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RecruitingNCT03160274Updated Oct 15, 2025

Genetic Analysis of Pheochromocytomas, Paragangliomas and Associated Conditions

An observational study in Pheochromocytoma, Paraganglioma and Inherited Cancer Syndrome, sponsored by The University of Texas Health Science Center at San Antonio. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2025-10-15.

Sponsored by The University of Texas Health Science Center at San Antonio · Observational

Study type
Observational
Model
Cohort
Time perspective
Other
Enrollment
2,000
Sex
All
01

Study summary

Pheochromocytomas and paragangliomas are neural crest-derived tumors of the nervous system that are often inherited and genetically heterogeneous. Genetic screening is recommended for patients and their relatives, and can guide clinical decisions. However, a mutation is not found in all cases. The aims of this proposal are to: 1) to map gene(s) involved in pheochromocytoma, and 2) identify genotype-phenotype correlations in patients with pheochromocytoma/paraganglioma of various genetic origins.

Read the detailed description

Pheochromocytoma and paragangliomas are tumors originated from neuroectoderm cells located in the adrenal or extra-adrenal paraganglia, often leading to increased secretion of hormones known as catecholamines. These tumors represent a potentially curable cause of hypertension and are malignant in about 10-15% of the cases. Approximately 40% of patients with pheochromocytomas and/or paraganglioma have an inherited mutation. In addition, some patients and/or their relatives that are mutation carriers can develop other tumors as part of inherited cancer susceptibility syndromes. Therefore, detection of the susceptibility mutation is important for diagnosis and follow up. However, the susceptibility gene mutation cannot be identified in all cases. Studies that aim to identify novel susceptibility genes for pheochromocytoma are required.

The fist aim of this study is to identify novel pheochromocytoma susceptibility genes. Characterization of such gene(s) can improve our understanding of the pathogenesis pheochromocytoma and paraganglioma and have an impact in diagnosis, therapeutic planning and genetic screening of relatives.

The second aim of this project is to characterize relationships between mutations and clinical features that can provide insights into clinical surveillance and screening of at-risk individuals.

02

Conditions studied

  • Pheochromocytoma
  • Paraganglioma
  • Inherited Cancer Syndrome
  • Associated Conditions
  • Kidney Neoplasms
  • Bone Cancer
  • Thyroid Neoplasms
  • Other Cancer

Keywords

  • tumor suppressor gene
  • oncogene
  • mutation
  • susceptibility gene
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
Yes
Sampling method
Probability sample

Study population

Individuals must have confirmed personal or family history of pheochromocytoma, paraganglioma or associated conditions. Relatives of patients with pheochromocytoma and/or paraganglioma are also eligible. Consent form will be obtained from all patients. It is expected that a number of participants in the study will be from outside our institution and also outside U.S.

Inclusion criteria

  • diagnosis of pheochromocytoma and or paraganglioma
  • family member with diagnosis of pheochromocytoma and or paraganglioma
  • diagnosis of a pheochromocytoma- and or paraganglioma-associated condition
  • family member with diagnosis of a pheochromocytoma- and or paraganglioma-associated condition

Exclusion criteria

Exclusion Criteria:

  • unconfirmed diagnosis of pheochromocytoma and/or paraganglioma or associated condition
04

Study design

Observational model
Cohort
Time perspective
Other
Enrollment
2,000 participants (estimated)
Target follow-up
30 Years
Patient registry
Yes
Biospecimen retention
Samples with dna

Interventions

  • GeneticGenetic screening

    Germline and/or tumor samples will be screened for mutations

05

What researchers measure

Primary outcomes

  1. Identification of germline driver mutation

    Genetic screen detects a mutation that is likely responsible for tumor development

    Time frame: through study completion- average time approximately 6 months

  2. Identification of somatic driver mutation

    Genetic screen detects a mutation that is likely responsible for tumor development

    Time frame: through study completion- average time approximately 6 months

Secondary outcomes

  1. Identification of additional, potentially pathogenic genetic variants

    Genetic screen detects other mutations with potential pathogenic effects

    Time frame: through study completion- average time approximately 6 months

  2. Identification of clinical features other than pheochromocytoma and/or paraganglioma that segregate with disease

    Clinical data reveals other features that might associate with the main disease phenotype

    Time frame: through study completion- average time approximately 6 months

06

Study locations

1 of 1 sites recruiting
  • University of Texas Health Science Center
    San Antonio, Texas 78229, United States
    Recruiting
07

References and documents

Publications

  • Dahia PL. Pheochromocytoma and paraganglioma pathogenesis: learning from genetic heterogeneity. Nat Rev Cancer. 2014 Feb;14(2):108-19. doi: 10.1038/nrc3648. Epub 2014 Jan 20. PubMed 24442145 ↗

Individual participant data

Plan to share: No — Aggregate data will be available in the form of publications and pertinent data will be deposited in public depositories

08

Registry details

Key details

Study ID
NCT03160274
Lead sponsor
The University of Texas Health Science Center at San Antonio
Collaborators
National Institute of General Medical Sciences (NIGMS), The Paradifference Foundation, National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
May 19, 2017
Start date
Oct 19, 2005
Primary completion
Dec 31, 2030 (estimated)
Completion
Dec 31, 2030 (estimated)
Last update
Oct 15, 2025

Study contacts

Patricia L Dahia, MD,PhD
Contact
dahia@uthscsa.edu
2105674866
Patricia L Dahia, MD, PhD
principal investigator · The University of Texas Health Science Center at San Antonio

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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