An observational study in Panhypopituitarism, Gigantism/Acromegaly and Prolactinoma, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). Recruiting at 1 site in United States. Open to participants aged 2 Years to 70 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-10-19.
Sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · Observational
There is a variety of tumors affecting the pituitary gland in childhood; some of these tumors (eg craniopharyngioma) are included among the most common central nervous system tumors in childhood. The gene(s) involved in the pathogenesis of these tumors are largely not known; their possible association with other developmental defects or inheritance pattern(s) has not been investigated. The present study serves as a (i) screening/training, and, (ii) a research protocol.
As a screening and training study, this protocol allows our Institute to admit children with tumors of the hypothalamic-pituitary unit to the pediatric endocrine clinics and wards of the NIH Clinical Center for the purposes of
(i) training our fellows and students in the identification of genetic defects associated with pituitary tumor formation, and
(ii) teaching our fellows and students the recognition, management and complications of pituitary tumors
As a research study, this protocol aims at
(i) developing new clinical studies for the recognition and therapy of pituitary tumors; as an example, two new studies have emerged within the context of this protocol: (a) investigation of a new research magnetic resonance imaging (MRI) tool and its usefulness in the identification of pituitary tumors, and (b) investigation of the psychological effects of cortisol secretion in pediatric patients with Cushing disease. Continuation of this protocol will eventually lead to new, separate protocols that will address all aspects of diagnosis of pituitary tumors and their therapy in childhood.
(ii) Identifying the genetic components of pituitary oncogenesis; those will be investigated by (a) studying the inheritance pattern of pituitary tumors in childhood and their possible association with other conditions in the families of the patients, and (ii) collecting tumor tissues and examining their molecular genetics. As with the clinical studies, the present protocol may help generate ideas for future studies on the treatment and clinical follow up of pediatric patients with tumors of the pituitary gland and, thus, lead to the development of better therapeutic regimens for these neoplasms.
Study Description:\<TAB>
This protocol aims to evaluate subjects with tumors of the hypothalamic pituitary unit to: identify genetic components of pituitary and hypothalamic oncogenesis, to develop new clinical studies for the recognition and therapy of pituitary tumors, and to investigate the psychological effects of cortisol secretion.
Objectives:\<TAB>
Primary Objective:
To collect peripheral blood samples and tumor tissues and examine the molecular genetics of the specimens, in an effort to elucidate developmental pathways leading to pituitary gland oncogenesis and/or other defects.
Secondary Objectives:
To serve as a screening protocol for future studies on the treatment and clinical follow up of our patients with pituitary tumors; it is our hope that the protocol will continue to be a vehicle for the development of more related clinical studies.
To collect biospecimens.
To evaluate the cognitive, psychological, and patient-reported health status of mental and social well-being and symptoms of hypercortisolemia and adrenal insufficiency (AI) associated with recovery from Cushing syndrome (CS) in children with this disease.
Endpoints:
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Primary Endpoint:
Molecular genetic testing
Secondary Endpoints:
Pre- and/or post- treatment assessment of research variables related to pituitary/hypothalamic tumors and comorbidities.
Cognitive, behavioral, psychological, and patient-reported outcomes
202 studies on the registry are indexed under Acromegaly; 19 are open to participants now.
This study's planned enrollment of 2,000 is above the median of 100 across 81 observational studies indexed under Acromegaly.
Browse Acromegaly studies →Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) is the lead sponsor of 422 studies on the registry; 24 are open to participants now.
Of its 14 completed or terminated interventional studies of FDA-regulated products, 11 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Subjects with known or suspected pituitary or hypothalamic tumors or related disorders. Family members (adult and pediatric; affected and unaffected) may be enrolled in the DNA linkage analysis part of this protocol.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
Male or female with:
Evidence for the existence of a tumor of the hypothalamic-pituitary unit or related disorder, as indicated by previously obtained imaging studies or biochemical investigation of the hypothalamo-hypophyseal function (aged 2 years to 70 years)
or
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this study:
Pregnancy: Pregnancy will be evaluated only in participants of reproductive age (from 10 years old until 60 years of age unless menopause has already occurred per clinical report of the participant).
For participants enrolled as external participants or under the Linkage study (where research activities include no more than blood draws), any female who could possibly become pregnant will be screened using clinical criteria (history, with pregnancy testing only if indicated) for exclusion and this information will be documented in the consent process note in EMR. If a participant has initially been registered as external location and then presents on-site, then pregnancy test will be performed if within the reproductive age group.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients with pituitary tumors or hypothalmic defects
Procedure: Tissue specimen collection · Procedure: MRI
Tissue specimen collection
MRI scan
Molecular genetic testing, whole exome sequencing
To collect peripheral blood samples and tumor tissues and examine the molecular genetics of the specimens, in an effort to elucidate developmental pathways leading to pituitary gland oncogenesis and/or other defects.
Time frame: ongoing
To investigate the usefulness of a new MRI of the pituitary gland
Comparison of MRI techniques to identify micro adenomas
Time frame: completed
To identify the clinical and genetic features of pituitary tumors by investigating their inheritance pattern and possible association with other conditions in the families of the patients.
Genetic studies of pituitary and hypothalamic tumors or related disorders
Time frame: ongoing
To evaluate the cognitive, psychological, and patient-reported health status of mental and social well-being and symptoms of adrenal insufficiency associated with Cushing syndrome in children with this disease.
Descriptive study of cognitive, psychological, and patient-reported health status of mental and social well-being associated with Cushing and after treatment
Time frame: ongoing
To collect peripheral blood samples and tumor tissues and examine the molecular genetics of the specimens, in an effort to elucidate developmental pathways leading to pituitary gland oncogenesis and/or other defects
Genetic studies of pituitary and hypothalamic tumors or related disorders
Time frame: ongoing
Plan to share: Yes — Deidentified Samples/data, including genomic data, will be shared with NIH \& non-NIH collaborators after an MTA is obtained. If coded data shared, the key code will not be shared with collaborators \& stay at NIH. If Data \& samples shared with AIs and IC with an FWA/operating under the DOH and reported at the time of CR. Sharing with AIs without an FWA/operating under the DoH will be submitted for prospective IRB approval. Submissions to NIH-sponsored or supported databases and repositories will be reported at the time of CR. Submission to non-NIH sponsored or supported databases \& repositories will be submitted for prospective IRB approval.@@@@@@Required approvals from the collaborating institution will be obtained and materials will be shipped in accordance with NIH and federal regulations. This study will comply with the NIH GDS Policy, which applies to all NIH-funded research that generates large-scale human or non-human GD, as well as the use of these data for subsequent research.
Supporting information: Study protocol
Time frame: AIs in the NIH intramural program may participate with NIH/ non-NIH collaborators under which de-identified human materials are transferred from the intramural program to another site for correlative studies that are part of the approved protocol. In this case, the protocol clearly documents the tests conducted under the correlative studies\& each institution participating in the clinical study is bound by the terms of this Protocol\& their obligations under the statutes \& regulations. @@@@@@This study will comply with the NIH DSP \&Policy on the Dissemination of NIH-Funded CT Information \& Registration \& Results Information Submission rule. As such, this trial will be registered at CT.gov,\& results information from this trial will be submitted to CT.gov. In addition, every attempt will be made to publish results in peer-reviewed journals. Data from this study may be requested from other researchers 3 years after the completion of the primary endpoint by contacting PI or NICHD-DASH.
Access criteria: This study will comply with the NIH Data Sharing Policy and Policy on the Dissemination of NIH-Funded Clinical Trial Information and the Clinical Trials Registration and Results Information Submission rule. As such, this trial will be registered at ClinicalTrials.gov, and results information from this trial will be submitted to ClinicalTrials.gov. In addition, every attempt will be made to publish results in peer-reviewed journals. Data from this study may be requested from other researchers 3 years after the completion of the primary endpoint by contacting PI or NICHD-DASH.
Started Apr 1997 (actual).
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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