Trial Outcomes & Findings for Primary Ovarian Insufficiency: Phenotype and Optimal Treatment (NCT NCT03568708)
NCT ID: NCT03568708
Last Updated: 2024-06-25
Results Overview
Change in height adjusted areal BMD Z-score of the lumbar spine from baseline to 24 months within groups. BMI Z-score, calcium intake, vitamin D intake and physical activity were included in the analysis. As DXA BMD Z-scores already include race, age, and sex, these variables were not included in the analysis. Z-scores ranging between -2.0 and 2.0 are considered normal. A Z-score \<-2.0 is considered low. This analysis considers change in Z-score, therefore a high value reflects a greater increase in BMD Z-score.
COMPLETED
PHASE3
19 participants
Change in bone mineral density and body composition from baseline to 24 months
2024-06-25
Participant Flow
Participant milestones
| Measure |
Control Participants
The control group will reflect a comparison group similar to the POI patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
Primary Ovarian Insufficiency (POI) Participants
This group will be participants who have been recently diagnosed with Primary ovarian Insufficiency (POI). In an open-label fashion, participants with POI will receive Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
10
|
|
Overall Study
COMPLETED
|
8
|
8
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
| Measure |
Control Participants
The control group will reflect a comparison group similar to the POI patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
Primary Ovarian Insufficiency (POI) Participants
This group will be participants who have been recently diagnosed with Primary ovarian Insufficiency (POI). In an open-label fashion, participants with POI will receive Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
Baseline Characteristics
Primary Ovarian Insufficiency: Phenotype and Optimal Treatment
Baseline characteristics by cohort
| Measure |
Control Participants
n=9 Participants
The control group will reflect a comparison group similar to the POI patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
Primary Ovarian Insufficiency (POI) Participants
n=10 Participants
This group will be participants who have been recently diagnosed with POI. In an open-label fashion, participants with POI will receive Transdermal Estrogen(beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Bone Mineral Density Whole Body less head
|
0.00 Z-score
STANDARD_DEVIATION 0.96 • n=99 Participants
|
-1.93 Z-score
STANDARD_DEVIATION 0.84 • n=107 Participants
|
-1.02 Z-score
STANDARD_DEVIATION 1.32 • n=206 Participants
|
|
Age, Categorical
<=18 years
|
8 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
16.9 years
STANDARD_DEVIATION 0.94 • n=99 Participants
|
16.8 years
STANDARD_DEVIATION 1.25 • n=107 Participants
|
16.8 years
STANDARD_DEVIATION 1.8 • n=206 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
9 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Body Mass Index (BMI)
|
25.90 kg/m^2
STANDARD_DEVIATION 4.78 • n=99 Participants
|
26.37 kg/m^2
STANDARD_DEVIATION 6.48 • n=107 Participants
|
26.15 kg/m^2
STANDARD_DEVIATION 5.79 • n=206 Participants
|
|
Bone Mineral Density Lumbar Spine
|
0.80 Z-score
STANDARD_DEVIATION 0.76 • n=99 Participants
|
-1.80 Z-score
STANDARD_DEVIATION 1.05 • n=107 Participants
|
-0.57 Z-score
STANDARD_DEVIATION 1.61 • n=206 Participants
|
|
Bone Mineral Density Total Hip
|
0.83 Z-score
STANDARD_DEVIATION 0.76 • n=99 Participants
|
-0.94 Z-score
STANDARD_DEVIATION 0.68 • n=107 Participants
|
-0.10 Z-score
STANDARD_DEVIATION 1.15 • n=206 Participants
|
|
Bone Mineral Density Femoral Neck
|
0.91 Z-score
STANDARD_DEVIATION 0.68 • n=99 Participants
|
-1.12 Z-score
STANDARD_DEVIATION 0.76 • n=107 Participants
|
-0.16 Z-score
STANDARD_DEVIATION 1.26 • n=206 Participants
|
|
Screen for Child Anxiety Related Disorders (SCARED)
|
17.2 units on a scale
STANDARD_DEVIATION 12.2 • n=99 Participants
|
27.0 units on a scale
STANDARD_DEVIATION 14.9 • n=107 Participants
|
22.4 units on a scale
STANDARD_DEVIATION 14.2 • n=206 Participants
|
|
Children's Depression Inventory-II (CDI-2)
|
51.0 units on a scale
STANDARD_DEVIATION 11.4 • n=99 Participants
|
55.3 units on a scale
STANDARD_DEVIATION 10.9 • n=107 Participants
|
53.3 units on a scale
STANDARD_DEVIATION 11.1 • n=206 Participants
|
|
Child and Adolescent Memory Profile (CHaMP) Total Memory Index
|
89.5 units on a scale
STANDARD_DEVIATION 9.1 • n=99 Participants
|
93.7 units on a scale
STANDARD_DEVIATION 11.7 • n=107 Participants
|
91.8 units on a scale
STANDARD_DEVIATION 10.6 • n=206 Participants
|
|
Child Health Questionnaire (CHQ-87) Total Score
|
76.1 units on a scale
STANDARD_DEVIATION 11.6 • n=99 Participants
|
70.1 units on a scale
STANDARD_DEVIATION 12.3 • n=107 Participants
|
72.9 units on a scale
STANDARD_DEVIATION 12.0 • n=206 Participants
|
PRIMARY outcome
Timeframe: Change in bone mineral density and body composition from baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
Change in height adjusted areal BMD Z-score of the lumbar spine from baseline to 24 months within groups. BMI Z-score, calcium intake, vitamin D intake and physical activity were included in the analysis. As DXA BMD Z-scores already include race, age, and sex, these variables were not included in the analysis. Z-scores ranging between -2.0 and 2.0 are considered normal. A Z-score \<-2.0 is considered low. This analysis considers change in Z-score, therefore a high value reflects a greater increase in BMD Z-score.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Dual Energy X-ray Absorptiometry (DXA) Measure of Bone Mineral Density (BMD) of the Lumbar Spine
|
0.228 Z-score
Standard Deviation 0.289
|
0.673 Z-score
Standard Deviation 0.289
|
SECONDARY outcome
Timeframe: baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
To assess changes in bone mineral density DXA height adjusted BMD Z-scores of the whole body less head, total hip and femoral neck were measured. BMI, calcium intake, vitamin D intake and physical activity were included in the analysis. As DXA BMD Z-scores already include race, age, and sex, these variables were not included in the analysis. Z-scores ranging between -2.0 and 2.0 are considered normal. A Z-score \<-2.0 is considered low. This analysis considers change in Z-score, therefore a high value reflects a greater increase in BMD Z-score.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
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
Whole Body Less Head
|
0.433 Z-score
Standard Deviation 0.199
|
0.815 Z-score
Standard Deviation 0.198
|
|
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
Total Hip
|
0.231 Z-score
Standard Deviation 0.170
|
0.370 Z-score
Standard Deviation 0.170
|
|
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
Femoral Neck
|
0.337 Z-score
Standard Deviation 0.195
|
0.556 Z-score
Standard Deviation 0.196
|
SECONDARY outcome
Timeframe: Change from baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
To assess the appendicular (peripheral) skeleton, pQCT (Stratec XCT 2000, Orthometrix, Inc., White Plains, NY) bone measures were obtained of the non-dominant radius at the 3% and 66% sites. Measurements were acquired with a voxel size of 0.4 mm, slice thickness of 2.3 mm, and scan speed of 25 mm/sec, and analyzed with manufacturer software version 6.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Volumetric Bone Mineral Density (vBMD) at the Distal Radius as Measured by Peripheral Quantitative Computed Tomography (pQCT)
3% trabecular volumetric BMD
|
-1.245 mg/mm^3
Standard Deviation 14.484
|
-5.415 mg/mm^3
Standard Deviation 14.638
|
|
Change in Volumetric Bone Mineral Density (vBMD) at the Distal Radius as Measured by Peripheral Quantitative Computed Tomography (pQCT)
66% cortical volumetric BMD
|
-0.073 mg/mm^3
Standard Deviation 17.728
|
13.945 mg/mm^3
Standard Deviation 18.178
|
SECONDARY outcome
Timeframe: Baseline and 24 monthsThe mean BMI in kg/m\^2 is presented for each study group at baseline and at the 24 months follow up visit to show that there was no significant difference between groups nor a significant change in BMI over the duration of the study.
Outcome measures
| Measure |
Control Participants
n=8 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=8 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Anthropometrics
BMI at Baseline
|
25.9 kg/m^2
Standard Deviation 4.78
|
27.01 kg/m^2
Standard Deviation 6.93
|
|
Anthropometrics
BMI at 24 months
|
27.64 kg/m^2
Standard Deviation 6.04
|
27.86 kg/m^2
Standard Deviation 9.62
|
SECONDARY outcome
Timeframe: Change in lean mass from baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
Lean mass was obtained from the whole body DXA scan. Change in baseline to 24 months was assessed.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Lean Mass as Measured by DXA Body Composition
|
-0.999 kg
Standard Deviation 1.754
|
1.577 kg
Standard Deviation 1.753
|
SECONDARY outcome
Timeframe: Change from SCARED score baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
A 41 item self-report tool to assess for anxiety where each question receives a score of either 0, 1 or 2. Range of scores is 0 to 82. A total score of ≥ 25 may indicate the presence of an Anxiety Disorder. A higher score indicates there are more endorsed symptoms of anxiety.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Symptoms of Anxiety as Measured by Screen for Child Anxiety Related Disorders (SCARED)
|
8.339 score on a scale
Standard Deviation 7.508
|
-1.261 score on a scale
Standard Deviation 7.454
|
SECONDARY outcome
Timeframe: Change from CDI-II score from baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
A brief self-report test that helps assess cognitive, affective and behavioral signs of depression in children and adolescents 7 to 17 years old. Scales include Emotional and Functional Problems, along with subscales of Negative mood/Physical symptoms, Negative Self-Esteem, Interpersonal Problems, and Ineffectiveness. The total score is converted into a T-score (mean=50, standard deviation=10) where a result \>64 is considered elevated. A higher score indicates there are more endorsed symptoms of depression.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Symptoms of Depression as Measured by Child Depression Inventory-II (CDI-II)
|
1.246 T-score
Standard Deviation 5.949
|
-1.187 T-score
Standard Deviation 5.793
|
SECONDARY outcome
Timeframe: Change from score from baseline to 24 monthsThe ChAMP is a norm-referenced test of memory and learning that was designed for use with children, adolescents, and young adults ranging from 5 through 21 years. The ChAMP includes 4 Subtests of visual and verbal memory to generate a total memory index score as a measure of overall memory. The total memory index score ranges from 50-150 with a mean=100 and standard deviation=15. Higher scores indicating better memory. The data presented here is the change in the total memory index score from baseline visit to the 24 month follow up time.
Outcome measures
| Measure |
Control Participants
n=8 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=8 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Memory as Assessed by the Children and Adolescent Memory Profile (CHAMP) Total Memory Index Score
|
17.203 score on a scale
Standard Deviation 5.454
|
24.619 score on a scale
Standard Deviation 5.379
|
SECONDARY outcome
Timeframe: Change from baseline to 24 monthsPopulation: One POI participant had prior oral contraceptive exposure, no matched control, and did not follow up for subsequent visits. For these reasons it was recommended that her data not be included in the analyses.
The CHQ-87 is an 87-item self-report survey is designed to measure the physical and psychosocial health of adolescents. The total score ranges from 0-100. Higher scores indicate better quality of life. This instrument is reliable and valid for evaluating aspects of health pertinent across age, gender, health condition, and socioeconomic status in adolescents.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=9 Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Change in Quality of Life as Assessed by the Child Health Questionnaire-Child Self-Report Form (CHQ-CF87)
|
2.710 score on a scale
Standard Deviation 6.028
|
5.686 score on a scale
Standard Deviation 5.988
|
SECONDARY outcome
Timeframe: Patch Calendars were collected at 6 months, 12 months, 18 months and 24 months. Data presented is through study completion.Population: As only participants with POI were prescribed TDE2 patches, we only report data on this cohort.
Participants with primary ovarian insufficiency (POI) were prescribed weekly transdermal estrogen (TDE2) patches and asked to log on a patch calendar when they changed the patch. Patch calendars were reviewed for compliance and weeks where at least one patch was applied were considered to be in compliance. Weeks in compliance generated the numerator whereas total weeks of participation in the study constituted the numerator.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Compliance With Transdermal Estrogen Patch
|
69 percentage of weeks with TDE2 use
Standard Deviation 31.4
|
—
|
SECONDARY outcome
Timeframe: Baseline, 12 months, 24 monthsPopulation: The study was originally designed as a 12 month longitudinal trial. Subjects were given the option to continue for 24 months. One case participant declined the extension. As only participants with POI were prescribed transdermal estradiol patches, we only report data on this cohort.
Mean serum estradiol levels as measured in participants with POI.
Outcome measures
| Measure |
Control Participants
n=9 Participants
The control group reflects a comparison group similar to the Primary Ovarian Insufficiency (POI) patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
This group includes participants diagnosed with POI who completed the transdermal estrogen titration. In an open-label fashion, participants with POI received Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
|
|---|---|---|
|
Study Medications - Serum Estradiol
Estradiol 12 month
|
55.41 pg/mL
Standard Deviation 34.75
|
—
|
|
Study Medications - Serum Estradiol
Estradiol 24 month
|
98.63 pg/mL
Standard Deviation 93.96
|
—
|
|
Study Medications - Serum Estradiol
Estradiol Baseline
|
7.49 pg/mL
Standard Deviation 2.93
|
—
|
Adverse Events
Control Participants
POI Participants
Serious adverse events
| Measure |
Control Participants
n=9 participants at risk
The control group will reflect a comparison group similar to the POI patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=10 participants at risk
This group will be participants who have been recently diagnosed with POI. In an open-label fashion, participants with POI will receive Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
For the reporting of adverse events, we included all participants who were enrolled in the study regardless of whether data on our primary or secondary outcomes were analyzed.
|
|---|---|---|
|
Blood and lymphatic system disorders
DVT
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Endocrine disorders
Diabetic ketoacidosis (DKA) hospitalization
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Psychiatric disorders
Suicidality
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
Other adverse events
| Measure |
Control Participants
n=9 participants at risk
The control group will reflect a comparison group similar to the POI patient group. As bone density, body composition, and cognitive domains continue to mature throughout the teenage years, this comparison group will provide an important metric of normal growth and development.
|
POI Participants
n=10 participants at risk
This group will be participants who have been recently diagnosed with POI. In an open-label fashion, participants with POI will receive Transdermal Estrogen (beginning at a dose of 25 μg/patch applied weekly), with the dose increased at 3, 6 12, and 18 months (to 37.5, 50, 75, and 100 µg/patch).
For the reporting of adverse events, we included all participants who were enrolled in the study regardless of whether data on our primary or secondary outcomes were analyzed.
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Pain/irritation at patch site
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
30.0%
3/10 • Number of events 5 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Injury, poisoning and procedural complications
Foot injury
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
30.0%
3/10 • Number of events 4 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Infections and infestations
Bronchitis
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Reproductive system and breast disorders
Breast Lump
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
General disorders
Vasovagal Syncope
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Surgical and medical procedures
Dental extraction
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Ear and labyrinth disorders
Otoralgia
|
22.2%
2/9 • Number of events 2 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Nervous system disorders
Motor Tics
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Injury, poisoning and procedural complications
Folliculitis
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Endocrine disorders
Diabetic ketoacidosis (DKA)
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Endocrine disorders
Insulin pump site malfunction
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
General disorders
Weight loss
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Surgical and medical procedures
Tympanoplasty
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Infections and infestations
Viral Illness
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Injury, poisoning and procedural complications
Metatarsal fracture
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Social circumstances
Mood Changes
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Injury, poisoning and procedural complications
Headache
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
General disorders
Motor Vehicle Accident
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Injury, poisoning and procedural complications
Abdominal Pain
|
11.1%
1/9 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
0.00%
0/10 • Adverse events were collected from enrollment to completion of the 24 month visit
|
|
Reproductive system and breast disorders
Excessive menstrual bleeding
|
0.00%
0/9 • Adverse events were collected from enrollment to completion of the 24 month visit
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from enrollment to completion of the 24 month visit
|
Additional Information
Dr. Halley Wasserman
CIncinnati Children's Hospital Medical Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place