Pheochromocytoma and Paraganglioma (PPGL)Phase 4NCT07907341
Safety and Efficacy of Belzutifan in Preoperative Preparation for Patients With Pheochromocytoma/Paraganglioma (PPGL)
A Phase 4 study testing Belzutifan in people with Pheochromocytoma and Paraganglioma (PPGL), run by Longfei Liu，MD at 1 site in China.
Trial summary
It is preliminarily clarified that belzutifan exerts an inhibitory effect on catecholamine synthesis in pheochromocytoma and paraganglioma (PPGL), and its safety is simultaneously confirmed, thereby providing preliminary experimental results for the further exploration of belzutifan as a perioperative preparation drug… Read more about this study
- Who is it for?
- Ages 14 to 85 years
- Conditions the study is for
- Pheochromocytoma and Paraganglioma (PPGL)
- What is being tested?
- Belzutifan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Apr 2026to 31 Dec 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 85 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 14 to 85 years.
- Adrenal pheochromocytoma or retroperitoneal paraganglioma suspected based on imaging and biochemical diagnoses.
- Scheduled for surgical treatment.
- American Society of Anesthesiologists (ASA) physical status classification of Grade Ⅰ to Ⅲ.
- Informed consent obtained from the patient or their legal guardian.
- Confirmed non-pregnant and non-lactating status.
Show all 14Show fewer
- Laboratory requirements:
- Absolute Granulocyte Count (AGC) > 1.5 × 10⁹/L;
- Platelet count > 80 × 10⁹/L;
- Hemoglobin (Hb) > 90 g/L;
- Serum bilirubin < 1.5 × the upper limit of normal (ULN);
- Serum creatinine < 1.5 × ULN or creatinine clearance >= 60 mL/min;
- Negative serum human chorionic gonadotropin (hCG) test result.
- Capacity to understand the study and willingness to sign a written informed consent form.
Who cannot join
- Incapable of correctly understanding the content of questionnaires and unable to cooperate with researchers.
- Receiving other anti-tumor drug therapies.
- Pregnant or lactating women, and men or women of childbearing age who refuse to take appropriate contraceptive measures.
- A history of allergic reactions to belzutifan or hypoxia-inducible factor (HIF) inhibitors.
- Presence of multiple factors affecting oral drug administration (e.g., dysphagia, chronic diarrhea, intestinal obstruction, etc.) or other conditions interfering with medication intake.
- Severe myelosuppression or coagulation abnormalities (International Normalized Ratio (INR) > 1.5, or Prothrombin Time (PT) > upper limit of normal (ULN) by 4 seconds, or Activated Partial Thromboplastin Time (APTT) > 1.5 × ULN), a bleeding tendency, or ongoing thrombolytic or anticoagulant therapy.
Show all 21Show fewer
- A history of treatment with other HIF inhibitors.
- Prior receipt of chemotherapy, targeted therapy, biological agents, or other investigational treatments within the 4 weeks prior to the first administration of the study intervention.
- Active or uncontrolled comorbid diseases, including but not limited to:
- Uncontrolled myocardial ischemia or myocardial infarction, cardiac arrhythmias (including corrected QT interval (QTc) >= 480 ms), and uncontrolled congestive heart failure of New York Heart Association (NYHA) Grade >= 2;
- Clinically significant heart disease, including unstable angina pectoris, acute myocardial infarction, or a history of Coronary Artery Bypass Grafting (CABG) or Percutaneous Transluminal Coronary Angioplasty (PTCA) within 6 months prior to study enrollment, or congestive heart failure of NYHA Grade Ⅲ or Ⅳ;
- Persistent or active infection;
- Liver cirrhosis, decompensated liver disease, active hepatitis, or chronic hepatitis requiring antiviral therapy;
- Renal failure requiring hemodialysis or peritoneal dialysis;
- A history of immunodeficiency, including Human Immunodeficiency Virus (HIV) infection or other acquired/congenital immunodeficiency diseases, or a history of organ transplantation.
- A history of seizures requiring medical treatment.
- Occurrence of any of the following conditions within 6 months prior to study registration: Cerebrovascular Accident (CVA) or Transient Ischemic Attack (TIA); severe or unstable cardiac arrhythmia; pulmonary embolism or untreated Deep Venous Thrombosis (DVT).
- A history of psychoactive substance abuse with inability to abstain, or presence of mental disorders.
- Concomitant use of drugs that interact with belzutifan.
- Patients with stable disease who decline surgical treatment.
- Resting pulse oximetry reading < 92%, or requirement for intermittent oxygen supplementation, or long-term oxygen therapy.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Xiangya Hospital of Central South University
Changsha, Hunan, 410008
About this study
Official title: Safety and Efficacy of Belzutifan in Preoperative Preparation for Patients With Pheochromocytoma/Paraganglioma (PPGL): A Pilot Study
It is preliminarily clarified that belzutifan exerts an inhibitory effect on catecholamine synthesis in pheochromocytoma and paraganglioma (PPGL), and its safety is simultaneously confirmed, thereby providing preliminary experimental results for the further exploration of belzutifan as a perioperative preparation drug for patients with PPGL.
- Being tested
- Belzutifan
- Keywords
- pheochromocytoma and paraganglioma, belzutifan
- Medical subject headings
- Pheochromocytoma, Paraganglioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BelzutifanDrug
Belzutifan, 120 mg (3 × 40 mg) once daily for 5 consecutive days preoperatively, administered fasting or with a low- to moderate-fat diet.
| Study group | Role | What they receive |
|---|---|---|
| Belzutifan group | Experimental | Not described Receives: Drug: Belzutifan |
What is being measured
The primary outcome is the main question the study is designed to answer.
plasma Metanephrines
Measured: Perioperative
Secondary outcomes (10)
Intraoperative blood pressure
Measured: Perioperative
Intraoperative heart rate
Measured: Perioperative
Intraoperative dosage of vasoactive agents
Measured: Perioperative
Intraoperative fluid infusion volume
Measured: Perioperative
Postoperative ICU admission rate
Measured: Perioperative
Incidence of perioperative cardiovascular and cerebrovascular complications and mortality
Measured: Perioperative
Postoperative use of vasoactive agents
Measured: Perioperative
Postoperative blood MNs level
Measured: Perioperative
Catecholamine levels in surgically resected tumor tissue
The concentrations of catecholamines (epinephrine, norepinephrine, dopamine) within surgically resected pheochromocytoma/paraganglioma tumor tissue will be measured and compared between study groups.
Measured: Perioperative
Safety evaluation of belzutifan
Measured: After patient discharge
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2026
- Study start1 Apr 2026
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pheochromocytoma and Paraganglioma (PPGL)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Longfei Liu，MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Longfei Liu，MD, Xiangya Hospital of Central South University
- Investigators
- Longfei Liu, Doctor of Medicine, Principal investigator, Xiangya Hospital of Central South University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07907341
- Sponsor's protocol ID
- 202601017
- Other IDs
- ChiCTR2600120686, MR-43-26-069503
- Status last verified
- Oct 2026
- First submitted
- 12 Oct 2026
- Last updated
- 30 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Belzutifan in Preoperative Preparation for Patients With Pheochromocytoma/Paraganglioma (PPGL): A Pilot Study
- ClinicalTrials.gov ID
- NCT07907341
- Acronym / sponsor ID
- 202601017
- Keywords
- pheochromocytoma and paraganglioma, belzutifan
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Longfei Liu，MDUniversity, hospital or other
- Study start
- 1 Apr 2026 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 30 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
It is preliminarily clarified that belzutifan exerts an inhibitory effect on catecholamine synthesis in pheochromocytoma and paraganglioma (PPGL), and its safety is simultaneously confirmed, thereby providing preliminary experimental results for the further exploration of belzutifan as a perioperative preparation drug for patients with PPGL.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Belzutifan group | Experimental | Drug: Belzutifan |
Who may qualify
- Ages
- 14 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- Aged 14 to 85 years.
- Adrenal pheochromocytoma or retroperitoneal paraganglioma suspected based on imaging and biochemical diagnoses.
- Scheduled for surgical treatment.
- American Society of Anesthesiologists (ASA) physical status classification of Grade Ⅰ to Ⅲ.
- Informed consent obtained from the patient or their legal guardian.
Key exclusion criteria (21 listed)
- Incapable of correctly understanding the content of questionnaires and unable to cooperate with researchers.
- Receiving other anti-tumor drug therapies.
- Pregnant or lactating women, and men or women of childbearing age who refuse to take appropriate contraceptive measures.
- A history of allergic reactions to belzutifan or hypoxia-inducible factor (HIF) inhibitors.
- Presence of multiple factors affecting oral drug administration (e.g., dysphagia, chronic diarrhea, intestinal obstruction, etc.) or other conditions interfering with medication intake.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BelzutifanBelzutifan, 120 mg (3 × 40 mg) once daily for 5 consecutive days preoperatively, administered fasting or with a low- to moderate-fat diet. | Drug | Belzutifan group |
Primary outcome
plasma Metanephrines
Time frame: Perioperative
10 secondary outcomes
Intraoperative blood pressure
Time frame: Perioperative
Intraoperative heart rate
Time frame: Perioperative
Intraoperative dosage of vasoactive agents
Time frame: Perioperative
Intraoperative fluid infusion volume
Time frame: Perioperative
Postoperative ICU admission rate
Time frame: Perioperative
Incidence of perioperative cardiovascular and cerebrovascular complications and mortality
Time frame: Perioperative
Postoperative use of vasoactive agents
Time frame: Perioperative
Postoperative blood MNs level
Time frame: Perioperative
Catecholamine levels in surgically resected tumor tissue
Time frame: Perioperative
Safety evaluation of belzutifan
Time frame: After patient discharge
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Pheochromocytoma and Paraganglioma (PPGL)
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 2
Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumor
RecruitingPhase 2
18F-Fibroblast Activation Protein Inhibitor ([18F]FAPI-74) PET Imaging for Cancer Detection
Sarcoma, Small Cell Lung Cancer or Extrapulmonary Neuroendocrine Cancer (EP-NEC)
Not yet recruitingPhase 2
STOMP OUT: A Phase 2 Study To Evaluate The Effects Of Ivonescimab In Patients With Unresectable/Metastatic Adrenocortical Carcinoma (ACC) Or Unresectable Pheochromocytoma/Paraganglioma (PPGL)
Adrenocortical Carcinoma, Pheochromocytoma
Not yet recruitingObservational study
Italian Multicenter Experience With Radioreceptor-assisted Therapy (PRRT)
Pheochromocytomas, Paragangliomas
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Longfei Liu, Doctor of Medicine principal investigator
Xiangya Hospital of Central South University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Belzutifan in people with Pheochromocytoma and Paraganglioma (PPGL), run by Longfei Liu，MD at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chao Wang, Master (+86 151 1103 1075, wangchao296@csu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
plasma Metanephrines (time frame: Perioperative). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2026.