Pre-pubertal Low Dose Transdermal Estradiol in Turner Syndrome

ConditionsTurner Syndrome Primary Ovarian Insufficiency

Trial statusNot yet recruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexFemale

Age8-12

SponsorChildren's National Research Institute

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

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

Treatment groups

15 Participants

are divided into 1 treatment group

Group A: Estradiol TreatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

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.

Time frame

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.

Time frame

6 months

Secondary outcomes

Breast Tanner stage

Changes in breast sexual maturity rating (Tanner staging) on examination over 6 months with ultra-low dose estradiol treatment

Time frame

6 months

Pubertal hormone changes

Changes in serum estradiol (measured in pg/mL), after 6 months of ultra-low dose estradiol treatment compared to baseline.

Time frame

6 months

Luteinizing hormone (LH) (measured in U/L) over 6 months of estradiol therapy

Time frame

6 months

FSH

Change in Follicle stimulating hormone (FSH) measured in U/L over 6 months of estradiol therapy

Time frame

6 months

Other outcomes

Height velocity

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.

Time frame

6 months

Lipid profile

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.

Time frame

6 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's National Research Institute

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: