Central Adrenal InsufficiencyNCT04700644
Assessment of CAI in Adults With PWS.
An observational study of Central Adrenal Insufficiency and Prader-Willi Syndrome, run by Medical University of Warsaw at 1 site in Poland.
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… Read more about this study
- Who is it for?
- Ages 18 to 26 years
- Conditions the study is for
- Central Adrenal Insufficiency, Prader-Willi Syndrome
- How many people
- 21 participants
- Where
- 1 sitePoland
- Study dates
- 15 May 2020to 6 Jun 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 26 years
- All sexes
- No healthy volunteers
- Adult
Study population: Consecutive patients with PWS, referred to the Department of Endocrinology Medical University of Warsaw.
Who may be able to join
- PWS
- age >=18 years
Who cannot join
- age <18 years
- GC treatment at last 6 months
Trial locations and contacts
1 site in 1 country.
Poland1 site
Central Clinical Hospital Medical University of Warsaw
Warsaw, 02-097
About this study
Official title: 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.
- Medical subject headings
- Prader-Willi Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: At first visit (before HCT treatment).
Symptoms of CAI before hydrocortisone substitution: fatigue
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: fatigue
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: fatigue
Incidence of the fatigue in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: loss of appetite
Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: loss of appetite
Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: loss of appetite
Incidence of loss of appetite in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: muscle weakness
Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocortisone treatment: muscle weakness
Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocortisone treatment: muscle weakness
Incidence of the muscle weakness in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: myalgia
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: myalgia
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: myalgia
Incidence of the myalgia in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: arthralgia
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: arthralgia
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: arthralgia
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: weight loss
Incidence of the weight loss (>3 kg) in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
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.
Measured: After 6 month HCT treatment
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.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: nausea
Incidence of the nausea in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: nausea
Incidence of the nausea in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: nausea
Incidence of the nausea in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: vomiting
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: vomiting
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: vomiting
Incidence of the vomiting in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
Symptoms of CAI before hydrocortisone substitution: abdominal pain
Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Measured: Before HCT treatment
Symptoms of CAI after 6 month hydrocorisone treatment: abdominal pain
Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Measured: After 6 month HCT treatment
Symptoms of CAI after 12 month hydrocorisone treatment: abdominal pain
Incidence of the abdominal pain in the CAI, intermediate and the AS groups.
Measured: After 12 month HCT treatment
BMI before hydrocortisone substitution in PWS patients with CAI
Measurement of weight and height will be combined to report BMI in kg/m^2.
Measured: Before HCT treatment
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.
Measured: After 6 month HCT treatment
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.
Measured: After 12 month HCT treatment
Body fat percentage before hydrocortisone substitution in PWS patients with CAI
Measurement of the body fat percentage using bioelectrical impedance method.
Measured: Before HCT treatment
Body fat percentage after 6 month hydrocortisone substitution in PWS patients with CAI
Measurement of the body fat percentage using bioelectrical impedance method.
Measured: After 6 month HCT treatment
Body fat percentage after 12 month hydrocortisone substitution in PWS patients with CAI
Measurement of the body fat percentage using bioelectrical impedance method.
Measured: After 12 month HCT treatment
Secondary outcomes (4)
Concentration of morning cortisol
Results of morning cortisol in the entire study group.
Measured: At first visit (before HCT treatment).
Concentration of ACTH
Results of ACTH in the entire study group.
Measured: At first visit (before HCT treatment).
Concentration of dihydroepiandrosterone sulfate
Results of dihydroepiandrosterone sulfate measurements in the entire study group.
Measured: At first visit (before HCT treatment).
Laboratory findings - short Synacthen test
Results of the short Synacthen test in the entire study group.
Measured: At first visit (before HCT treatment).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2021
- Study start15 May 2020
- Primary completion6 Jun 2020
- Study completion6 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Central Adrenal InsufficiencyStudies of the same condition, open and under way.
- More trials run by Medical University of WarsawThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of Warsaw
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Magda Goralska, Medical University of Warsaw
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04700644
- Sponsor's protocol ID
- CAIPWS
- Status last verified
- Jan 2021
- First submitted
- 21 Jun 2020
- Last updated
- 8 Jan 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Central Adrenal Insufficiency in Adults With Prader-Willi Syndrome.
- ClinicalTrials.gov ID
- NCT04700644
- Acronym / sponsor ID
- CAIPWS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Medical University of WarsawUniversity, hospital or other
- Study start
- 15 May 2020 (actual)
- Primary completion
- 6 Jun 2020 (actual)
- Study completion
- 6 Jun 2020 (actual)
- First posted
- 8 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Consecutive patients with PWS, referred to the Department of Endocrinology Medical University of Warsaw.
Key inclusion criteria (2 listed)
- PWS
- age >=18 years
Key exclusion criteria (2 listed)
- age <18 years
- GC treatment at last 6 months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Symptoms of CAI before hydrocortisone substitution: fatigue
Time frame: Before HCT treatment
Incidence of the fatigue in the CAI, intermediate and the AS groups.
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.
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.
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.
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.
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.
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.
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.
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.
Symptoms of CAI before hydrocortisone substitution: myalgia
Time frame: Before HCT treatment
Incidence of the myalgia in the CAI, intermediate and the AS groups.
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.
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.
Symptoms of CAI before hydrocortisone substitution: arthralgia
Time frame: Before HCT treatment
Incidence of the arthralgia in the CAI, intermediate and the AS groups.
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.
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.
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.
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.
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.
Symptoms of CAI before hydrocortisone substitution: nausea
Time frame: Before HCT treatment
Incidence of the nausea in the CAI, intermediate and the AS groups.
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.
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.
Symptoms of CAI before hydrocortisone substitution: vomiting
Time frame: Before HCT treatment
Incidence of the vomiting in the CAI, intermediate and the AS groups.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
4 secondary outcomes
Concentration of morning cortisol
Time frame: At first visit (before HCT treatment).
Concentration of ACTH
Time frame: At first visit (before HCT treatment).
Concentration of dihydroepiandrosterone sulfate
Time frame: At first visit (before HCT treatment).
Laboratory findings - short Synacthen test
Time frame: At first visit (before HCT treatment).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Central Adrenal Insufficiency and Prader-Willi Syndrome, run by Medical University of Warsaw at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The prevalence of CAI in adult PWS patients based on HDSST. (time frame: At first visit (before HCT treatment).). The study lists 34 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2021.