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Protocol Details

Thyroid Hormone to Induce Non-Insulin Mediated Glucose Disposal in Patients with Insulin Receptor Mutations

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

15-DK-0119

Sponsoring Institute

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 12 Years
Max Age: 65 Years

Referral Letter Required

No

Population Exclusion(s)

None

Keywords

Brown Adipose Tissue;
Liothyronine;
Insulin Receptors

Recruitment Keyword(s)

None

Condition(s)

Insulin Resistance;
Diabetes Mellitus;
Abnormal Glucose Metabolism

Investigational Drug(s)

None

Investigational Device(s)

None

Intervention(s)

Drug: Liothyronine

Supporting Site

National Institute of Diabetes and Digestive and Kidney Diseases

Background:

- Insulin receptor mutation causes high blood sugars and sometimes diabetes complications. Researchers want to see if thyroid hormone helps.

Objectives:

- To see if thyroid hormone treatment changes how the body handles sugar in people with insulin receptor mutation and improves blood sugar in people with diabetes.

Eligibility:

- People ages 12 65 with an insulin receptor mutation.

Design:

- Study part 1:19-day clinic stay. Participants will be monitored for 4 days. Then for 15 days they will take a thyroid hormone pill 3 times a day. Participants will have:

- Blood tests.

- Heart rate and skin temperature monitored.

- All their food provided.

- Two 5-hour sessions in a special room. They will wear special clothes and sometimes sit still.

- Two small tubes inserted in veins. One will deliver tiny amounts of sugar and fat with a non-radioactive tracer. Participants will also drink water with a tracer. The other tube will collect blood.

- A sweet drink. Participants may have finger stick blood sugar tests.

- Glucose-monitoring device inserted into body fat for two 24-hour periods.

- Adults may have samples of fat and muscle taken.

- Heart ultrasound.

- PET-CT scan in a machine. An intravenous catheter will be placed in an arm vein. A small amount of radioactive substance will be injected.

- DEXA scan of body fat and bone density.

- Participants with poorly controlled diabetes will then take thyroid hormone at home for 6 months. They will have blood drawn and sent to the study team monthly.

- After about 3 months, they will have an overnight visit. After 6 months, they will have a 4-day visit.

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Eligibility

2 WEEKS STUDY:

INCLUSION CRITERIA:

1. Mutation of the insulin receptor (either recessive or dominant negative). If mutation status is not known prior to enrollment, subjects will undergo genotyping at enrollment. In the unanticipated event that a patient does not have a mutation of the insulin receptor, he or she will not complete the study and his or her data will not be included in the analysis.

2. Age 12 to 65 years old

EXCLUSION CRITERIA:

1. Changes in doses of diabetes medications (including metformin, insulin, sulfonylureas, thiazolidinediones, leptin, GLP-1 agonists, DPP4 inhibitors, etc.) in the preceding 10 weeks.

2. Any medical condition or medication that will increase risk to the subject (e.g. ischemic or structural heart disease, congestive heart failure, uncontrolled hypertension, or arrhythmia) or that will interfere with interpretation of study data.

3. Disorders that would lead to erratic gastrointestinal absorption or loss of thyroid hormone from the gut (severe diarrhea, celiac disease, use of bile acid sequestrants, excessive consumption of soybean products).

4. Any form of endogenous hyperthyroidism or hypothyroidism at baseline.

5. Current or recent (past 8 weeks) use of thyroid hormone or anti-thyroid drugs.

6. Extreme disorders of thyroid hormone binding to thyroid binding globulin (excess or deficiency) or protein loss (nephrotic range proteinuria) that would lead to difficulties achieving a consistent thyroid hormone level for study.

7. Known presence of a rare clinical disorder that leads to thyroid hormone insensitivity (known T3 receptor mutations, selenocysteine insertion sequence-binding protein 2 (SBP2) abnormalities, monocarboxylate transporter defects).

8. Current use of beta blockers

9. Pregnancy or breast feeding

10. Any EKG abnormality that could increase risk of T3 treatment (resting sinus tachycardia (age adjusted norms), atrial fibrillation, myocardial ischemia, left or right ventricular excitation block, left ventricular hypertrophy or extrasystoles)

11. Known allergy or hypersensitivity to any form of thyroid hormone

12. Known adrenal insufficiency

13. Dependence on oral anticoagulant medications (adults only)

14. Use of tricyclic anti-depressants, as transient cardiac arrhythmias have been observed with the concomitant use of thyroid hormone.

15. Use of cholestyramine.

16. History of clinically significant osteoporosis per investigator judgment (e.g. previous fragility fracture)

6 MONTHS STUDY:

Patients must meet all inclusion and exclusion criteria for the short-term study, plus have poorly controlled diabetes, defined as a hemoglobin A1c greater than or equal to 7%.


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Citations:

Kushchayeva YS, Startzell M, Cochran E, Auh S, Sekizkardes H, Soldin SJ, Kushchayev SV, Dieckmann W, Skarulis M, Abdul Sater Z, Brychta RJ, Cypess AM, Lin TC, Lightbourne M, Millo C, Brown RJ. Thyroid Hormone Effects on Glucose Disposal in Patients With Insulin Receptor Mutations. J Clin Endocrinol Metab. 2020 Mar 1;105(3):e158 71. doi: 10.1210/clinem/dgz079. PMID: 31588494; PMCID: PMC7093053.

Sekizkardes H, Chung ST, Chacko S, Haymond MW, Startzell M, Walter M, Walter PJ, Lightbourne M, Brown RJ. Free fatty acid processing diverges in human pathologic insulin resistance conditions. J Clin Invest. 2020 Jul 1;130(7):3592-3602. doi: 10.1172/JCI135431. PMID: 32191645; PMCID: PMC7324196.

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Contacts:

Principal Investigator

Referral Contact

For more information:

Rebecca J. Brown, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIHBC 10 - CRC BG RM 6-5942
10 CENTER DR
BETHESDA MD 20892
(301) 594-0609
brownrebecca@mail.nih.gov

Rebecca J. Brown, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIHBC 10 - CRC BG RM 6-5942
10 CENTER DR
BETHESDA MD 20892
(301) 594-0609
brownrebecca@mail.nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1
ccopr@nih.gov

Clinical Trials Number:

NCT02457897

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