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Pre-pubertal Low Dose Transdermal Estradiol in Turner Syndrome

24. august 2026 oppdatert 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.

Studieoversikt

Status

Har ikke rekruttert ennå

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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

Studietype

Intervensjonell

Registrering (Antatt)

15

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studer Kontakt Backup

Studiesteder

  • Forente stater
    • District of Columbia
      • Washington D.C., District of Columbia, Forente stater, 20010
        • Children's National Hospital
        • Ta kontakt med:
        • Hovedetterforsker:
          • Roopa Kanakatti Shankar, MBBS, MS

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn

Tar imot friske frivillige

Nei

Beskrivelse

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Estradiol Treatment
Patients opt to receive 6 months of transdermal ultra low dose estradiol treatment
Legemiddel: 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

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Pattern Comparison processing speed test
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Breast Tanner stage
Tidsramme: 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
Tidsramme: 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
Tidsramme: 6 months
Luteinizing hormone (LH) (measured in U/L) over 6 months of estradiol therapy
6 months
FSH
Tidsramme: 6 months
Change in Follicle stimulating hormone (FSH) measured in U/L over 6 months of estradiol therapy
6 months

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Height velocity
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Children's National Research Institute

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

1. september 2026

Primær fullføring (Antatt)

1. juni 2027

Studiet fullført (Antatt)

1. juni 2027

Datoer for studieregistrering

Først innsendt

5. august 2026

Først innsendt som oppfylte QC-kriteriene

24. august 2026

Først lagt ut (Faktiske)

25. august 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

25. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

24. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • STUDY00002034

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

IPD-planbeskrivelse

Patient Privacy and on account of being a pilot trial

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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