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Pre-pubertal Low Dose Transdermal Estradiol in Turner Syndrome

24 augusti 2026 uppdaterad av: Children's National Research Institute

Ultra-low Dose Transdermal Estradiol Therapy in Prepubertal Girls With Turner Syndrome and Primary Ovarian Insufficiency.

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.

Studieöversikt

Status

Har inte rekryterat ännu

Betingelser

Intervention / Behandling

Detaljerad beskrivning

The investigators hypothesize that ultra-low dose TDE will improve neurocognitive measures without a negative impact on growth and pubertal maturation in the peripubertal period. To test this hypothesis, investigators will conduct a prospective single center pilot trial and recruit up to 15 girls with TS and POI (FSH>10 in the past year), ages 8 to <11.5 years who will receive ultra-low dose TDE [defined as 1/8th patch of 0.025mg/24h applied weekly (delivering 3.125mcg/24h)] for 6 months to address the following objectives.

Primary: Study the effect of ultra-low dose TDE therapy on processing speed and working memory. The investigators will administer the Pattern Comparison Processing Speed Test as well as the List Sorting Working Memory test NIH toolbox EnglishV3 at baseline, and 6-months of TDE treatment.

Secondary: Assess change in pubertal status, FSH, LH and estradiol levels with ultra-low dose TDE therapy. Height, weight measurements, assessment of Tanner staging for breast development, laboratory measurements of ultrasensitive estradiol, FSH, LH, lipid profile at baseline and 6-months of TDE treatment will be completed

Studietyp

Interventionell

Inskrivning (Beräknad)

15

Fas

  • Fas 2

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studera Kontakt Backup

Studieorter

  • Förenta staterna
    • District of Columbia
      • Washington D.C., District of Columbia, Förenta staterna, 20010
        • Children's National Hospital
        • Kontakt:
        • Huvudutredare:
          • Roopa Kanakatti Shankar, MBBS, MS

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Barn

Tar emot friska volontärer

Nej

Beskrivning

Inclusion Criteria:

  1. Provision of signed and dated informed consent form
  2. Stated willingness to comply with all study procedures and availability for the duration of the study
  3. Female, aged 8 years up to 11 y and 180 days with karyotype confirmed Turner syndrome
  4. FSH result> 10miu/mL in the past 12 months. Since variability in FSH is not uncommon, a single measure of FSH >10mIU/mL in the past calendar year will be deemed sufficient for inclusion into the study even if repeat measures may be under 10mIU/mL.
  5. Ability to take the transdermal estradiol medication and be willing to adhere to the treatment regimen
  6. 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.

Exclusion Criteria:

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

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Estradiol Treatment
Patients opt to receive 6 months of transdermal ultra low dose estradiol treatment
Läkemedel: Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch
Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch will be applied and changed weekly for 6 months

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Pattern Comparison processing speed test
Tidsram: 6 months
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
Tidsram: 6 months
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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Breast Tanner stage
Tidsram: 6 months
Changes in breast sexual maturity rating (Tanner staging) on examination over 6 months with ultra-low dose estradiol treatment
6 months
Pubertal hormone changes
Tidsram: 6 months
Changes in serum estradiol (measured in pg/mL), after 6 months of ultra-low dose estradiol treatment compared to baseline.
6 months
LH
Tidsram: 6 months
Luteinizing hormone (LH) (measured in U/L) over 6 months of estradiol therapy
6 months
FSH
Tidsram: 6 months
Change in Follicle stimulating hormone (FSH) measured in U/L over 6 months of estradiol therapy
6 months

Andra resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Height velocity
Tidsram: 6 months
Change in annualized height velocity (in cm/year) over 6 months of treatment with ultra-low dose estradiol. This will be calculated based on height measurements (in cm) at baseline and 6 months.
6 months
Lipid profile
Tidsram: 6 months
Changes in lipid profile (total cholesterol, triglycerides, high density lipoproteins, low density lipoproteins) over 6 months of treatment with ultra-low dose estradiol will be assessed.
6 months

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Children's National Research Institute

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Beräknad)

1 september 2026

Primärt slutförande (Beräknad)

1 juni 2027

Avslutad studie (Beräknad)

1 juni 2027

Studieregistreringsdatum

Först inskickad

5 augusti 2026

Först inskickad som uppfyllde QC-kriterierna

24 augusti 2026

Första postat (Faktisk)

25 augusti 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

25 augusti 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

24 augusti 2026

Senast verifierad

1 augusti 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • STUDY00002034

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

IPD-planbeskrivning

Patient Privacy and on account of being a pilot trial

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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