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Treatment Trial of Subclinical Hypothyroidism in Down Syndrome

Information source: Children's Hospital of Philadelphia
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Down Syndrome; Subclinical Hypothyroidism

Intervention: Levothyroxine (Drug)

Phase: N/A

Status: Recruiting

Sponsored by: Children's Hospital of Philadelphia

Official(s) and/or principal investigator(s):
Andrea Kelly, MD, MSCE, Principal Investigator, Affiliation: Children's Hospital of Philadelphia

Overall contact:
Divya Prasad, MPH, Phone: 267-426-2778, Email: prasadd@email.chop.edu


The purpose of this research study is to learn about the effects of treating subclinical hypothyroidism (SCH) with thyroid hormone replacement in children and adolescents with Down syndrome (DS). We hypothesize that treatment of SCH with thyroid hormone replacement will improve cardiometabolic health and quality of life.

Clinical Details

Official title: Levothyroxine Treatment and Cardiometabolic Outcomes in Adolescents With Down Syndrome

Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver), Primary Purpose: Treatment

Primary outcome: Change in non-HDL cholesterol from baseline at 6, 12 and 18 months.

Secondary outcome: Change in quality of life from baseline at 6, 12 and 18 months.

Detailed description: The American Academy of Pediatrics (AAP) recommends yearly screening of thyroid studies in DS. Clinical experience suggests that TSH concentrations in the subclinical hypothyroid range (5-10 milli international units(mIU/L)) are not uncommon in DS, but the benefits and risks of treating SCH in the DS population are not known. In adults, SCH has been associated with increased cardiometabolic risk (CMR) and individuals with DS may be at increased cardiometabolic risk as well. Data in children with SCH are limited. Despite the recommendations to screen for thyroid dysfunction, evidence to guide management of elevated TSH in children with DS is equally sparse. In non-DS children, TSH>4. 65 mIU/L was associated with lower HDL. One year of levothyroxine treatment in short children with subclinical hypothyroidism and short stature improved growth velocity. Left ventricular (LV) function and LV mass (by echocardiography) was not different in 16 children with DS and subclinical hypothyroidism (TSH>6. 5 mIU/L; mean TSH = 7. 8 mIU/L) vs. 25 children with DS and normal TSH. However, these findings may be limited by the small sample size. An intervention study of 7 subjects age 2-42 years with DS and hypothyroidism, defined as low T4 and normal or elevated TSH (0. 2-18. 9 mIU/L) on 8 weeks of levothyroxine treatment did not improve developmental or functional outcomes. Anthropometrics and CMR factors were not examined. In contrast, increased TSH in the absence of overt congenital hypothyroidism is common in neonates with DS and prompted a randomized controlled trial (RCT) in 181 neonates with DS. TSH-directed levothyroxine treatment was associated with better growth, weight gain, and motor development after 24 months compared to placebo. These findings highlight that the "asymptomatic" component of subclinical hypothyroidism may have medically-relevant effects. This study will provide potentially clinically relevant preliminary evidence for the treatment of subclinical hypothyroidism in DS.


Minimum age: 8 Years. Maximum age: 20 Years. Gender(s): Both.


Inclusion Criteria:

- Males and females, ages 8 - 20 years

- Diagnosis of Down syndrome

- Subclinical hypothyroidism: TSH level between 5 - 10 mIU/L, normal T4

- Parental/guardian permission (informed consent) and if appropriate, child assent

- Females who are at least 11 years of age or who are menarchal must have a negative

urine/serum pregnancy test

- Committed to adherence to levothyroxine treatment and study completion

Exclusion Criteria:

- Pregnancy

- Type 1/Type 2 diabetes

- Chronic medical conditions or medication use that can affect growth, nutrition, blood

glucose, insulin secretion, or thyroid function (such as lithium or certain seizure medications)

- Current use of levothyroxine or anti-thyroid hormone

- Cyanotic congenital heart disease, or pulmonary hypertension (as described by last

echo report in subjects with CHD), or congenital heart disease considered medically unstable by the study cardiologists

Locations and Contacts

Divya Prasad, MPH, Phone: 267-426-2778, Email: prasadd@email.chop.edu

Children's National Medical Center, Washington, District of Columbia 20010, United States; Recruiting
Sheela Magge, MD, MSCE, Phone: 888-884-2327
Sheela Magge, MD, MSCE, Principal Investigator

The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States; Recruiting
Andrea Kelly, MD, MSCE, Phone: 215-590-3174, Email: kellya@email.chop.edu
Andrea Kelly, MD, MSCE, Principal Investigator

Additional Information

Starting date: January 2013
Last updated: May 4, 2015

Page last updated: August 23, 2015

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