Assessment of CAI in Adults With PWS.

Assessment of Central Adrenal Insufficiency in Adults With Prader-Willi Syndrome.

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04700644

Enrollment

Registered

2021-01-08

Start date

2020-05-15

Completion date

2020-06-06

Last updated

2021-01-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Central Adrenal Insufficiency, Prader-Willi Syndrome

Brief summary

Introduction: The prevalence and clinical significance of central adrenal insufficiency (CAI) in adult patients with Prader Willi Syndrome (PWS) remains unclear. Aim: To assess the prevalence of CAI in adults with PWS and to analyse the effects of replacement therapy with hydrocortisone (HCT) in patients with suspected CAI. Material and Methods: Twenty one adult patients with PWS were evaluated. Based on peak cortisol at the 30 minute of the high dose short Synacthen test (HDSST), patients were divided into three groups: CAI (central adrenal insufficiency) - peak cortisol \<500nmol/L, intermediate (partial AI) - peak cortisol ≥500 nmol/L and \<600 nmol/L and AS (adrenal sufficiency) - peak cortisol ≥ 600 nmol/L. In patients with diagnosed CAI HCT replacement treatment was initiated. Body weight, body fat percentage, signs, and symptoms of CAI were evaluated after 6 and 12 months of treatment.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 26 Years

Healthy volunteers

Inclusion criteria

* PWS * age \>=18 years

Exclusion criteria

* age \<18 years * GC treatment at last 6 months

Design outcomes

Primary

Measure	Time frame	Description
Body fat percentage after 12 month hydrocortisone substitution in PWS patients with CAI	After 12 month HCT treatment	Measurement of the body fat percentage using bioelectrical impedance method.
BMI after 6 month hydrocortisone substitution in PWS patients with CAI	After 6 month HCT treatment	Measurement of weight and height will be combined to report BMI in kg/m\^2.
BMI after 12 month hydrocortisone substitution in PWS patients with CAI	After 12 month HCT treatment	Measurement of weight and height will be combined to report BMI in kg/m\^2.
Body fat percentage before hydrocortisone substitution in PWS patients with CAI	Before HCT treatment	Measurement of the body fat percentage using bioelectrical impedance method.
Body fat percentage after 6 month hydrocortisone substitution in PWS patients with CAI	After 6 month HCT treatment	Measurement of the body fat percentage using bioelectrical impedance method.
The prevalence of CAI in adult PWS patients based on HDSST.	At first visit (before HCT treatment).	The diagnosis of CAI was based on the high-dose short Synacthen test (HDSST). The following cut-off points were used: 1. CAI group (suspected CAI): • cortisol \< 500 nmol/L at the 30th minute 2. AS group (adrenal sufficiency - CAI excluded): • cortisol ≥ 600 nmol/L at the 30th minute 3. Intermediate group • cortisol ≥500 nmol/L and \<600 nmol/L at the 30th minute
Symptoms of CAI before hydrocortisone substitution: fatigue	Before HCT treatment	Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: fatigue	After 6 month HCT treatment	Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: fatigue	After 12 month HCT treatment	Incidence of the fatigue in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: loss of appetite	Before HCT treatment	Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: loss of appetite	After 6 month HCT treatment	Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: loss of appetite	After 12 month HCT treatment	Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: muscle weakness	Before HCT treatment	Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocortisone treatment: muscle weakness	After 6 month HCT treatment	Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocortisone treatment: muscle weakness	After 12 month HCT treatment	Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: myalgia	Before HCT treatment	Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: myalgia	After 6 month HCT treatment	Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: myalgia	After 12 month HCT treatment	Incidence of the myalgia in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: arthralgia	Before HCT treatment	Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: arthralgia	After 6 month HCT treatment	Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: arthralgia	After 12 month HCT treatment	Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: weight loss	Before HCT treatment	Incidence of the weight loss (\>3 kg) in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: weight loss	After 6 month HCT treatment	Incidence of the weight loss (\>3 kg) in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: weight loss	After 12 month HCT treatment	Incidence of the weight loss (\>3 kg) in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: nausea	Before HCT treatment	Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: nausea	After 6 month HCT treatment	Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: nausea	After 12 month HCT treatment	Incidence of the nausea in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: vomiting	Before HCT treatment	Incidence of the vomiting in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: vomiting	After 6 month HCT treatment	Incidence of the vomiting in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: vomiting	After 12 month HCT treatment	Incidence of the vomiting in the CAI, intermediate and the AS groups.
Symptoms of CAI before hydrocortisone substitution: abdominal pain	Before HCT treatment	Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Symptoms of CAI after 6 month hydrocorisone treatment: abdominal pain	After 6 month HCT treatment	Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Symptoms of CAI after 12 month hydrocorisone treatment: abdominal pain	After 12 month HCT treatment	Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
BMI before hydrocortisone substitution in PWS patients with CAI	Before HCT treatment	Measurement of weight and height will be combined to report BMI in kg/m\^2.

Secondary

Measure	Time frame	Description
Concentration of ACTH	At first visit (before HCT treatment).	Results of ACTH in the entire study group.
Concentration of dihydroepiandrosterone sulfate	At first visit (before HCT treatment).	Results of dihydroepiandrosterone sulfate measurements in the entire study group.
Laboratory findings - short Synacthen test	At first visit (before HCT treatment).	Results of the short Synacthen test in the entire study group.
Concentration of morning cortisol	At first visit (before HCT treatment).	Results of morning cortisol in the entire study group.

Countries

Poland

Outcome results