Phase 3 N=19 Treatment

Primary Ovarian Insufficiency: Phenotype and Optimal Treatment

Primary Ovarian Insufficiency

Bottom Line

View on ClinicalTrials.gov: NCT03568708 ↗

Enrolled (actual)

Serious AEs

5.3%

Results posted

Jun 2024

Primary outcome: Primary: Change in Dual Energy X-ray Absorptiometry (DXA) Measure of Bone Mineral Density (BMD) of the Lumbar Spine — 0.228; 0.673 Z-score

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Transdermal Estrogen (Drug)
Age: Pediatric, Adult · 11+ yrs
Sex: Female
Sponsor: Children's Hospital Medical Center, Cincinnati
Primary completion: Jan 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Dual Energy X-ray Absorptiometry (DXA) Measure of Bone Mineral Density (BMD) of the Lumbar Spine	0.228; 0.673	—
SECONDARY Change in Dual Energy X-ray Absorptiometry (DXA) Measure of Bone Mineral Density (BMD) at the Whole Body Less Head, Total Hip, and Femoral Neck	0.433; 0.815; 0.231; 0.370; 0.337; 0.556	—
SECONDARY Change in Volumetric Bone Mineral Density (vBMD) at the Distal Radius as Measured by Peripheral Quantitative Computed Tomography (pQCT)	-1.245; -5.415; -0.073; 13.945	—
SECONDARY Anthropometrics	25.9; 27.01; 27.64; 27.86	—
SECONDARY Change in Lean Mass as Measured by DXA Body Composition	-0.999; 1.577	—
SECONDARY Change in Symptoms of Anxiety as Measured by Screen for Child Anxiety Related Disorders (SCARED)	8.339; -1.261	—
SECONDARY Change in Symptoms of Depression as Measured by Child Depression Inventory-II (CDI-II)	1.246; -1.187	—
SECONDARY Change in Memory as Assessed by the Children and Adolescent Memory Profile (CHAMP) Total Memory Index Score	17.203; 24.619	—
SECONDARY Change in Quality of Life as Assessed by the Child Health Questionnaire-Child Self-Report Form (CHQ-CF87)	2.710; 5.686	—
SECONDARY Compliance With Transdermal Estrogen Patch	69	—
SECONDARY Study Medications - Serum Estradiol	7.49; 55.41; 98.63	—

Summary

This pilot study will observe the progression of newly diagnosed POI patients physical and psychology outcomes after initiating standard of care HRT treatment in comparison to healthy female control participants' physical and psychology health over 24 months.

Eligibility Criteria

Inclusion Criteria for POI patients

The participant must:

Be willing to give informed consent/assent
Have a diagnosis of POI based on 2 elevated serum follicle stimulating hormone (FSH) levels obtained >1 month apart.
Be English-speaking

Exclusion Criteria for POI patients

The participant must not:

Have other chronic disease known to affect bone health (e.g., cystic fibrosis, celiac disease, etc.)
Have an identified secondary cause of ovarian insufficiency
Have POI in the setting of Turner syndrome, Fanconi Anemia, galactosemia, or Perrault syndrome (as associated neurological/medical sequelae could confound baseline measures)
Have used medications known to affect bone metabolism over previous 3 months (e.g. anticonvulsants, chronic use of glucocorticoids, Depo-Provera, oral contraceptive pills)
Be currently pregnant (to be confirmed by pregnancy testing)

Inclusion Criteria for Healthy Adolescent Control Participants

The participant must:

Be similar in age and race group to the idiopathic POI group
Control participants age must be within one year of age from the POI participant at the time of enrollment. Age may be within one year older or one year younger
Race of controls participants will be matched based on race of POI patient participants
Have a BMI within 20% of the BMI of the case-matched participant
If postmenarchal, will be regularly menstruating (cycles between 21-35 days)

a. if POI participant is <12.5yrs (mean age of menarche) will match with a pre- menarchal control participant

Be English-speaking

Exclusion Criteria for Healthy Adolescent Control Participants

The participant must not:

Have a chronic disease, known to affect bone metabolism (e.g., cystic fibrosis, celiac disease, sickle cell disease, inflammatory bowel disease etc.)
Be receiving medications known to affect bone metabolism over previous three months (e.g. anticonvulsants, chronic use of glucocorticoids, Depo-Provera, oral contraceptive pills, etc.)
Have a learning disability or a developmental delay
Be currently taking any selective serotonin reuptake inhibitors (SSRIs), antipsychotics or have any documented problems with anxiety or depression.
Be currently pregnant (as confirmed by pregnancy testing)

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03568708). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.