Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age1-120
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)

About this trial

The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIIES; Job's syndrome) is caused by STAT3 mutations, and is a multi-system disorder with skeletal, vascular, and connective tissue manifestations. Understanding how STAT3 mutations cause these diverse clinical manifestations is critical to our complete understanding of bone metabolism, bronchiectasis, dental maturation, and atherosclerosis. Bi-allelic mutations in DOCK8 cause a combined immunodeficiency previously described as autosomal-recessive Hyper IgE syndrome. These individuals suffer from extensive viral infections as well as have a high incidence of malignancy and mortality. The pathogenesis of this disease and long-term natural history is being investigated. Therefore, we seek to enroll patients and families with a confirmed or suspected diagnosis of HIES syndrome for extensive phenotypic and genotypic study as well as disease management. Patients will be carefully examined by a multidisciplinary team and followed longitudinally. Through these studies we hope to better characterize the clinical presentation of STAT3-mutated HIES, DOCK8 deficiency and other causes of the hyper IgE phenotype, and to be able to identify further genetic etiologies, as well as understand the pathogenesis of HIES. We seek to enroll 300 patients and 300 relatives.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Were referred to the NIH with a diagnosis or a suspicion of Hyper IgE syndrome.

Are patients referred for other immune syndromes that demonstrate some of the characteristics of HIES.

>=1 month for affected subjects

Aged >=2 years for unaffected subjects

Disqualifiers

None

Trial population

primary clinical

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

600 Participants
are grouped into 2 trial groups
Group A: Affected adults and children
Group B: Relatives

Trial outcomes

Primary outcomes

1

To clinically phenotype AD-HIES, DOCK8 deficiency, PGM3 deficiency and other related hyper IgE syndromes

established clinical phenotype of AD-HIES, DOCK8 deficiency, PGM3 deficiency and other related hyper IgE syndromes

Time frame
end of study
2

To assess quality of life on the basis of clinical and immunologic evaluations

quality of life assessments based on clinical and immunologic evaluations

Time frame
end of study
3

To understand the pathogenesis of the immunologic defect in hyper IgE syndromes as well as the diverse clinical features such as wound healing abnormalities

understanding of the pathogenesis of the immunologic defect in hyper IgE syndromes as well as the diverse clinical features such as wound healing abnormalities

Time frame
end of study
4

To identify, characterize, and treat complications of the hyper IgE syndromes as they arise

identification, characterization, and treatment of complications of the hyper IgE syndromes

Time frame
end of study

Secondary outcomes

Other outcomes

Sponsors and contacts

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