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Completed

NCT Number: NCT04700644

Assessment of CAI in Adults With PWS.

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.

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Key information

Conditions

Central Adrenal Insufficiency Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight Prader-Willi Syndrome

Age range

18 year–26 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Central Clinical Hospital Medical University of Warsaw

Warsaw, 02-097, Poland

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • PWS
  • age >=18 years

Exclusion criteria

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

Treatment and study plan

Primary outcomes

  1. The prevalence of CAI in adult PWS patients based on HDSST.

    Time frame: 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:

    • 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

  2. Symptoms of CAI before hydrocortisone substitution: fatigue

    Time frame: Before HCT treatment

    Incidence of the fatigue in the CAI, intermediate and the AS groups.

  3. Symptoms of CAI after 6 month hydrocorisone treatment: fatigue

    Time frame: After 6 month HCT treatment

    Incidence of the fatigue in the CAI, intermediate and the AS groups.

  4. Symptoms of CAI after 12 month hydrocorisone treatment: fatigue

    Time frame: After 12 month HCT treatment

    Incidence of the fatigue in the CAI, intermediate and the AS groups.

  5. Symptoms of CAI before hydrocortisone substitution: loss of appetite

    Time frame: Before HCT treatment

    Incidence of loss of appetite in the CAI, intermediate and the AS groups.

  6. Symptoms of CAI after 6 month hydrocorisone treatment: loss of appetite

    Time frame: After 6 month HCT treatment

    Incidence of loss of appetite in the CAI, intermediate and the AS groups.

  7. Symptoms of CAI after 12 month hydrocorisone treatment: loss of appetite

    Time frame: After 12 month HCT treatment

    Incidence of loss of appetite in the CAI, intermediate and the AS groups.

  8. Symptoms of CAI before hydrocortisone substitution: muscle weakness

    Time frame: Before HCT treatment

    Incidence of the muscle weakness in the CAI, intermediate and the AS groups.

  9. Symptoms of CAI after 6 month hydrocortisone treatment: muscle weakness

    Time frame: After 6 month HCT treatment

    Incidence of the muscle weakness in the CAI, intermediate and the AS groups.

  10. Symptoms of CAI after 12 month hydrocortisone treatment: muscle weakness

    Time frame: After 12 month HCT treatment

    Incidence of the muscle weakness in the CAI, intermediate and the AS groups.

  11. Symptoms of CAI before hydrocortisone substitution: myalgia

    Time frame: Before HCT treatment

    Incidence of the myalgia in the CAI, intermediate and the AS groups.

  12. Symptoms of CAI after 6 month hydrocorisone treatment: myalgia

    Time frame: After 6 month HCT treatment

    Incidence of the myalgia in the CAI, intermediate and the AS groups.

  13. Symptoms of CAI after 12 month hydrocorisone treatment: myalgia

    Time frame: After 12 month HCT treatment

    Incidence of the myalgia in the CAI, intermediate and the AS groups.

  14. Symptoms of CAI before hydrocortisone substitution: arthralgia

    Time frame: Before HCT treatment

    Incidence of the arthralgia in the CAI, intermediate and the AS groups.

  15. Symptoms of CAI after 6 month hydrocorisone treatment: arthralgia

    Time frame: After 6 month HCT treatment

    Incidence of the arthralgia in the CAI, intermediate and the AS groups.

  16. Symptoms of CAI after 12 month hydrocorisone treatment: arthralgia

    Time frame: After 12 month HCT treatment

    Incidence of the arthralgia in the CAI, intermediate and the AS groups.

  17. Symptoms of CAI before hydrocortisone substitution: weight loss

    Time frame: Before HCT treatment

    Incidence of the weight loss (>3 kg) in the CAI, intermediate and the AS groups.

  18. Symptoms of CAI after 6 month hydrocorisone treatment: weight loss

    Time frame: After 6 month HCT treatment

    Incidence of the weight loss (>3 kg) in the CAI, intermediate and the AS groups.

  19. Symptoms of CAI after 12 month hydrocorisone treatment: weight loss

    Time frame: After 12 month HCT treatment

    Incidence of the weight loss (>3 kg) in the CAI, intermediate and the AS groups.

  20. Symptoms of CAI before hydrocortisone substitution: nausea

    Time frame: Before HCT treatment

    Incidence of the nausea in the CAI, intermediate and the AS groups.

  21. Symptoms of CAI after 6 month hydrocorisone treatment: nausea

    Time frame: After 6 month HCT treatment

    Incidence of the nausea in the CAI, intermediate and the AS groups.

  22. Symptoms of CAI after 12 month hydrocorisone treatment: nausea

    Time frame: After 12 month HCT treatment

    Incidence of the nausea in the CAI, intermediate and the AS groups.

  23. Symptoms of CAI before hydrocortisone substitution: vomiting

    Time frame: Before HCT treatment

    Incidence of the vomiting in the CAI, intermediate and the AS groups.

  24. Symptoms of CAI after 6 month hydrocorisone treatment: vomiting

    Time frame: After 6 month HCT treatment

    Incidence of the vomiting in the CAI, intermediate and the AS groups.

  25. Symptoms of CAI after 12 month hydrocorisone treatment: vomiting

    Time frame: After 12 month HCT treatment

    Incidence of the vomiting in the CAI, intermediate and the AS groups.

  26. Symptoms of CAI before hydrocortisone substitution: abdominal pain

    Time frame: Before HCT treatment

    Incidence of the abdominal pain in the CAI, intermediate and the AS groups.

  27. Symptoms of CAI after 6 month hydrocorisone treatment: abdominal pain

    Time frame: After 6 month HCT treatment

    Incidence of the abdominal pain in the CAI, intermediate and the AS groups.

  28. Symptoms of CAI after 12 month hydrocorisone treatment: abdominal pain

    Time frame: After 12 month HCT treatment

    Incidence of the abdominal pain in the CAI, intermediate and the AS groups.

  29. BMI before hydrocortisone substitution in PWS patients with CAI

    Time frame: Before HCT treatment

    Measurement of weight and height will be combined to report BMI in kg/m^2.

  30. BMI after 6 month hydrocortisone substitution in PWS patients with CAI

    Time frame: After 6 month HCT treatment

    Measurement of weight and height will be combined to report BMI in kg/m^2.

  31. BMI after 12 month hydrocortisone substitution in PWS patients with CAI

    Time frame: After 12 month HCT treatment

    Measurement of weight and height will be combined to report BMI in kg/m^2.

  32. Body fat percentage before hydrocortisone substitution in PWS patients with CAI

    Time frame: Before HCT treatment

    Measurement of the body fat percentage using bioelectrical impedance method.

  33. Body fat percentage after 6 month hydrocortisone substitution in PWS patients with CAI

    Time frame: After 6 month HCT treatment

    Measurement of the body fat percentage using bioelectrical impedance method.

  34. Body fat percentage after 12 month hydrocortisone substitution in PWS patients with CAI

    Time frame: After 12 month HCT treatment

    Measurement of the body fat percentage using bioelectrical impedance method.

Secondary outcomes

  1. Concentration of morning cortisol

    Time frame: At first visit (before HCT treatment).

    Results of morning cortisol in the entire study group.

  2. Concentration of ACTH

    Time frame: At first visit (before HCT treatment).

    Results of ACTH in the entire study group.

  3. Concentration of dihydroepiandrosterone sulfate

    Time frame: At first visit (before HCT treatment).

    Results of dihydroepiandrosterone sulfate measurements in the entire study group.

  4. Laboratory findings - short Synacthen test

    Time frame: At first visit (before HCT treatment).

    Results of the short Synacthen test in the entire study group.

Sponsors and collaborators

Lead sponsor

Medical University of Warsaw

Other

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2020
Study completion
2020
First posted
Jan 8, 2021
Registry last updated
Jan 8, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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