benefits To Taking Part In This Study?

You may have side effects while on the study. Everyone taking part in the study will be watched carefully for any side effects. Side effects can be mild or serious. Your health care team may give you medicines to help lessen side effects. Many side effects go away soon after you stop taking the drug. In some cases, side effects can be serious, long lasting, or may never go away, and can even result in death.  
You should talk to your study doctor about any side effects that you have while taking part in the study. Likely side effects are those seen in up to one third of patients who received single agent axitinib in earlier trials.  
| Risks and side effects of axitinib may include:  Likely Less Likely   | Rare but Serious                                                                                                             |                                                                                                                                                                                     |
|-----------------------------------------------------------------------|------------------------------------------------------------------------------------------------------------------------------|-------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|
| - Fatigue                                                             | - Hoarseness of voice                                                                                                        | - Decrease in the white blood  cells (the cells that fight  infection) and can result in  infection that may or may not  require treatment with  antibiotics                        |
| - Diarrhea                                                            | - Shortness of breath                                                                                                        |                                                                                                                                                                                     |
| - Hypertension                                                        | - Weight loss                                                                                                                |                                                                                                                                                                                     |
| - Nausea                                                              | - Constipation                                                                                                               |                                                                                                                                                                                     |
| - Anorexia                                                            | - Headache                                                                                                                   | - Decrease in red blood cells  (the cells that carry oxygen to  your body), which can cause  tiredness and shortness of  breath and may required a  transfusion of red blood cells) |
| - Vomiting  - Cough  - Leg pain  - Gastroesophageal reflux  disease   | - Decrease in platelets (the cells  that help with blood clotting)  which can increase the risk of  bleeding or bruising     |                                                                                                                                                                                     |
| - Mouth soars  - Abdominal pain  - Back pain                          | - Loss of protein in urine                                                                                                   |                                                                                                                                                                                     |
| - Insomnia                                                            | - Intestinal perforations                                                                                                    |                                                                                                                                                                                     |
| - Leg swelling                                                        | - Stroke                                                                                                                     |                                                                                                                                                                                     |
| - Rash                                                                | - Thrombosis ( Blood clots)                                                                                                  |                                                                                                                                                                                     |
| - Dizziness                                                           | - Myocardial Infarction ( heart                                                                                              |                                                                                                                                                                                     |
| PATIENT IDENTIFICATION                                                | CONTINUATION SHEET for either: NIH-2514-1 (07-09)  NIH-2514-2 (10-84)  P.A.: 09-25-0099  File in Section 4: Protocol Consent |                                                                                                                                                                                     |  
STUDY NUMBER: 14-C-0001 CONTINUATION: page 5 of 11 pages  
| attacks)                                                                      |                                                                                                                                                                                                                                                                                                                                      |
|-------------------------------------------------------------------------------|--------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------|
| - Muscle pains                                                                | - Arrhythmias (Heart rate or  rhythm abnormalities)                                                                                                                                                                                                                                                                                  |
| - Thyroid abnormalities  - Increased blood glucose                            | - Abnormal kidney function or  failure                                                                                                                                                                                                                                                                                               |
| - Electrolyte abnormalities  - Low blood pressures                            | - Liver function abnormality or  failure                                                                                                                                                                                                                                                                                             |
| - Skin peeling from hands and  feet also known as the hand  and foot syndrome | - Reversible posterior  leukoencephalopathy, a disorder  associated with findings on  MRI. The symptoms include  headache, high blood pressure,  decreased alertness, change in  mental functioning, seizures,  and vision loss, including  blindness. These symptoms are  usually reversible.  - Elevated levels of  triglycerides. |  
Since the drug is still being studied there may be other side effects that have not been described before. If you are pregnant you will not be permitted to participate in this research study. If you are breast feeding and the protocol involves injection of radioactive material you will not be permitted to participate. It is best to avoid radiation exposure to unborn or nursing infants since they are more sensitive to radiation than adults.

The aim of this study is to see if this experimental treatment will cause your tumors to shrink. We do not know if you will receive personal, medical benefit from taking part in this study. These potential benefits could include shrinking of your tumor or lessening of your symptoms, such as pain, that are caused by the cancer. Because there is not much information about the drug's effect on your cancer, we do not know if you will benefit from taking part in this study, although the knowledge gained from this study may help others in the future who have cancer.  
PATIENT IDENTIFICATION **CONTINUATION SHEET for either:**  
NIH-2514-1 (07-09)  
NIH-2514-2 (10-84)  
P.A.: 09-25-0099  
File in Section 4: Protocol Consent  
STUDY NUMBER: 14-C-0001 CONTINUATION: page 6 of 11 pages
Clinical trial: NCT01967576
Section: Procedures
Subsections: What Will Happen If You Take Part In This Research Study? Evaluation Tests Before The Study Begins|What Does This Study Involve? During The Study

Prior to starting (or "enrolling") on the study, an evaluation will be done at the NIH Oncology Clinic to determine if this study is appropriate for you. This is called "screening" and a different consent form explains the tests required for screening. All cancer diagnoses must be confirmed by a pathologist at the NIH, who will look at the specimen slides from your prior biopsy or surgery. If a pathology specimen is not available from previous surgery, a pathology report from a prior biopsy or surgery confirming your diagnosis will be accepted. Results from an imaging study that would only be positive in an adult patient with your diagnosis may also be accepted.

If you enroll in the study you will need the following tests and procedures as part of the study. Many of these are part of regular cancer care. They are being done more often because you are in this study.  
- Blood and urine tests will be done once every 4 weeks during the first 3 months, and then once every 12 weeks after the first 3 months - Blood tests for research-only purposes will be done at the beginning of the first cycle and then at 8, 16 and 24 weeks. - You will check your blood pressure twice a day during the first 4 weeks, then once a  
PATIENT IDENTIFICATION **CONTINUATION SHEET for either:**  
NIH-2514-1 (07-09)  
NIH-2514-2 (10-84)  
P.A.: 09-25-0099  
File in Section 4: Protocol Consent  
STUDY NUMBER: 14-C-0001 CONTINUATION: page 3 of 11 pages week after the first 4 weeks. You will have a diary to write down your blood pressure readings.  
- You will have a CT and/or MRI scan every 12 weeks as part of this study. The scan compares the baseline scan (before you begin axitinib) to the scan after receiving axitinib, in order to figure out if the tumor(s) are the same size, smaller, or larger. You will need to have the scans in order to participate in the study.
Clinical trial: NCT01967576
Section: Purpose of Research
Subsections: Why Is This Study Being Done?|Why Are You Being Asked To Take Part In This Study?|Description Of Research Study

Axitinib (AG-013736) is an investigational (experimental) drug that is being studied in patients with metastatic pheochromocytoma/paraganglioma. This drug was approved by the FDA in 2012 for the treatment of kidney cancer. Although it is FDA-approved, it is still considered experimental in this study because it has never been studied in people with pheochromocytoma/paraganglioma. The main goal of this study is to find out if axitinib is able to stop tumors from growing, or shrink tumors in patients with this type of cancer.  
PATIENT IDENTIFICATION **CONSENT TO PARTICIPATE IN A CLINICAL RESEARCH STUDY**  
- Adult Patient or - **Parent, for Minor Patient** NIH-2514-1 (07-09)  
P.A.: 09-25-0099 File in Section 4: Protocol Consent (1)  
STUDY NUMBER: 14-C-0001 CONTINUATION: page 2 of 11 pages

You are being asked to be a part of this study because you have a diagnosis of metastatic pheochromocytoma/paraganglioma and have tried at least one treatment for the disease that either did not work or is no longer working. Taking part in this study may or may not make your health better. While doctors hope axitinib will be useful against your cancer, there is no proof of this yet. We do know that the information from this study will help doctors learn more about axitinib as a treatment for cancer and could help future cancer patients.

You will receive the study drug, axitinib, orally twice a day, every day, in 28-day cycles. You will start at a dose of 5mg twice a day. After 4 weeks, your dose may be increased to 7mg twice a day and later on to 10mg twice a day after you are carefully checked by your study team and are feeling good without any significant side effects. You may take axitinib with or without food.