Assessment of CAI in Adults With PWS.

Prader-Willi Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04700644
• Other study ID #: CAIPWS
• Status: Completed
• Start date: May 15, 2020
• Est. completion date: June 6, 2020

Study information

• Verified date: January 2021
• Source: Medical University of Warsaw
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: June 6, 2020
• Est. primary completion date: June 6, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 26 Years

Eligibility

Inclusion Criteria:

• PWS
• age >=18 years

Exclusion Criteria:

• age <18 years
• GC treatment at last 6 months

Related Conditions & MeSH terms

• Adrenal Insufficiency
• Central Adrenal Insufficiency
• Prader-Willi Syndrome
• Syndrome

Locations

Country	Name	City	State
Poland	Central Clinical Hospital Medical University of Warsaw	Warsaw

Sponsors (1)

Lead Sponsor	Collaborator
Medical University of Warsaw

Country where clinical trial is conducted

Poland, 

Outcome

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