Study of Proteus Syndrome and Related Congenital Disorders

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age1-99
SponsorNational Human Genome Research Institute (NHGRI)

About this trial

This study will examine rare congenital disorders that involve malformations and abnormal growth. It will focus on patients with Proteus syndrome, whose physical features are characterized by overgrowth, benign tumors of fatty tissue or blood vessels, asymmetric arms or legs, and large feet with very thick soles. The study will explore the genetic and biochemical cause and course of the disease, the changes in symptoms over time, and the effects of the disease on patients.

Patients with Proteus syndrome may be eligible for this study. Study candidates will have a medical history and physical examination, including X-rays and possibly other imaging tests, such as computerized tomography (CT), magnetic resonance imaging (MRI) and ultrasound. Other tests and examinations may be done if needed.

Those enrolled in the study may be interviewed or complete questionnaires, or both, about how their disease affects them. Patients will provide a small blood sample for research.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial population

Patients with overgrowth syndromes.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

1,500 Participants
are grouped into 1 trial group
Group A: Proteus Syndrome

Trial outcomes

Primary outcomes

1

Molecular delineation of disorders under study

Determine causative genotypes of overgrowth disorders

Time frame
ongoing
2

Determination of natural history of disorders under study

Determine natural history of a variety of overgrowth disorders

Time frame
ongoing

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

National Human Genome Research Institute (NHGRI)

Lead sponsor

Children's National Research Institute

Collaborator

Uniformed Services University of the Health Sciences

Collaborator