Assessment of CAI in Adults With PWS.

January 5, 2021 updated by: Magda Goralska, Medical University of Warsaw

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Poland
      • Warsaw, Poland, 02-097
        • Central Clinical Hospital Medical University of Warsaw

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 26 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Consecutive patients with PWS, referred to the Department of Endocrinology Medical University of Warsaw.

Description

Inclusion Criteria:

  • PWS
  • age >=18 years

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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:

  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
At first visit (before HCT treatment).
Symptoms of CAI before hydrocortisone substitution: fatigue
Time Frame: Before HCT treatment
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
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.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
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.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: myalgia
Time Frame: Before HCT treatment
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: arthralgia
Time Frame: Before HCT treatment
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
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.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: nausea
Time Frame: Before HCT treatment
Incidence of the nausea in the CAI, intermediate and the AS groups.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: vomiting
Time Frame: Before HCT treatment
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
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.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
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.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment
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.
Before HCT treatment
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.
After 6 month HCT treatment
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.
After 12 month HCT treatment

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Concentration of morning cortisol
Time Frame: At first visit (before HCT treatment).
Results of morning cortisol in the entire study group.
At first visit (before HCT treatment).
Concentration of ACTH
Time Frame: At first visit (before HCT treatment).
Results of ACTH in the entire study group.
At first visit (before HCT treatment).
Concentration of dihydroepiandrosterone sulfate
Time Frame: At first visit (before HCT treatment).
Results of dihydroepiandrosterone sulfate measurements in the entire study group.
At first visit (before HCT treatment).
Laboratory findings - short Synacthen test
Time Frame: At first visit (before HCT treatment).
Results of the short Synacthen test in the entire study group.
At first visit (before HCT treatment).

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Medical University of Warsaw

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 15, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 6, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 6, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 21, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 5, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 8, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 8, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 5, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CAIPWS

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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