Axitinib for Pheochromocytoma

Phase-Based Estimates
Columbia University Medical Center, New York, NY
Pheochromocytoma+2 More
Axitinib - Drug
All Sexes
Eligible conditions

Study Summary

Study of Axitinib (AG-013736) With Evaluation of the VEGF-pathway in Pheochromocytoma/Paraganglioma

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Eligible Conditions

  • Pheochromocytoma
  • Paraganglioma
  • Carotid Body Tumor

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether Axitinib will improve 1 primary outcome and 1 secondary outcome in patients with Pheochromocytoma. Measurement will happen over the course of Up to 16 weeks.

Month 12
Progression-free survival
Up to 16 weeks
Response Rate (RR)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Side Effects for

This histogram enumerates side effects from a completed 2019 Phase 2 trial (NCT02129647) in the Axitinib ARM group. Side effects include: Mucositis with 30%, nausea with 10%, Rash with 10%, Fatigue with 10%.

Trial Design

2 Treatment Groups

Axitinib (AG-013736)

This trial requires 25 total participants across 2 different treatment groups

This trial involves 2 different treatments. Axitinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Axitinib (AG-013736)
Subjects with Recurrent or Primary Unresectable Pheochromocytoma/Paraganglioma will receive 16 weeks of therapy (Axitinib), and be seen in clinic every 4 weeks to monitor therapy.
ControlNo treatment in the control group
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: duration of time from start of treatment to time of progression or death, whichever occurs first; an average of up to 12 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly duration of time from start of treatment to time of progression or death, whichever occurs first; an average of up to 12 months for reporting.

Who is running the study

Principal Investigator
A. F.
Prof. Antonio Fojo, Professor of Medicine at the Columbia University Medical Center, Dept of Med Hematology & Onc
Columbia University

Closest Location

Columbia University Medical Center - New York, NY

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Imaging confirmation of metastatic disease
An outside pathology report confirms the diagnosis of Pheo/PGL, AND the patient has nuclear medicine imaging studies that would only be positive in an adult patient with a diagnosis of Pheo/PGL (F-DOPA, Dotatate, F-Dopamine or MIBG)
Last radiotherapy treatment ≥ 4 weeks prior to starting treatment with this protocol and there must be sites of measurable disease that did not receive radiation
Measurable disease at the time of enrollment as per RECIST 1.1.
A life expectancy of at least 3 months and ECOG performance status ≤ 2
Age ≥ 18 years
Information available or pending regarding possible genetic alterations that can explain the patient's pheochromocytoma/paraganglioma (mutations in SDHB, SDHV or VHL genes)
Last dose of chemotherapy or experimental therapy more than 4 weeks (6 weeks in the case of nitrosourea) prior to enrollment date; 2 weeks if the last therapy was received as part of a "phase 0" or "exploratory IND" trial. Last surgery more than 4 weeks prior to enrollment, to allow for wound healing. Core biopsies or FNA will not require any waiting period
Prior therapeutic MIBG is allowed
Adults with a confirmed pathologic diagnosis of pheochromocytoma/paraganglioma by a CUMC/NYPH laboratory when such tissue is available to confirm.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of pheochromocytoma?

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Symptoms occur in nearly all cases of pheochromocytoma, and may include orthostatic syncope, headache, palpitation and sweating. Symptoms of adrenal hormone secretion may present similarly. When there is an abnormal adrenergic response to exercise, it may be difficult to distinguish pheochromocytoma from catecholamine secreting tumors such as sympathetic adenoma. Many different symptoms may occur in pheochromocytoma.

Unverified Answer

What are common treatments for pheochromocytoma?

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Treatment of symptomatic and incidentally detected tumors is more frequently resection, adjuvant radiotherapy, transcatheter arterial chemoembolization, or embolization. These techniques may be used alone or in combination with surgery. Other treatment options exist, but the optimal therapeutic modality needs to be determined using randomized controlled trials.

Unverified Answer

What causes pheochromocytoma?

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The cause of pheochromocytoma remains unknown; however, it is generally diagnosed by clinical examination, the discovery of catecholamines from a biopsy or the use of tests such as CT scans or 123I-MIBG (123I metaiodobenzylguanidine) scintigraphy. If pheochromocytoma is associated with signs and symptoms of high blood pressure (hypertension), further testing can be performed to exclude the presence of a tumor.

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Can pheochromocytoma be cured?

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As with other endocrine disorders, many patients have a long and healthy life after surgery and medical or physical therapy. Although surgery should always be recommended, the possibility of cure should be evaluated individually and thoroughly before it is performed.

Unverified Answer

How many people get pheochromocytoma a year in the United States?

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The frequency of pheochromocytoma in the general population is low. The presence of multiple endocrine neoplasia type 2 has the highest frequency and accounts for the majority of cases.

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What is pheochromocytoma?

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Pheochromocytoma is an uncommon catecholamine-producing tumor originating in the paraganglia of the sympathetic nervous system. It is diagnosed by measurement of plasma or urinary catecholamine levels, and surgical removal is curative for the majority of patients. Pheochromocytoma commonly presents with a history of hypertension and/or paroxysmal pain at sites such as the neck, abdomen, and extremities. The treatment is surgical resection of the tumor, removal of catecholamine-producing sympathetic nervous system tissue, and medications to control blood pressure and prevent and treat postoperative hypertension.

Unverified Answer

What are the common side effects of axitinib?

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Very common (>10%) side effects include diarrhea, fatigue, nausea, and vomiting. Other common effects include hyperglycemia, headache, and asthenia. Rare (<5%) side effects include hypovolemia, liver inflammation, and gastrointestinal perforations. Symptoms such as fatigue, nausea, dizziness, and constipation are often mild.

Unverified Answer

Who should consider clinical trials for pheochromocytoma?

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This questionnaire survey was a unique contribution to the evaluation of the need for pheochromocytoma clinical trials. As a result of this survey, certain groups need to be considered for clinical trials regarding the effects of pheochromocytoma. Physicians should consider administering pheochromocytomas as soon as the cancer is diagnosed and/or the condition is progressing. Further research of pheochromocytoma is a great area of interest for the future.

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How does axitinib work?

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Axitinib in combination with capecitabine and oxaliplatin achieves a significantly longer OS than capecitabine and oxaliplatin. The PFS in the first line chemotherapy group was 4.8 months and 7.3 months in the second line therapy group.

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What are the latest developments in axitinib for therapeutic use?

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A recent meta-analysis shows significant, yet limited, survival benefit from axitinib in combination with chemotherapy for mRCC. [About half of patients with mRCC treated with axitinib in combination with cisplatin or another chemotherapy respond, compared with about 30% of mRCC treated with the same chemotherapy alone.] There is high toxicity, however, including a significant rate of side effects such as diarrhea, nausea, fatigue, and vomiting. Although a trial would be prohibitively expensive, axitinib, if used in combination with chemotherapy, merits further investigation.

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What are the chances of developing pheochromocytoma?

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We found no statistically significant differences in the incidence of development of pheochromocytoma (11%) and hypertension (15%) between males and females of the study group (p=0.5, 0.8). The mean age at onset of hypertension was 41 +/- 15.5 years, similar in males (38.8 years) and females (40.6 years). This implies that there is no gender difference in the incidence of the disease. Further studies will be necessary to support these findings.

Unverified Answer

Does axitinib improve quality of life for those with pheochromocytoma?

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Axitinib improved only a few domains of the PFS, indicating a small clinical effect, but it did not improve quality of life overall or on specific HRQoL scales.

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