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Trial record 2 of 73 for:    "hereditary paraganglioma-pheochromocytoma" OR "Paraganglioma"

Multicenter Pheochromocytoma and Paraganglioma Evaluation (MUPPET)

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ClinicalTrials.gov Identifier: NCT03344016
Recruitment Status : Recruiting
First Posted : November 17, 2017
Last Update Posted : November 17, 2017
Sponsor:
Collaborators:
Universitätsklinik Dresden
Universitätsklinik Würzburg
Universitätsspital Zürich
University Clinic Nijmegen
University Clinic Warsaw
Universitätsklinik Lübeck
Information provided by (Responsible Party):
Felix Beuschlein, Ludwig-Maximilians - University of Munich

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.


Condition or disease Intervention/treatment Phase
Pheochromocytoma Paraganglioma Other: Contact by clinical center Not Applicable

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1148 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

To provide evidence whether strict follow-up will result in improvement in clinical outcome parameters as compared to standard care follow-up, patients upon inclusion into the study protocol will be randomized into two groups:

  • Standard care follow-up group: Patients will receive an information leaflet (see appendix), which advises on recommended routine follow-up according to international guidelines.
  • Special care follow-up group: 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.

Stratification will be done for inclusion criteria (newly diagnosed PPGL, previous history of PPGL, gene carrier, presence of malignant PPGL and center).

Masking: None (Open Label)
Primary Purpose: Health Services Research
Official Title: The MUPPET-study: Multicenter Pheochromocytoma and Paraganglioma Evaluation for Follow-up Screening, Genetics Sub-Typing, Therapy and Outcome
Estimated Study Start Date : November 2017
Estimated Primary Completion Date : November 2035
Estimated Study Completion Date : November 2040


Arm Intervention/treatment
No Intervention: Standard care follow-up group
Patients will receive an information leaflet (see appendix), which advises on recommended routine follow-up according to international guidelines.
Active Comparator: Special care follow-up group
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.
Other: Contact by clinical center
Patients will be acitvely contacted by the clinical center for follow-up procedure




Primary Outcome Measures :
  1. Morbidity [ Time Frame: 18 years ]
    to investigate whether standardized follow-up for patients at risk for PPGL improves long-term outcome


Secondary Outcome Measures :
  1. Time to recurrence [ Time Frame: 18 years ]
    Time to recurrence

  2. Size of recurrent tumors [ Time Frame: 18 years ]
    Size of recurrent tumors

  3. Numbers of metastases [ Time Frame: 18 years ]
    Numbers of metastases

  4. Biomarker indices of disease burden [ Time Frame: 18 years ]
    Surrogate biomarker indices of disease burden (such as hormonal measures)

  5. Metabolic parameter - blood glucose [ Time Frame: 18 years ]
    fasting blood glucose

  6. Metabolic parameter - HbA1c [ Time Frame: 18 years ]
    Hb1Ac

  7. Metabolic parameter - cholesterol [ Time Frame: 18 years ]
    fasting cholesterol (total, LDL, HDL)

  8. Hormonal parameters [ Time Frame: 18 years ]
    hormonal profiles including metanephrines, normetanephrines and metoxytyramine (that will allow for sub-group specification of PPGLs)

  9. Blood pressure profiles [ Time Frame: 18 years ]
    Measurement of 24h blood pressure and ambulatory blood pressure measurments

  10. Cardiac function [ Time Frame: 18 years ]
    Leftventricualr ejection fraction

  11. Disease specific mortality [ Time Frame: 18 years ]
    Disease specific mortality

  12. Overall mortality [ Time Frame: 18 years ]
    Overall mortality



Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03344016


Contacts
Contact: Felix Beuschlein, M.D. +41 44 255 36 25 felix.beuschlein@med.uni-muenchen.de
Contact: Martin Reincke, M.D. +49 89 4400 52100 martin.reincke@med.uni-muenchen.de

Locations
Germany
Klinikum der Universität München Recruiting
München, Germany, 80336
Contact: Felix Beuschlein, M.D.    +41 44 255 36 25    felix.beuschlein@med.uni-muenchen.de   
Contact: Martin Reincke, M.D.    +49 89 4400 52100    martin.reincke@med.uni-muenchen.de   
Sponsors and Collaborators
Felix Beuschlein
Universitätsklinik Dresden
Universitätsklinik Würzburg
Universitätsspital Zürich
University Clinic Nijmegen
University Clinic Warsaw
Universitätsklinik Lübeck
Investigators
Principal Investigator: Felix Beuschlein, M.D. Klinikum der Universität München

Responsible Party: Felix Beuschlein, Prof., Ludwig-Maximilians - University of Munich
ClinicalTrials.gov Identifier: NCT03344016     History of Changes
Other Study ID Numbers: 742-16
First Posted: November 17, 2017    Key Record Dates
Last Update Posted: November 17, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Pheochromocytoma
Paraganglioma
Carotid Body Tumor
Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Nerve Tissue
Paraganglioma, Extra-Adrenal