Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia
Summary
Linked Publications
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Adult Height Following Prepubertal Treatment With Antiandrogen, Aromatase Inhibitor, and Reduced Hydrocortisone in CAH.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Adult Height Relative to General Population |
-0.34; -0.6 | — |
| SECONDARY Adult Height Relative to Mid-parental Height |
-0.45; -0.83; -0.89; -0.93; -0.02; -0.67 | — |
| SECONDARY Predicted Adult Height |
-2.0; -2.1; -0.46; -0.98 | — |
| SECONDARY Predicted Adult Height Change |
1.93; 1.10; -0.10; 0.14; 1.64; 1.20 | — |
| SECONDARY Number of Years Bone Age Remained Unchanged |
3.86; 2.78; 0.39; 0.70 | — |
| SECONDARY Change in Body Mass Index (BMI) |
0.12; 0.36; -0.06; -0.20 | — |
| SECONDARY Body Mass Index (BMI) |
1.07; 1.09; 0.79; 0.89 | — |
| SECONDARY Average Annual Growth Velocity |
-1.01; -0.34; 0.65; -0.29 | — |
| SECONDARY Dose of Oral Hydrocortisone |
14.7; 13.9; 7.6; 15.0; 16.4; 16.8 | — |
| SECONDARY Average Daily Dose of Oral Hydrocortisone |
10.1; 15.0; 9.5; 15.0; 9.1; 13.9 | — |
| SECONDARY Percent of Visits With 17-hydroxyprogesterone in the Optimal Range (<1,200 ng/dL) |
11.8; 35.9; 0; 28.6 | — |
| SECONDARY Percent of Visits With Androstenedione in Normal Range |
0; 45; 20.2; 50.0 | — |
| SECONDARY Average Testosterone |
86.3; 24.5; 336.0; 418.4; 42.3; 34.4 | — |
| SECONDARY Number of Participants With Onset of Early Central Puberty |
12; 9 | — |
| SECONDARY Average Age at Menarche |
14.7; 13.6 | — |
| SECONDARY Number of Female Participants With Normal Menstrual Cyclicity at Final Visit |
4; 7 | — |
| SECONDARY Number of Participants With Insulin Resistance Based on Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) > 2.5 |
8; 12 | — |
| SECONDARY Average (Median) Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) |
2.42; 2.29 | — |
| SECONDARY Number of Male Participants With Testicular Adrenal Rest Tumors (TART) |
4; 3; 7; 7 | — |
| SECONDARY Anterior Posterior Spine Bone Mineral Density (BMD) at Final Visit |
-0.19; -0.25 | — |
| SECONDARY Femoral Neck Bone Mineral Density (BMD) at Final Visit |
-0.07; -0.32 | — |
Eligibility Criteria
- INCLUSION CRITERIA:
Subjects will be boys with bone ages 2 to 13 years and girls with bone ages 2 to 11 years with CAH due to classic 21-hydroxylase deficiency.
Subjects must either not yet have undergone pubertal activation of the hypothalamic-pituitary-gonadal axis, or, if pubertal activation has occurred, must be receiving a GnRH agonist to suppress secondary central precocious puberty.
Children with a bone age of 1 to 2 years may enroll in the protocol for optimization of conventional therapy, but will not be randomized to a study arm until the bone age reaches 2.
EXCLUSION CRITERIA
Children who have concurrent illnesses requiring glucocorticoid treatment (such as severe asthma), or requiring drugs that markedly alter hydrocortisone metabolism (such as anticonvulsants), and children who cannot be brought into reasonable control with conventional treatment (an unusual occurrence).
Data sourced from ClinicalTrials.gov (NCT00001521) and the linked publication. 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. Informational only — not medical advice.