Different types of people were included in the AZEDRA clinical trial
The clinical trial for AZEDRA was the largest one of its kind for unresectable, locally advanced, or metastatic pheochromocytoma and paraganglioma. Participants in the clinical trial included:
- Both men and women
- People between the ages of 16–76 (the study was open to people 12 or older)
- People who had received previous treatments, including:
- Surgery
- Chemotherapy
- Other radiation therapies
What the AZEDRA clinical trial measured
Researchers designed the AZEDRA clinical trial to measure two outcomes that reflect the dual treatment goals for treating advanced pheochromocytoma and paraganglioma:


Reduce the need for hypertension medication
Many people living with pheochromocytoma and paraganglioma must take daily medication to control hypertension caused by tumor-released hormones, which is one of the most common symptoms of this disease. The AZEDRA clinical trial measured reduction in use of hypertension medication over time.
Control the growth of tumors
Tumor growth is the leading cause of death for people with advanced pheochromocytoma and paraganglioma. The AZEDRA clinical trial measured the number of tumors that stopped growing and how much they shrank after AZEDRA treatment.
There’s a lot to understand about the AZEDRA clinical trial. Find definitions for words in our Glossary and answers to common questions in our Frequently Asked Questions.
Results of the AZEDRA clinical trial
AZEDRA was proven to reduce the need for hypertension medication
The AZEDRA clinical trial measured significant antihypertensive medication reduction, which means the reduction was substantial and sustained over time.
Substantial reduction

50% or more reduction in a patient’s use of hypertension medication
Sustained reduction

or longer reduction in a patient's use of hypertension medication
25%
of patients who received at least one dose of AZEDRA had significant and sustained medication reduction
(17 out of 68 patients)


For people with significant medication reduction, the reduction lasted anywhere from 8 months to 5 years. Most of these people experienced sustained medication reduction for more than 1 year.
AZEDRA reduced the size of tumors
In the AZEDRA clinical trial, patients experienced fast and prolonged tumor reduction.

22% of patients had pheochromocytoma and paraganglioma tumors that significantly decreased in size in the year following treatment (15 out of 68 patients)
For patients with a significant decrease in tumor size, over half (53%) experienced durable tumor reduction for

or longer
Understanding the side effects of AZEDRA
The side effects of AZEDRA are based on the experiences of people in the clinical trial. The most common side effects were*:
Myelosupression
- Lymphopenia—A decrease in the number of lymphocytes, a class of white blood cell that helps fight infections
- Neutropenia—A decrease in the number of neutrophils, another class of white blood cell that helps fight infections
- Thrombocytopenia—A decrease in the number of platelets, which are cellular structures involved in blood clotting
- Anemia—A decrease in the levels of hemoglobin, an iron-bearing protein found in red blood cells
Cardiovascular
- Increased international normalized ratio–A measure of how quickly blood coagulates or solidifies; when increased, blood may clot more slowly
- Hypertension—Increase in blood pressure to 140/90 or higher
Gastrointestinal
- Nausea
- Vomiting
General
- Dizziness
- Fatigue
*Experienced by more than 10% of patients
Be sure to tell your doctor about any symptoms you experience, even if they are not listed above.
Your treatment team may ask you to take certain steps to help avoid some of these side effects, including:

Taking medications to help reduce nausea and to block radiation from affecting healthy organs before treatment

Drinking lots of water before and after treatment
AZEDRA treatment involves two steps
Step 1. Dosimetry, to confirm the correct dose

The dosimetry step involves an injection of a small amount of AZEDRA. After the injection, doctors will schedule three body scans over the next 5 days. These scans allow doctors to confirm the correct dose of AZEDRA for you.

Step 2. Therapy, to treat tumors

The therapy step involves an infusion of the treatment dose of AZEDRA, lasting about 30 minutes, followed by a few days in the hospital. You will receive another treatment dose about 3 months after the first one. AZEDRA will continue to treat pheochromocytoma and paraganglioma tumors even after you leave the hospital. When you leave the hospital, you will receive additional instructions about reducing radiation exposure to others. The radiation will eventually go away completely. Until then, you may need to minimize contact with other people.

What to expect after AZEDRA treatment
Within 3 months
-
- Doctors will monitor you frequently. During this period of time, AZEDRA will still be working and you may continue to feel side effects. Tell your doctor if you have any side effects that bother you or do not go away.
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- Do not breastfeed during treatment with AZEDRA and for 80 days after your final dose.
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- The second dose of AZEDRA will be scheduled after approximately 90 days.
Within 12 months
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- Doctors will continue to monitor you.
-
-
If you or your partner could become pregnant, use effective contraception during treatment with AZEDRA, and for 4 months for males and 7 months for females after your
final dose.
After 1 year
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Long-term
monitoring for:- Any long-term radiation effects
- Pheochromocytoma and paraganglioma tumor reduction or disease progression
- Signs and symptoms of an underactive thyroid