Multicenter Pheochromocytoma and Paraganglioma Evaluation
- Conditions
- ParagangliomaPheochromocytoma
- Interventions
- Other: Contact by clinical center
- Registration Number
- NCT03344016
- Lead Sponsor
- Felix Beuschlein
- Brief Summary
Target population:
Patients with (1) newly diagnosed or (2) past history of pheochromocytomas and paragangliomas (PPGL) or (3) carrier of genetic mutations in known PPGL susceptibility genes.
International multicenter prospective cohort study with randomized intervention (special care follow-up vs. standard care follow-up).
All patients will receive instructions about follow-up at the time point of study inclusion. Patients randomized to the standard care follow-up group will be advised to return annually for follow-up according to current routine practice (without active re-scheduling). In contrast, patients randomized to the special care follow-up group will also be advised to return annually for follow-up but these patients will be actively invited, re-scheduled and reminded by the centers to meet scheduled follow-up appointments.
- Detailed Description
The long-term goal of the research planned under this protocol is to reduce morbidity and mortality of patients with PPGLs by improving approaches for management, follow-up and therapy of affected patients. As a first step towards attaining this goal, the primary objective of this protocol is to investigate whether standardized follow-up results in improved long-term outcome in terms of less morbidity and mortality as compared. The central hypothesis is that pro-active, structured and periodic disease screening and management of patients at risk for developing PPGLs and other neoplasms can lead to earlier detection of tumors and reduce adverse outcomes associated with cardiovascular, metabolic and oncologic complications of the tumors than standard care follow-up. The underlying rationale is that establishing improved outcomes for patients at risk for PPGLs will enable evidence-based recommendations for disease follow-up and management, thereby establishing wider acceptance and use of outlined practices with ensuing improvements in the health and quality of life of affected patients and their families.
In addition to the primary objective directed at establishing whether standardized and structured follow-up of patients with an increased risk for new events of PPGL (recurrent tumor, new tumor, or metastases) will result in improved longterm outcome, this protocol will enable several secondary objectives to be addressed using clinical (e.g. age, mode of presentation), biochemical, metabolic and genetic characteristics. These include:
1. to identify prognostic markers of disease progression
2. to assess whether clinical presentation, cardiovascular, metabolic and biochemical phenotype, genetic background and tumor characteristics (location, size, recurrence, pathology) are useful for development of personalized follow-up strategies.
3. to investigate whether standardized follow-up affects quality of life
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 1148
male and female patients (≥ 5 years of age), who fulfill one or more of the following criteria: (i) Patients with a newly diagnosed PPGL. (ii) Patients with a previous history of PPGLs. (iii) Carrier of genetic mutations known to predispose for the development of PPGLs.
All subjects must have read, understood and signed the informed consent form, before inclusion into the study protocol. Signed parental consent must be obtained for children with suspected PPGLs who are enrolled in the study.
- Patients with impaired mental capacity that precludes informed consent.
- Pregnancy does not constitute criteria for exclusion from the protocol. However, in pregnant women no Clonidine testing, no PET scanning, MIBG scanning or contrast CT will be performed.
- Patients at risk from injury from the MRI magnet due to implantable metal or who suffer from anxiety in enclosed spaces are excluded from MRI.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Special care follow-up group Contact by clinical center In addition to the information leaflet patients will be actively contacted by the clinical center to increase the likelihood that patients meet recommended follow-up schedules.
- Primary Outcome Measures
Name Time Method Morbidity 18 years to investigate whether standardized follow-up for patients at risk for PPGL improves long-term outcome
- Secondary Outcome Measures
Name Time Method Blood pressure profiles 18 years Measurement of 24h blood pressure and ambulatory blood pressure measurments
Cardiac function 18 years Leftventricualr ejection fraction
Time to recurrence 18 years Time to recurrence
Numbers of metastases 18 years Numbers of metastases
Metabolic parameter - blood glucose 18 years fasting blood glucose
Disease specific mortality 18 years Disease specific mortality
Size of recurrent tumors 18 years Size of recurrent tumors
Biomarker indices of disease burden 18 years Surrogate biomarker indices of disease burden (such as hormonal measures)
Metabolic parameter - HbA1c 18 years Hb1Ac
Overall mortality 18 years Overall mortality
Metabolic parameter - cholesterol 18 years fasting cholesterol (total, LDL, HDL)
Hormonal parameters 18 years hormonal profiles including metanephrines, normetanephrines and metoxytyramine (that will allow for sub-group specification of PPGLs)
Trial Locations
- Locations (1)
University Hospital Zurich
🇨🇭Zurich, Switzerland