Clinical trial NCT04700644: Assessment of CAI in Adults With PWS.
Official title: Assessment of Central Adrenal Insufficiency in Adults With Prader-Willi Syndrome.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Central Adrenal Insufficiency
Prader-Willi Syndrome
Lead sponsor: Medical University of Warsaw
Sponsor class: OTHER
First posted: 2021-01-08
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 26
Standard ages: ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Consecutive patients with PWS, referred to the Department of Endocrinology Medical University of Warsaw.

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.


Eligibility criteria: Inclusion Criteria:

* PWS
* age \>=18 years

Exclusion Criteria:

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

Primary outcomes:
measure: The prevalence of CAI in adult PWS patients based on HDSST.
timeFrame: At first visit (before HCT treatment).
measure: Symptoms of CAI before hydrocortisone substitution: fatigue
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: fatigue
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: fatigue
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: loss of appetite
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: loss of appetite
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: loss of appetite
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: muscle weakness
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocortisone treatment: muscle weakness
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocortisone treatment: muscle weakness
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: myalgia
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: myalgia
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: myalgia
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: arthralgia
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: arthralgia
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: arthralgia
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: weight loss
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: weight loss
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: weight loss
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: nausea
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: nausea
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: nausea
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: vomiting
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: vomiting
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: vomiting
timeFrame: After 12 month HCT treatment
measure: Symptoms of CAI before hydrocortisone substitution: abdominal pain
timeFrame: Before HCT treatment
measure: Symptoms of CAI after 6 month hydrocorisone treatment: abdominal pain
timeFrame: After 6 month HCT treatment
measure: Symptoms of CAI after 12 month hydrocorisone treatment: abdominal pain
timeFrame: After 12 month HCT treatment
measure: BMI before hydrocortisone substitution in PWS patients with CAI
timeFrame: Before HCT treatment
measure: BMI after 6 month hydrocortisone substitution in PWS patients with CAI
timeFrame: After 6 month HCT treatment
measure: BMI after 12 month hydrocortisone substitution in PWS patients with CAI
timeFrame: After 12 month HCT treatment
measure: Body fat percentage before hydrocortisone substitution in PWS patients with CAI
timeFrame: Before HCT treatment
measure: Body fat percentage after 6 month hydrocortisone substitution in PWS patients with CAI
timeFrame: After 6 month HCT treatment
measure: Body fat percentage after 12 month hydrocortisone substitution in PWS patients with CAI
timeFrame: After 12 month HCT treatment
Secondary outcomes:
measure: Concentration of morning cortisol
timeFrame: At first visit (before HCT treatment).
measure: Concentration of ACTH
timeFrame: At first visit (before HCT treatment).
measure: Concentration of dihydroepiandrosterone sulfate
timeFrame: At first visit (before HCT treatment).
measure: Laboratory findings - short Synacthen test
timeFrame: At first visit (before HCT treatment).
Other outcomes:
Locations:
city: Warsaw
country: Poland
facility: Central Clinical Hospital Medical University of Warsaw
Study officials:
References: