Pre-pubertal Low Dose Transdermal Estradiol in Turner Syndrome

NCT07784582 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: STUDY00002034
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This is a research study to find out if treatment with transdermal estradiol patches started between 8 to 11.5 years is safe in girls with Turner syndrome who have ovarian failure and to see whether it may improve performance on two tests of working speed and short-term memory.

Conditions

Turner Syndrome; Primary Ovarian Insufficiency

Keywords

Turner syndrome; Pre-pubertal; Transdermal estradiol; Primary Ovarian Insufficiency

Outcome Measures

Primary Outcomes (2)

• Pattern Comparison processing speed test

  Changes in the Pattern Comparison processing speed test (PC) at 6 months on the NIH toolbox EnglishV3 compared to baseline. Pattern Comparison processing speed test (PC) is a measure of the speed of information processing. Participants see a pair of simple pictures on the iPad screen and must quickly discern whether the pictures are the same or different. The total raw score is the number of items answered correctly during the 80 seconds of presentation time (Max 133 items). The raw score is converted to a age-adjusted normative scores. The Median Response Time Per Item is also available.

  6 months

• List Sorting Working Memory (LSWM) test

  Changes in the List sorting working memory test on the NIH toolbox EnglishV3 at 6 months compared to baseline. The List Sorting Working Memory test measures a participant's working memory, the ability to hold information in a short-term buffer and then manipulate the information. Pictures and written names of different foods and animals are displayed with accompanying audio-recorded and visual prompts, and the participant is asked to say the items back in size order from smallest to largest, first within a single dimension and then on two dimensions. The raw score (up to 24 Max) is then converted to age-adjusted normative scores.

  6 months

Secondary Outcomes (4)

• Breast Tanner stage

  6 months

• Pubertal hormone changes

  6 months

• LH

  6 months

• FSH

  6 months

Other Outcomes (2)

• Height velocity

  6 months

• Lipid profile

  6 months

Study Arms (1)

Estradiol Treatment

EXPERIMENTAL

Patients opt to receive 6 months of transdermal ultra low dose estradiol treatment

Drug: Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch

Interventions

Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch DRUG

Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch will be applied and changed weekly for 6 months

Estradiol Treatment

Eligibility Criteria

• Age: 8 Years - 12 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Female, aged 8 years up to 11 y and 180 days with karyotype confirmed Turner syndrome
• Ability to take the transdermal estradiol medication and be willing to adhere to the treatment regimen
• Ability to complete the primary endpoint testing in English. Non-English speakers may be recruited if the participant is capable of performing the English version of the tests.

You may not qualify if:

• Spontaneous thelarche with breast Tanner stage 3 or more, spontaneous menarche or history of abnormal uterine bleeding.
• Previous estrogen exposure
• Cognitive impairment is severe enough to prevent assessment of the primary outcome measures.
• Untreated hypothyroidism or celiac disease: Individuals may be recruited if on treatment and stable medication dose in past 3 months.
• Subjects known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
• History of any type of malignancy
• History of a known clotting disorder/deep venous thrombosis
• Any clinically significant abnormality as determined by the principal investigator.

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Turner Syndrome; Primary Ovarian Insufficiency

Condition Hierarchy (Ancestors)

Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases; Ovarian Diseases; Adnexal Diseases; Genital Diseases, Female; Genital Diseases

Central Study Contacts

Roopa Kanakatti Shankar, MBBS, MS

CONTACT

202-476-2121; roopa.shankar@childrensnational.org

Kimberly Pitner, MSHS, BSN, RN

CONTACT

202-476-1403; kboucher@childrensnational.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Pilot trial for biomarkers and safety

Study Record Dates

• First Submitted: August 5, 2026
• First Posted: August 25, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: August 25, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)