An Investigation of Pituitary Tumors and Related Hypothalmic Disorders
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| ClinicalTrials.gov Identifier: NCT00001595 |
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Recruitment Status :
Recruiting
First Posted : November 4, 1999
Last Update Posted : February 6, 2023
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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)<TAB>training our fellows and students in the identification of genetic defects associated with pituitary tumor formation, and
(ii)<TAB>teaching our fellows and students the recognition, management and complications of pituitary tumors
As a research study, this protocol aims at
(i)<TAB>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)<TAB>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....
| Condition or disease | Intervention/treatment |
|---|---|
| Panhypopituitarism Gigantism/Acromegaly Prolactinoma Cushing Disease | Procedure: MRI Procedure: Tissue specimen collection |
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 DNA 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 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.
Endpoints:
<TAB>
Primary Endpoint:
Molecular genetic testing, whole exome sequencing
Secondary Endpoints:
Pre-post treatment comparison of research variables related to pituitary/hypothalamic tumors and comorbidities.
Cognitive, behavioral, psychological, and patient-reported outcomes
| Study Type : | Observational |
| Estimated Enrollment : | 2000 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | A Clinical and Genetic Investigation of Pituitary and HYPOTHALAMIC Tumors and Related Disorders |
| Actual Study Start Date : | April 21, 1997 |
| Group/Cohort | Intervention/treatment |
|---|---|
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Patients with pituitary tumors or hypothalmic defects
Patients with pituitary tumors or hypothalmic defects
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Procedure: MRI Procedure: Tissue specimen collection |
- Molecular genetic testing, whole exome sequencing [ Time Frame: ongoing ]To collect peripheral blood DNA 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.
- To investigate the usefulness of a new MRI of the pituitary gland [ Time Frame: completed ]Comparison of MRI techniques to identify micro adenomas
- 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, [ Time Frame: ongoing ]Genetic studies of pituitary and hypothalamic tumors or related disorders
- 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. [ Time Frame: ongoing ]Descriptive study of cognitive, psychological, and patient-reported health status of mental and social well-being associated with Cushing and after treatment
- to collect peripheral blood DNA 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 ]Genetic studies of pituitary and hypothalamic tumors or related disorders
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 3 Years to 70 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
| Sampling Method: | Non-Probability Sample |
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INCLUSION CRITERIA:
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, aged 3 years to 70 years
- Evidence for the existence of a tumor of the hypothalamic-pituitary unit, as indicated by previously obtained imaging studies or biochemical investigation of the hypothalamohypophyseal function.
- Family members (any age) of patients with a family history of tumors and who will agree to participate in the DNA/linkage analysis study.
- Member of a kindred suspected of having an inherited form of pituitary neoplasia, as evidenced by results of a patient enrolled in this protocol.
- Ability of subject or Legally Authorized Representative (LAR)) to understand and the willingness to sign a written informed consent document.
EXCLUSION CRITERIA:
1. Pregnancy.
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): NCT00001595
| Contact: Samah M Agabein | (301) 451-7615 | samah.agabein@nih.gov | |
| Contact: Christina Tatsi, M.D. | (301) 451-7170 | christina.tatsi3@nih.gov |
| United States, Maryland | |
| National Institutes of Health Clinical Center | Recruiting |
| Bethesda, Maryland, United States, 20892 | |
| Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY dial 711 ccopr@nih.gov | |
| Principal Investigator: | Christina Tatsi, M.D. | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
| ClinicalTrials.gov Identifier: | NCT00001595 |
| Other Study ID Numbers: |
970076 97-CH-0076 |
| First Posted: | November 4, 1999 Key Record Dates |
| Last Update Posted: | February 6, 2023 |
| Last Verified: | February 1, 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | .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 Materials: |
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. |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Developmental Defect Oncogenesis Evaluation and Management |
Psychological Cushing Disease Natural History |
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Acromegaly ACTH-Secreting Pituitary Adenoma Prolactinoma Gigantism Pituitary ACTH Hypersecretion Pituitary Diseases Hypothalamic Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Endocrine System Diseases Bone Diseases, Endocrine |
Bone Diseases Musculoskeletal Diseases Hyperpituitarism Adenoma Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms Pituitary Neoplasms Endocrine Gland Neoplasms Neoplasms by Site Bone Diseases, Developmental |