Congenital Adrenal Hyperplasia Clinical Trial
Official title:
Final Height in Patients With Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is the most common inherited disorder in the adrenal gland in children. Growth is usually affected in CAH patients either due to the disease itself or treatment consequences.
CAH comprises a group of autosomal recessive disorders caused by a deficiency of one of five
enzymes needed for the synthesis of cortisol leading to defect in cortisol synthesis with or
without aldosterone deficiency and an increase in the production of adrenocorticotropic
hormone through negative feedback.
The most common form is 21-hydroxylase deficiency (21OHD), which forms more than 90 % of the
cases.
In classic CAH, 75% of the patients have the salt wasting (SW) and 25% have the non
salt-wasting phenotype (NSW).There are no clinical signs at birth in male infants and in
female patients, CAH is suspected shortly after birth if there is genital ambiguity, ranging
from slight clitromegaly to complete masculinization with acceleration of growth and pubertal
development.
The non-classic (late onset) form of CAH is a less severe form of 21OHD, and is diagnosed
later in life.
Final height in early and late onset patients has been reported as diminished (Hauffa et al,
1997).This could be attributed to androgen excess or treatment with steroids. Androgen excess
can occur at any age leading to accelerated growth, early epiphyseal closure and compromised
final adult height.
Despite that all forms of CAH differ in their degree of enzymatic deficiency, they all
represent a therapeutic challenge to pediatric endocrinologists attempting to optimize
growth.
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT03687242 -
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
|
Phase 2 | |
| Active, not recruiting |
NCT04544410 -
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
|
Phase 2 | |
| Not yet recruiting |
NCT04087148 -
Linear Growth of Children With Congenital Adrenal Hyperplasia
|
||
| Completed |
NCT03162159 -
Adult Height Prediction in Congenital Adrenal Hyperplasia
|
||
| Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
| Enrolling by invitation |
NCT05162950 -
Effects and Importance of Epinephrine/Adrenalin Deficiency in CAH
|
||
| Withdrawn |
NCT03532022 -
Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy
|
Phase 3 | |
| Recruiting |
NCT02795871 -
Prenatal Dex Study
|
N/A | |
| Completed |
NCT02934399 -
Dynamic Hormone Diagnostics in Endocrine Disease
|
||
| Recruiting |
NCT04903587 -
Gonadal Changes In Congenital Adrenal Hyperplasia Patients
|
||
| Not yet recruiting |
NCT04536662 -
Comparisons of Different Forms of Glucocorticoid on the Recovery of Reproductive Function in Patients With 21α-hydroxylase Deficiency
|
Phase 4 | |
| Recruiting |
NCT04463316 -
GROWing Up With Rare GENEtic Syndromes
|
||
| Active, not recruiting |
NCT04490915 -
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
|
Phase 3 | |
| Completed |
NCT01807364 -
Cardiovascular Risk Profile in Patients With Congenital Adrenal Hyperplasia
|
N/A | |
| Completed |
NCT02804178 -
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
|
Phase 2 | |
| Completed |
NCT03019614 -
An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisone
|
Phase 1 | |
| Recruiting |
NCT03897504 -
Surgical Evaluation of Using the Prepuce in Feminizing Genitoplasty
|
N/A | |
| Recruiting |
NCT05663320 -
A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia
|
N/A | |
| Active, not recruiting |
NCT04806451 -
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
|
Phase 3 | |
| Completed |
NCT01875640 -
Decision Support for Parents Receiving Information About Child's Rare Disease
|