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Completed

NCT Number: NCT00335062

TSH Receptor Antibody Heterogeneity in Children and Adolescents With Graves' Disease

Graves' disease, the most common form of hyperthyroidism in children, is caused by Thyrotropin (TSH) Receptor Antibodies (TRAbs) that mimic the action of TSH. The disease leads to significant morbidity in children both due to the prolonged course of antithyroid medication often required for sustained immunological remission and the high risk of relapse when medication is withdrawn. The ability to predict which patients are most likely to fail medical management would greatly improve the choice of therapy. In the past, large goiter size, age at diagnosis, increased biochemical severity, and decreased body mass index have all been associated with a poorer prognosis, but these clinical indicators lack sensitivity and specificity. Preliminary data suggest that the new TRAb assays are both sensitive and specific for the measurement of TRAbs in children with Graves' disease. In addition, variation in these antibodies over time is not the same in all patients. The goal of this proposal will be to prospectively follow children with newly diagnosed Graves' disease and use microarray technology to determine if there are genes whose expression differ in patients who respond to medical therapy versus those who will need more definitive therapy earlier in their disease.

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Key information

Conditions

Graves' Disease Autoimmune Diseases Endocrine System Diseases Exophthalmos Eye Diseases Goiter Graves Disease Hyperthyroidism Immune System Diseases Orbital Diseases Thyroid Diseases

Age range

2 year–21 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Childrens' Hospital Boston

Boston, Massachusetts, 02115, United States

About this study

In the present grant proposal, we plan to utilize two new assays (binding and bioassay) in order to identify additional predictors of Graves' disease and apply them to a well characterized group of patients with Graves' disease followed prospectively. More specifically, we plan to further investigate the antibodies by measuring lambda: kappa light chain antibody ratios in pediatric patients. We will assess epitope heterogeneity by using novel chimeric proteins in which specific portions of the TSH receptor have been replaced with the closely related LH receptor. We will utilize microarray technology to determine if there are differences in gene expression profiles in responders versus non responders. It is hoped that these methods will lead to an improved ability to follow disease progression and to monitor efficacy of therapy.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age 2-21 years
  • Suppressed Thyroid Stimulating Hormone (TSH)
  • Elevated Triiodothyronine (T3), Thyroxine (T4)

Exclusion criteria

  • Pregnancy
  • Toxic Nodule
  • Currently receiving steroids or thyroid hormone replacement
  • Bacterial, Viral, Radiation, or Autoimmune thyroiditis

Treatment and study plan

Primary outcomes

  1. The primary outcome will be the disappearance of TSH receptor Abs (as assessed by both ELISA and bioassay) from the circulation.

    Time frame: end of study

Secondary outcomes

  1. 2) The secondary outcome will be normalization of thyroid function tests (T4, free T4, Total T3, and TSH) on a low dose of Tapazole 2.5-5.0 mg per day.

    Time frame: end of study

Other outcomes

  1. 3) In the neonatal Graves' disease patient, the primary outcome will be the clearance of both TBII and TSI from the infant's sera (as assessed by both ELISA and bioassay).

    Time frame: end of study

Sponsors and collaborators

Lead sponsor

Boston Children's Hospital

Other

Registry information

Important dates

Study start
2005
Primary completion
2010
Study completion
2010
First posted
Jun 8, 2006
Registry last updated
Jul 25, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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