Assessment of CAI in Adults With PWS.

• Sponsor: Medical University of Warsaw (Other)
• Overall Status: Completed
• CT.gov ID: NCT04700644
• Collaborator: (none)
• Enrollment: 21
• Location: 1
• Actual Duration (Days): 22
• Patients Per Site Per Month: 29.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Central Adrenal Insufficiency; Prader-Willi Syndrome

Study Design

• Study Type: Observational
• Actual Enrollment: 21 participants
• Observational Model: Cohort
• Time Perspective: Retrospective
• Official Title: Assessment of Central Adrenal Insufficiency in Adults With Prader-Willi Syndrome.
• Actual Study Start Date: May 15, 2020
• Actual Primary Completion Date: Jun 6, 2020
• Actual Study Completion Date: Jun 6, 2020

Outcome Measures

Primary Outcome Measures

1. 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: 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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [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 [After 12 month HCT treatment]

    Measurement of the body fat percentage using bioelectrical impedance method.

Secondary Outcome Measures

1. Concentration of morning cortisol [At first visit (before HCT treatment).]

   Results of morning cortisol in the entire study group.

2. Concentration of ACTH [At first visit (before HCT treatment).]

   Results of ACTH in the entire study group.

3. Concentration of dihydroepiandrosterone sulfate [At first visit (before HCT treatment).]

   Results of dihydroepiandrosterone sulfate measurements in the entire study group.

4. Laboratory findings - short Synacthen test [At first visit (before HCT treatment).]

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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 26 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• PWS

• age >=18 years

Exclusion Criteria:

• age <18 years

• GC treatment at last 6 months

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Central Clinical Hospital Medical University of Warsaw	Warsaw		Poland	02-097

Sponsors and Collaborators

• Medical University of Warsaw

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Magda Goralska, Principal Investigator, Medical University of Warsaw
• ClinicalTrials.gov Identifier: NCT04700644
• Other Study ID Numbers: CAIPWS
• First Posted: Jan 8, 2021
• Last Update Posted: Jan 8, 2021
• Last Verified: Jan 1, 2021

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Prader-Willi Syndrome; Adrenal Insufficiency; Syndrome

Study Results

No Results Posted as of Jan 8, 2021