Multicenter Pheochromocytoma and Paraganglioma Evaluation

Pheochromocytoma Clinical Trial

— MUPPET  

Official title:

The MUPPET-study: Multicenter Pheochromocytoma and Paraganglioma Evaluation for Follow-up Screening, Genetics Sub-Typing, Therapy and Outcome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03344016
• Other study ID #: 742-16
• Status: Recruiting
• Phase: N/A
• Start date: November 1, 2017
• Est. completion date: November 1, 2040

Study information

• Verified date: July 2020
• Source: Ludwig-Maximilians - University of Munich
• Contact: Felix Beuschlein, M.D.
• Phone: +41 44 255 36 25
• Email: felix.beuschlein[@]usz.ch
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 1148
• Est. completion date: November 1, 2040
• Est. primary completion date: November 1, 2035
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years and older

Eligibility

Inclusion Criteria:

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.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Paraganglioma
• Pheochromocytoma

Intervention

Other:
• Contact by clinical center
Patients will be acitvely contacted by the clinical center for follow-up procedure

Locations

Country	Name	City	State
Switzerland	University Hospital Zurich	Zurich

Sponsors (7)

Lead Sponsor	Collaborator
Felix Beuschlein	Lübeck University Clinic, Ludwig-Maximilians - University of Munich, Radboud University, Technische Universität Dresden, University of Zurich, Wuerzburg University Hospital

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Morbidity	to investigate whether standardized follow-up for patients at risk for PPGL improves long-term outcome	18 years
Secondary	Time to recurrence	Time to recurrence	18 years
Secondary	Size of recurrent tumors	Size of recurrent tumors	18 years
Secondary	Numbers of metastases	Numbers of metastases	18 years
Secondary	Biomarker indices of disease burden	Surrogate biomarker indices of disease burden (such as hormonal measures)	18 years
Secondary	Metabolic parameter - blood glucose	fasting blood glucose	18 years
Secondary	Metabolic parameter - HbA1c	Hb1Ac	18 years
Secondary	Metabolic parameter - cholesterol	fasting cholesterol (total, LDL, HDL)	18 years
Secondary	Hormonal parameters	hormonal profiles including metanephrines, normetanephrines and metoxytyramine (that will allow for sub-group specification of PPGLs)	18 years
Secondary	Blood pressure profiles	Measurement of 24h blood pressure and ambulatory blood pressure measurments	18 years
Secondary	Cardiac function	Leftventricualr ejection fraction	18 years
Secondary	Disease specific mortality	Disease specific mortality	18 years
Secondary	Overall mortality	Overall mortality	18 years