Pediatric Thyroid Center Clinical Research Studies
Current thyroid studies
Triac Phase II Trial
The Triac Trial II is an international, multi-center clinical trial involving 19 centers across North America and Europe to evaluate the effect of a thyroid hormone analogue named Triac in children younger than 30 months of age with MCT8 deficiency. Primary outcome measures include developmental tests, such as the Bayley Scale of Infant Development (BSID) and the Gross Motor Function Measure (GMFM).
Enrollment closed at the beginning of summer 2022. Patients enrolled on the trial are currently being treated, and data analysis is ongoing.
Triac Phase III Withdrawal Trial
The Triac Phase III Withdrawal Trial is a double-blinded, placebo-controlled withdrawal study which aims to evaluate the effects of Triac on thyroid hormone levels in patients older than 4 years of age with MCT8 deficiency. This is a multi-center study involving three sites: two in the United States (CHOP and Oregon Health & Science University [OHSU] in Portland, Oregon) and one in Europe. This study seeks to enroll 16 patients, and recruitment is scheduled to begin in the fall of 2022.
In this study, participants will begin with treatment of Triac. After several weeks of treatment, participants will be randomized to either continue treatment with Triac or begin to take a placebo for four weeks. Neither doctors nor caregivers will know which treatment the children are assigned to for these four weeks. At the end of these four weeks, all patients will resume treatment with Triac for a follow-up period.
Child and Adolescent Thyroid Consortium
The Child and Adolescent Thyroid Consortium (CATC) is a CHOP-initiated effort that began as a single-site project in 2017. The project’s original function was to serve as a data collection mechanism for the CHOP Thyroid Center to gather information pertaining to a variety of thyroid disorders and cancers.
In 2019, the protocol was expanded to encompass a consortium of collaborating institutions, including an Executive Board comprising five institutions — Harvard/Boston Children’s Hospital, MD Anderson Cancer Center, The Hospital for Sick Kids in Toronto, and Yale New Haven Hospital. The Executive Board worked to create a clinical registry database to collect information pertaining to pediatric thyroid cancer.
By 2021, the CHOP thyroid tissue biorepository was added: a tissue bank of biospecimens from patients who underwent thyroid surgeries at CHOP. These tissues are studied under the direction of Aime T. Franco, PhD, Director of the Translational Thyroid Laboratory at CHOP.
The goals of the CATC include:
- Expansion to 25+ institutions across the globe that have expressed interest in joining
- Expansion to include biospecimens from participating institutes, including thyroid tissue, blood and saliva from patients at all CATC member-sites
- Expansion to include patient populations beyond pediatric thyroid nodules/thyroid cancer to evaluate other pediatric thyroid disorders
The CATC biorepository allows us to explore genetic changes in different subtypes of thyroid cancer. Existing specimens from institutions participating in the CATC will be sent to the CATC Biorepository at CHOP for storage in the repository. Prospective specimens will be obtained at the time of the patient’s surgery or biopsy and will be sent to the CATC Biorepository. Formalin-fixed paraffin-embedded (FFPE) and flash-frozen specimens are used for subsequent cellular, biochemical, and/or genetic analysis, including RNA and DNA sequencing to identify driver alterations and differences in target gene expression.
We are currently working to add a web-based, patient-reported sociodemographic survey instrument to expand our understanding of diversity and potential discrimination in access to care for pediatric patients with thyroid disease.
Our patients and families play an essential role in our global research efforts to identify new ways to treat thyroid cancer and thyroid-related conditions through study participation, advocacy, donations, and education.
If you are interested in providing support to the CATC, please email firstname.lastname@example.org.
Targeted Therapy to Increase RAI Uptake in Metastatic Papillary Thyroid Cancer
This prospective, interventional clinical trial will investigate whether patients diagnosed with papillary thyroid cancer (PTC) and distant metastasis to the lungs benefit from treatment with targeted chemotherapies. The study will examine whether treatment with an oncogene-specific inhibitory targeted therapy will increase the avidity and success of radioactive iodine therapy, which is the standard-of-care treatment for thyroid cancer patients.
Distant metastasis must be confirmed with CT or MRI imaging for participants to enroll in this trial. This is a multi-center study with the Advanced Pediatric Thyroid Cancer Therapeutics Clinic at CHOP serving as the sponsoring site under the co-direction of Theodore Laetsch, MD, and Andrew J. Bauer, MD.
Psychosocial Functioning in Patients with Autoimmune Hyperthyroidism
This is a prospective, longitudinal study to assess how children and adolescents diagnosed with Graves’ disease (autoimmune hyperthyroidism) and are seeking definitive surgical treatment faired mentally, emotionally and socially before and after surgery.
Participants 12-19 years old pursuing definitive surgery for Graves’ disease and their parents were recruited to complete surveys before and at least six months following total thyroidectomy surgery. Surveys assessed motivations for pursuing surgery, quality-of-life, perceived stigmatization, self-esteem, scar appearance, and satisfaction with surgery.
Effects of Hyper- and Hypothyroidism on Driving Behavior in Adolescents
This prospective, observational cohort study is designed to determine whether hyperthyroidism and/or hypothyroidism have an impact on driving behaviors and outcomes. Participants with a valid driver’s license or permit who are experiencing hyper- or hypothyroidism are recruited to complete a virtual driving test when their thyroid stimulating hormone (TSH) is outside normal range and again when their TSH returns to normal range. Results will be compared to an existing normative dataset of typical young new drivers to determine what skill deficits, if any, are present in this population.
Relevant Peer-Reviewed Online Publications
- Fusion Oncogenes are Associated with Increased Metastatic Capacity and Persistent Disease in Pediatric Thyroid Cancers
- NTRK Fusions Identified in Pediatric Tumors: The Frequency, Fusion Partners and Clinical Outcome
- Oncogene-specific Inhibition in the Treatment of Advanced Pediatric Thyroid Cancer
- The Clinical Aspect of NTRK-fusions in Pediatric Papillary Thyroid Cancer
- Molecular Testing for Oncogenic Gene Alterations in Pediatric Thyroid Lesions
- Bilateral Papillary Thyroid Cancer in Children: Risk Factors and Frequency of Postoperative Diagnosis
- The Clinical Spectrum of PTEN Hamartoma Tumor Syndrome: Exploring the Value of Thyroid Surveillance