Tuberous Sclerosis Complex Natural History Study: Renal Manifestations
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ClinicalTrials.gov Identifier: NCT00598455 |
Recruitment Status
:
Completed
First Posted
: January 22, 2008
Last Update Posted
: May 4, 2017
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Condition or disease |
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Tuberous Sclerosis Complex |
Objectives.
Our objective is to test the hypothesis that serial MR and CT imaging will allow objective, reproducible quantification of angiomyolipoma growth by volumetric analysis, and analysis of lesions characteristics will identify angiomyolipomata with rapid growth potential that would require intervention.
The specific aim of this proposal is to collect clinically obtained serial abdominal imaging from the Tuberous Sclerosis Natural History Consortium Centers and analyze the volume and adiposity of the individual angiomyolipomata. The growth rate is hypothesized to have an inverse relationship to adiposity. The prognostic value of identifying lesions with aggressive growth characteristics is very large, and intervention can be instituted early in order to reduce the renal damage.
Study Population.
- The target population for this study will be patients with tuberous sclerosis who attend a tuberous sclerosis clinic that is part of the consortium. The data collected will include routine imaging data, age, gender, and if know, the genotype. Approximately 855 patients throughout the United States will be asked to participate in this natural history study and 450 of those are anticipated to consent and have complete data on 3 years of CT and MR Imaging Scans to measure angiomyolipoma growth and adiposity.
- Patients attending the tuberous sclerosis clinics that are members of the consortium will be asked if they would be involved in the study.
- Imaging will be obtained as part of the standard of care at the Consortium Centers. Imaging done on pregnant patients will not be excluded. MRI has been used now extensively for pregnant patients.
Protocol Design.
This is a natural history study involving the imaging characteristics of angiomyolipomata found in patients with tuberous sclerosis complex. This study offers the potential benefit that the lesion characteristics and growth rate will be monitored. Patel et al. posit that growth rates of greater than 0.5 cm/year for solid tumors are worrisome for malignancy. Patients harboring lesions that exhibit faster growth rates will be identified so that they can be more carefully monitored. During the course of the study, should we detect concerning features, the Consortium Center TS Clinic Director will be alerted. Imaging modalities that may be used include CT scans. The patient data being collected will be done so for clinical reasons, and every effort will be made to use the least possible radiation exposure. There are several ways to reduce the exposure, and these limiting techniques will be employed for each scan. The scans will be performed both pre and post contrast using an agent such as Optiray® so that vascularity can be assessed. Patients may also be imaged with a 1.5 Tesla magnet in a supine position using a phased array torso coil or body coil, depending on patient size. When compatible, respiratory compensation will be used to diminish respiratory artifact. Overall imaging time for the following sequences will be approximately 45 minutes.
a. Recruitment process: Patients will be recruited from patients seen at the Tuberous Sclerosis Natural History Consortium Centers, including Cincinnati Children's Hospital, Loma Linda University, University of Pennsylvania Medical Center, Connecticut Children's Medical Center, Vanderbilt University, Columbus Children's, Texas Scottish Rite Hospital, Massachusetts General Hospital, Miami Children's Hospital, Minnesota Epilepsy Group, Dartmouth University, Washington Children's Hospital, New York University School of Medicine, University of Texas, Houston, Children's Hospital, Boston, and Children's Hospital, Pittsburgh.
Study Type : | Observational |
Estimated Enrollment : | 450 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Tuberous Sclerosis Complex Natural History Study: Renal Manifestations |
Study Start Date : | February 2008 |
Actual Primary Completion Date : | September 2013 |
Actual Study Completion Date : | September 2013 |


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Ages Eligible for Study: | 7 Years to 65 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Probability Sample |
Inclusion Criteria:
- Patients who receive yearly follow-up at their respective tuberous sclerosis clinic. Such clinics have a director who is knows their patients well.
- Patients known to have an angiomyolipoma between the ages of 7-
- 65 years
- Pregnant women to be included if tested with MR
Exclusion Criteria:
- Patients who are not seen annually and therefore do not have yearly imaging.
- Patients who do not have an angiomyolipoma.
- Patients who are not likely to follow-up as recommended.
- Use of an investigational drug, including rapamycin, within the last 30 days.
- Pregnant women to be excluded if they cannot be tested with MR

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): NCT00598455
United States, Ohio | |
Cincinnati Children's | |
Cincinnati, Ohio, United States, 45229 |
Principal Investigator: | John J Bissler, M.D. | Children's Hospital Medical Center, Cincinnati |
Responsible Party: | Children's Hospital Medical Center, Cincinnati |
ClinicalTrials.gov Identifier: | NCT00598455 History of Changes |
Other Study ID Numbers: |
TS0500008 W81XWH-06-1-0538 |
First Posted: | January 22, 2008 Key Record Dates |
Last Update Posted: | May 4, 2017 |
Last Verified: | May 2017 |
Keywords provided by Children's Hospital Medical Center, Cincinnati:
angiomyolipomata tuberous sclerosis complex |
Additional relevant MeSH terms:
Sclerosis Tuberous Sclerosis Pathologic Processes Hamartoma Neoplasms Neoplasms, Multiple Primary Neoplastic Syndromes, Hereditary Malformations of Cortical Development, Group I |
Malformations of Cortical Development Nervous System Malformations Nervous System Diseases Neurocutaneous Syndromes Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Congenital Abnormalities Genetic Diseases, Inborn |