PheochromocytomaPhase 2NCT05885386
A Study on the Safety and Effectiveness of Temozolomide for Neoadjuvant Treatment of PPGL
A Phase 2 study testing Temozolomide in people with Pheochromocytoma and Paraganglioma, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
This phase II trial studies the effectiveness oftemozolomide in the neoadjuvant therapy oflocally advanced,or unresectable pheochromocytoma or paragangliom(PPGL). Temozolomide (TMZ) is a novel oral alkylation chemotherapeutic agent. Read more about this study
- Who is it for?
- Ages 10 to 70 years
- Conditions the study is for
- Pheochromocytoma, Paraganglioma
- What is being tested?
- Temozolomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Apr 2023to 1 Oct 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 10 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Provide written informed consent.
- Age 10-70 years old
- Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
- The patient is diagnosed as pheochromocytoma or paraganglioma which is unresectable with R0 surgery, or extensive and thus maybe requiring resection of important organs, or Inoperable due to heart and other complications, or with very high surgical risk.
- Estimated life expectancy longer than 6 months.
- Confirmed non-pregnancy and lactation. During the entire study period and within 6 months after the last administration, the subjects and their spouses are willing to use efficient contraceptive measures.
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- Laboratory requirements:
- Absolute granulocyte count (AGC) greater than 1.5 x 109/L;
- Platelet count greater than 80 x 109/L; 3) Hemoglobin greater than 90g/L;
- Serum bilirubin less than 1.5 x upper limit of normal (ULN);
- Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than 2.5 x ULN; Serum creatinine less than 1.5 x ULN or creatinine clearance (CCr)≥60ml/min;
- Doppler ultrasound assessment: left ventricular ejection fraction (LVEF) ≥ lower limit of normal value (50%).
Who cannot join
- Have other tumors.
- Patients were treated with other antitumor agents.
- Pregnant or nursing women.
- A history of allergic reactions to temozolomide or dacarbazine.
- Severe myelosuppression or abnormal coagulation.
- Severe liver and kidney insufficiency.
- Bowel obstruction or other conditions that interfere with taking medication.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing
Anli Tong, 13911413589, tonganli@hotmail.com
About this study
Official title: A Study on the Safety and Effectiveness of Temozolomide for Neoadjuvant Treatment of Pheochromocytoma or Paraganglioma Patients
This phase II trial studies the effectiveness oftemozolomide in the neoadjuvant therapy oflocally advanced,or unresectable pheochromocytoma or paragangliom(PPGL). Temozolomide (TMZ) is a novel oral alkylation chemotherapeutic agent. Inthisstudy,temozolomidewill be used preoperatively in order to change unresectable tumors to resectable and reduce the high risk of surgery.
Read the detailed description
The first choice for the treatment of PPGL is surgery. PPGL can be cured by complete resection of the lesion. However, some PPGLs could not be performed R0 resection due to the large tumor size and close relationship with surrounding tissues (blood vessels, kidneys, pancreas, liver, etc.). In this case, in order to achieve R0 resection, they need to undergo expand the scope of surgery, such as simultaneous resection of vital organs,and with extreamly high risks.There is no treatment option for those locally advanced or unresectable PPGLpatients currently. Temozolomide (TMZ), an oral alkylation chemotherapeutic agent, has been used in recent years and shown to have beneficial effects on metastatic PPGL with few side effects. TMZ has been recommended in National Comprehensive Cancer Network (NCCN) Guidelines Version 1.2022 for treating metastatic PPGL patients. This prospective, single arm, phase II study is designed to evaluate the efficacy of neoadjuvant therapy with TMZ in locally advanced,or unresectable PPGL patients or patients with severe catecholamine cardiomyopathy who are intolerance of operation.TMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days preoperatively. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days. Imaging examinations will be conducted after3 courses to re-evaluate the surgical possibility and surgery risks. If the patient's tumor shrinks after 3 courses but is still unresectable, the patients will continue TMZ therapy for another 3 courses.
- Conditions
- Pheochromocytoma, Paraganglioma
- Being tested
- Temozolomide
- Medical subject headings
- Pheochromocytoma, Paraganglioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TemozolomideDrug
TMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days preoperatively. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Pheochromocytoma or Paraganglioma patients | Other | TMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days preoperatively. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days. Receives: Drug: Temozolomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of patients whose tumor change from unresectable to resectable tumor
The proportion of PPGL patients whose tumor change from unresectable to resectable
Measured: At the end of Cycle 3 (each cycle is 28 days)
Secondary outcomes (7)
the objective response rate (ORR)
Defined for all patients whose tumor met the criteria of Complete Response (CR)and Partial Response (PR)
Measured: At the end of Cycle 3 (each cycle is 28 days)
The ratio of tumor shrinkage.
The proportion of decrease in the sum of total size of the target lesions after treatment compared to before treatment.
Measured: At the end of Cycle 3 (each cycle is 28 days)
The biochemical response.
An effective response of 24hCA, MNs meant that the concentration decreaseed by more than 40% than the baseline value or decreaseed to the normal range.
Measured: At the end of Cycle 3 (each cycle is 28 days)
R0 resection rate
The proportion of patients with surgical resection reached R0 resection
Measured: At the end of Cycle 3 (each cycle is 28 days)
Major pathological response rate (MPR)
Defined as the remaining surviving tumor after surgical resection do not exceed 10% of the initial tumor tissue.
Measured: At the end of Cycle 3 (each cycle is 28 days)
Pathologic complete remission (pCR)
There is no tumor cells microscopically.
Measured: At the end of Cycle 3 (each cycle is 28 days)
Safety of temozolomide treatment
Incidence of adverse events assessed by Common Terminology Criteria for Adverse Events
Measured: At the end of Cycle 1 (each cycle is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2023
- Study start1 Apr 2023
- Primary completion1 Oct 2025 (estimated)
- Study completion1 Oct 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PheochromocytomaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- PPGLs could not be performed R0 resection due to the large tumor size and close relationship with surrounding tissues (blood vessels, kidneys, pancreas, liver, etc.)
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anli Tong, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05885386
- Sponsor's protocol ID
- 06086-05
- Status last verified
- May 2023
- First submitted
- 22 May 2023
- Last updated
- 1 Jun 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study on the Safety and Effectiveness of Temozolomide for Neoadjuvant Treatment of Pheochromocytoma or Paraganglioma Patients
- ClinicalTrials.gov ID
- NCT05885386
- Acronym / sponsor ID
- 06086-05
- Conditions
- Pheochromocytoma, Paraganglioma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Apr 2023 (actual)
- Primary completion
- 1 Oct 2025 (estimated)
- Study completion
- 1 Oct 2025 (estimated)
- First posted
- 1 Jun 2023 (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.
This phase II trial studies the effectiveness oftemozolomide in the neoadjuvant therapy oflocally advanced,or unresectable pheochromocytoma or paragangliom(PPGL). Temozolomide (TMZ) is a novel oral alkylation chemotherapeutic agent. Inthisstudy,temozolomidewill be used preoperatively in order to change unresectable tumors to resectable and reduce the high risk of surgery.
Detailed description
The first choice for the treatment of PPGL is surgery. PPGL can be cured by complete resection of the lesion. However, some PPGLs could not be performed R0 resection due to the large tumor size and close relationship with surrounding tissues (blood vessels, kidneys, pancreas, liver, etc.). In this case, in order to achieve R0 resection, they need to undergo expand the scope of surgery, such as simultaneous resection of vital organs,and with extreamly high risks.There is no treatment option for those locally advanced or unresectable PPGLpatients currently. Temozolomide (TMZ), an oral alkylation chemotherapeutic agent, has been used in recent years and shown to have beneficial effects on metastatic PPGL with few side effects. TMZ has been recommended in National Comprehensive Cancer Network (NCCN) Guidelines Version 1.2022 for treating metastatic PPGL patients. This prospective, single arm, phase II study is designed to evaluate the efficacy of neoadjuvant therapy with TMZ in locally advanced,or unresectable PPGL patients or patients with severe catecholamine cardiomyopathy who are intolerance of operation.TMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days preoperatively. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days. Imaging examinations will be conducted after3 courses to re-evaluate the surgical possibility and surgery risks. If the patient's tumor shrinks after 3 courses but is still unresectable, the patients will continue TMZ therapy for another 3 courses.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- PPGLs could not be performed R0 resection due to the large tumor size and close relationship with surrounding tissues (blood vessels, kidneys, pancreas, liver, etc.)
| Arm | Type | Receives |
|---|---|---|
| Pheochromocytoma or Paraganglioma patients | Other | Drug: TemozolomideTMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days preoperatively. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days. |
Who may qualify
- Ages
- 10 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Provide written informed consent.
- Age 10-70 years old
- Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
- The patient is diagnosed as pheochromocytoma or paraganglioma which is unresectable with R0 surgery, or extensive and thus maybe requiring resection of important organs, or Inoperable due to heart and other complications, or with very high surgical risk.
- Estimated life expectancy longer than 6 months.
Key exclusion criteria (7 listed)
- Have other tumors.
- Patients were treated with other antitumor agents.
- Pregnant or nursing women.
- A history of allergic reactions to temozolomide or dacarbazine.
- Severe myelosuppression or abnormal coagulation.
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 |
|---|---|---|
| TemozolomideTMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days preoperatively. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days. | Drug | Pheochromocytoma or Paraganglioma patients |
Primary outcome
The proportion of patients whose tumor change from unresectable to resectable tumor
Time frame: At the end of Cycle 3 (each cycle is 28 days)
The proportion of PPGL patients whose tumor change from unresectable to resectable
7 secondary outcomes
the objective response rate (ORR)
Time frame: At the end of Cycle 3 (each cycle is 28 days)
The ratio of tumor shrinkage.
Time frame: At the end of Cycle 3 (each cycle is 28 days)
The biochemical response.
Time frame: At the end of Cycle 3 (each cycle is 28 days)
R0 resection rate
Time frame: At the end of Cycle 3 (each cycle is 28 days)
Major pathological response rate (MPR)
Time frame: At the end of Cycle 3 (each cycle is 28 days)
Pathologic complete remission (pCR)
Time frame: At the end of Cycle 3 (each cycle is 28 days)
Safety of temozolomide treatment
Time frame: At the end of Cycle 1 (each cycle is 28 days)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2023.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Anli Tong principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Temozolomide in people with Pheochromocytoma and Paraganglioma, run by Peking Union Medical College Hospital 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 12 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 1 Jun 2023. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anli Tong (13911413589, tonganli@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of patients whose tumor change from unresectable to resectable tumor (time frame: At the end of Cycle 3 (each cycle is 28 days)). 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 1 Jun 2023.