Trial · NCT00001521
Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia
NCT00001521, Phase 3, Completed, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); results first posted 2025-06-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia. Trials, record NCT00001521. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00001521
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia
- NCT number
- NCT00001521
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 66 participants, actual
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Conditions
- Congenital Adrenal Hyperplasia (CAH)
- Interventions
- Fludrocortisone, Hydrocortisone, Letrozole, Flutamide, Testolactone
- First posted
- 1999-11-04
- Last updated
- 2025-06-12
- Results first posted
- 2025-06-12
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Adult Height Relative to General Population | Followed to attainment of adult height, average of 11 years from date of randomization | Adult height expressed in standard deviation score (SDS) units relative to the general population, with attainment of adult height defined as incremental growth \< 1.5 cm over 12 months. Adult height SDS was based on National Health and Nutrition Examination Survey (Centers for Disease Control and Prevention, National Center for Health Statistics) data at 20 years old. |
| Secondary | Adult Height Relative to Mid-parental Height | Followed to attainment of adult height, average of 11 years from date of randomization | Adult height expressed in standard deviation score (SDS) units relative to the mid-parental height for the general population. Adult height is defined as incremental growth \< 1.5 cm over 12 months. Adult height SDS was based on National Health and Nutrition Examination Survey (Centers for Disease Control and Prevention, National Center for Health Statistics) data at 20 years old. Mid-parental height was calculated based on reported parental heights calculated as (father's height (cm) + mother's height (cm))/ 2 ± 6.5 (cm). |
| Secondary | Predicted Adult Height | At date of randomization and at pubertal onset (average of seven years from date of randomization) | Predicted adult height was calculated using the bone age at the baseline visit or the first available bone age, using the Bayley-Pinneau method. Predicted adult height was calculated as the standard deviation score (SDS) units relative to the general population based on the National Health and Nutrition Examination Survey data (Centers for Disease Control and Prevention (CDC), National Center for Health Statistics) at 20 years old. Baseline was defined as time of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). |
| Secondary | Predicted Adult Height Change | From date of randomization to pubertal onset visit (which on average was seven years), pubertal onset to final visit (average was 4 years), and date of randomization to final visit (average of 11 years) | Changes in predicted adult height from baseline to pubertal onset, pubertal onset to final visit, and baseline to final visit. Adult height standard deviation score (SDS) was based on National Health and Nutrition Examination Survey (Centers for Disease Control and Prevention, National Center for Health Statistics) data at 20 years old. Predicted adult height at baseline was calculated using the bone age at the baseline visit or the first available bone age according to the Bayley-Pinneau method. Baseline was defined as date of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was defined as attainment of adult height with incremental growth \< 1.5 cm over 12 months. |
| Secondary | Number of Years Bone Age Remained Unchanged | From date of randomization to pubertal onset visit (which on average was seven years) and pubertal onset to final visit (average was 4 years) | Number of prepubertal and pubertal years bone age remained unchanged. Baseline bone age was calculated using the bone age at the baseline visit or the first available bone age according to the Bayley-Pinneau method. Change in bone age from baseline to pubertal onset and pubertal onset to final visit was measured in years. Baseline was defined as date of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was defined as attainment of adult height with incremental growth \< 1.5 cm over 12 months. |
| Secondary | Change in Body Mass Index (BMI) | From date of randomization to pubertal onset visit (which on average was seven years) and pubertal onset to final visit (average was 4 years) | Changes in body mass index (BMI) were evaluated from baseline to puberty onset and puberty onset to final visit. BMI calculation was based on average of three early morning height measurements by stadiometer and weight measurement by scale for each timepoints. BMI was calculated as the standard deviation score (SDS) units relative to the general population based on the National Health and Nutrition Examination Survey data (Centers for Disease Control and Prevention (CDC), National Center for Health Statistics) at 20 years old. Baseline was defined as date of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was time at attainment of adult height, defined as incremental growth \< 1.5 cm over 12 months. |
| Secondary | Body Mass Index (BMI) | Pubertal onset visit (average of seven years from date of randomization) and at final visit (average of 11 years from date of randomization) | Body mass index (BMI) was calculated based on average of three early morning height measurements by stadiometer and weight measurement by scale. BMI was calculated as the standard deviation score (SDS) units relative to the general population based on the National Health and Nutrition Examination Survey data (Centers for Disease Control and Prevention (CDC), National Center for Health Statistics) at 20 years old. Measures evaluated at pubertal onset and at final visit. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was defined as attainment of adult height with incremental growth \< 1.5 cm over 12 months. |
| Secondary | Average Annual Growth Velocity | From date of randomization to pubertal onset visit (which on average was seven years) and pubertal onset to final visit (average was 4 years) | Average annual growth (height) velocity, measured as the change in height over time relative to the population mean and adjusted for age and sex. Measured during the study period from baseline visit to pubertal onset visit (visits occurring approximately every 6 months) and from pubertal onset to adult height. Baseline was defined as date of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was defined as attainment of adult height with incremental growth \< 1.5 cm over 12 months. |
| Secondary | Dose of Oral Hydrocortisone | At date of randomization, pubertal onset (average of seven years from date of randomization), and at final visit (average of 11 years from date of randomization) | Dose of oral hydrocortisone participant was taking adjusted for body surface area. Dose was recorded at baseline (first visit), pubertal onset, and at adult height (final visit). Baseline was defined as date of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was defined as attainment of adult height with incremental growth \< 1.5 cm over 12 months. |
| Secondary | Average Daily Dose of Oral Hydrocortisone | From date of randomization to pubertal onset visit (which on average was seven years), pubertal onset to final visit (average was 4 years), and date of randomization to final visit (average of 11 years) | Average daily dose of oral hydrocortisone adjusted for body surface area. Dose was measured by developmental periods and for differences between sexes. Doses recorded at every visit, approximately every 6 months. Average dose measured from baseline to pubertal onset, from pubertal onset visit to adult height (final visit), and from baseline to adult height. Baseline was defined as date of randomization. Pubertal onset was defined as when patients entered puberty (Tanner 2 breast in females, testicle size ≥ 4 mL in males) or completed therapy with either aromatase inhibitor and/or GnRHa (age 13 years in girls and 14 years in boys). Final visit was defined as attainment of adult height with incremental growth \< 1.5 cm over 12 months. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Adult Height Relative to General Population | Followed to attainment of adult height, average of 11 years from date of randomization | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Adult Height Relative to Mid-parental Height | Followed to attainment of adult height, average of 11 years from date of randomization | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Predicted Adult Height | At date of randomization and at pubertal onset (average of seven years from date of randomization) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Predicted Adult Height Change | From date of randomization to pubertal onset visit (which on average was seven years), pubertal onset to final visit (average was 4 years), and date of randomization to final visit (average of 11 years) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Number of Years Bone Age Remained Unchanged | From date of randomization to pubertal onset visit (which on average was seven years) and pubertal onset to final visit (average was 4 years) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Years | MEAN |
| SECONDARY | Change in Body Mass Index (BMI) | From date of randomization to pubertal onset visit (which on average was seven years) and pubertal onset to final visit (average was 4 years) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Body Mass Index (BMI) | Pubertal onset visit (average of seven years from date of randomization) and at final visit (average of 11 years from date of randomization) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Average Annual Growth Velocity | From date of randomization to pubertal onset visit (which on average was seven years) and pubertal onset to final visit (average was 4 years) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | Standard Deviation Score (SDS) units | MEAN |
| SECONDARY | Dose of Oral Hydrocortisone | At date of randomization, pubertal onset (average of seven years from date of randomization), and at final visit (average of 11 years from date of randomization) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | mg/m2 /day | MEAN |
| SECONDARY | Average Daily Dose of Oral Hydrocortisone | From date of randomization to pubertal onset visit (which on average was seven years), pubertal onset to final visit (average was 4 years), and date of randomization to final visit (average of 11 years) | Modified intention-to-treat population, includes patients who were reassigned from investigational to standard therapy due to drug intolerance. | mg/m2 /day | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group82
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Adult Height Relative to General Population | Investigational Therapy | -0.34 | Standard Deviation 0.93 | Standard Deviation Score (SDS) units | |
| Adult Height Relative to General Population | Standard Therapy | -0.6 | Standard Deviation 0.89 | Standard Deviation Score (SDS) units | |
| Adult Height Relative to Mid-parental Height | Investigational Therapy | Overall Participants | -0.45 | Standard Deviation 0.85 | Standard Deviation Score (SDS) units |
| Adult Height Relative to Mid-parental Height | Standard Therapy | Overall Participants | -0.83 | Standard Deviation 0.78 | Standard Deviation Score (SDS) units |
| Adult Height Relative to Mid-parental Height | Investigational Therapy | Males | -0.89 | Standard Deviation 0.75 | Standard Deviation Score (SDS) units |
| Adult Height Relative to Mid-parental Height | Standard Therapy | Males | -0.93 | Standard Deviation 0.78 | Standard Deviation Score (SDS) units |
| Adult Height Relative to Mid-parental Height | Investigational Therapy | Females | -0.02 | Standard Deviation 0.72 | Standard Deviation Score (SDS) units |
| Adult Height Relative to Mid-parental Height | Standard Therapy | Females | -0.67 | Standard Deviation 0.79 | Standard Deviation Score (SDS) units |
| Predicted Adult Height | Investigational Therapy | Baseline | -2.0 | Standard Deviation 1.5 | Standard Deviation Score (SDS) units |
| Predicted Adult Height | Standard Therapy | Baseline | -2.1 | Standard Deviation 1.5 | Standard Deviation Score (SDS) units |
1 to 10 of 82Source ClinicalTrials.gov
See all 82 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Investigational Therapy | 31 | |
| Overall Study | STARTED | Standard Therapy | 31 | |
| Overall Study | Moved From Investigational to Standard Therapy | Investigational Therapy | 4 | |
| Overall Study | Moved From Investigational to Standard Therapy | Standard Therapy | 0 | |
| Overall Study | COMPLETED | Investigational Therapy | 21 | |
| Overall Study | COMPLETED | Standard Therapy | 24 | |
| Overall Study | NOT COMPLETED | Investigational Therapy | 10 | |
| Overall Study | NOT COMPLETED | Standard Therapy | 7 |
Source ClinicalTrials.gov
Adverse events180
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypothyroidism | Endocrine disorders | Investigational Therapy | 1 | 31 | |
| other | Hypothyroidism | Endocrine disorders | Standard Therapy | 0 | 35 | |
| other | Thyroiditis | Endocrine disorders | Investigational Therapy | 1 | 31 | |
| other | Thyroiditis | Endocrine disorders | Standard Therapy | 0 | 35 | |
| other | Abdominal pain | Gastrointestinal disorders | Investigational Therapy | 6 | 31 | |
| other | Abdominal pain | Gastrointestinal disorders | Standard Therapy | 2 | 35 | |
| other | Dyspepsia | Gastrointestinal disorders | Investigational Therapy | 3 | 31 | |
| other | Dyspepsia | Gastrointestinal disorders | Standard Therapy | 0 | 35 | |
| other | Nausea | Gastrointestinal disorders | Investigational Therapy | 6 | 31 | |
| other | Nausea | Gastrointestinal disorders | Standard Therapy | 2 | 35 |
1 to 10 of 180Source ClinicalTrials.gov
See all 180 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Investigational Therapy | 0 | 31 |
| Total, all-cause mortality | Standard Therapy | 0 | 35 |
| Total, other adverse events | Investigational Therapy | 31 | 31 |
| Total, other adverse events | Standard Therapy | 31 | 35 |
| Total, serious adverse events | Investigational Therapy | 3 | 31 |
| Total, serious adverse events | Standard Therapy | 10 | 35 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39672600 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Investigational Therapy vs Standard Therapy: Number of Female Participants With Normal Menstrual Cyclicity at Final Visit | Trial comparisons |
| Investigational Therapy vs Standard Therapy: Number of Participants With Onset of Early Central Puberty | Trial comparisons |
| Investigational Therapy vs Standard Therapy: deaths from any cause | Trial comparisons |
| Investigational Therapy vs Standard Therapy: Number of Participants With Insulin Resistance Based on Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) > 2.5 | Trial comparisons |
| Investigational Therapy vs Standard Therapy: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00001521
GET https://conductscience.org/api/trials/v1/NCT00001521/registered?page=2