Cushing's SyndromePhase 3PROMPTNCT02297945
Effects of Metyrapone in Patients With Endogenous Cushing's Syndrome
A Phase 3 study testing metyrapone in people with Cushing's Syndrome, run by HRA Pharma at 36 sites in 8 countries.
Trial summary
The purpose of this prospective, international phase III/IV study is to assess the efficacy and safety of metyrapone in patients with endogenous Cushing's syndrome during up to 36 weeks of treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cushing's Syndrome
- What is being tested?
- metyrapone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 36 sitesin 8 countries
- Study dates
- Apr 2015to 29 Apr 2020
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Patients with endogenous Cushing's syndrome:
- Cushing disease patients with persistent or recurrent disease (after pituitary surgery) or who are newly diagnosed but are unsuitable for early surgery or wish to defer surgery;
- Patients with ectopic ACTH syndrome (either occult, after surgery failure, or inoperable or metastatic);
- Patients with Cushing's syndrome from adrenal causes
Who cannot join
- Pseudo Cushing's syndrome
- Cyclic Cushing's syndrome defined by at least one normal UFC value among at least three 24-hour urinary sampling measurements over the previous 2 months
- Advanced adrenocortical carcinoma or ectopic ACTH secretion (EAS) secondary to a small cell lung carcinoma
- Life expectancy less than 3 months
- Pituitary or adrenal surgery or pituitary irradiation or surgery of the ACTH-secreting ectopic tumor or bilateral adrenalectomy planned before the week 12 visit
- Pituitary irradiation within the previous 5 years (for Cushing's disease patients)
Show all 11Show fewer
- Enlarged pituitary adenoma (greater than 1 cm in vertical diameter and leaving less than 2 mm from the chiasma) or compression of the optic chiasma on the pituitary MRI for patients with Cushing's disease
- Severe uncontrolled hypertension (>180/110 mmHg) despite anti-hypertensive therapy (for otherwise eligible patients, blood pressure medication may be adjusted to meet this criterion)
- Severe hypokalemia (< 2.5 mmol/L) despite corrective measures
- White blood cell counts <3 x 109 /L; hemoglobin <10 g/dL; platelets <100 x 109 /L
- Any other severe acute or chronic medical or psychiatric condition, or laboratory abnormality that in the judgment of the investigator, would present excess risk associated with study participation or study drug administration, or which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
Trial locations and contacts
36 sites in 8 countries.
36 sites in 8 countries
Italy7 sites
Grande Ospedale Metropolitano Niguarda
Milan
Ospedale San Luca IRCCS Istituto Auxologico Italiano
Milan
S. Giuseppe Hospital
Milan
Federico II University
Naples
San Luigi Gonzaga Hospital, University of Turin
Orbassano, Turin, 10043
University of Padova
Padova
University of Turin
Turin
Romania7 sites
Institutul National de Endocrinologie 'C.I Parhon' - Endocrinologie II
Bucharest
Institutul National de Endocrinologie 'C.I Parhon' - Endocrinologie VI
Bucharest
Spitalul Universitar de Urgenta Militar Central 'Dr Carol Davila'
Bucharest
Spitalul Clinic Judetean de Urgenta Cluj-Napoca
Cluj-Napoca
S.C Centrul Clinic Mediquest SRL
Sângeorgiu de Mureş
Spitalul Clinic Judetean de Urgenta Sibiu
Sibiu
Spitalul Clinic Judetean Mures
Târgu Mureş
Hungary5 sites
State health center
Budapest, 10062
Semmelweis Egyetem II. Belgyógyászati Klinika
Budapest
University Debrecen
Debrecen
University of Pecs
Pécs
University of Szeged
Szeged
Belgium4 sites
University hospital Antwerp
Antwerp
CHU Erasme
Brussels
University hospital Saint Luc
Brussels
CHU Liège
Liège
Germany4 sites
Charité Berlin
Berlin, 10117
Universitätsklinikum Essen
Essen
Munich university
Munich
University hospital Wuerzburg
Würzburg
Turkey (Türkiye)4 sites
Ankara Numune Training and Research Hospital
Ankara
Dokuz Eylul UMF
Izmir
Ondokuz Mayıs University Medical Faculty
Samsun
Karadeniz Teknik University
Trabzon
Poland3 sites
Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology
Gliwice
University Hospital
Krakow
University Clinical Hospital
Wroclaw
Spain2 sites
University hospital clinic of Barcelona
Barcelona
Hospital Universitario La Ribera
Valencia
About this study
Official title: Prospective, Single Arm, Open-label, Multicenter, International Study to Assess the Effects of Metyrapone in Patients With Endogenous Cushing's Syndrome During a 12-week Treatment Period Followed by an Extension Period of 24 Weeks
The purpose of this prospective, international phase III/IV study is to assess the efficacy and safety of metyrapone in patients with endogenous Cushing's syndrome during up to 36 weeks of treatment. The ability of metyrapone (250 mg capsules) to normalize urinary free cortisol (UFC) levels will be assessed during up to 36 weeks (9 months) of treatment.
Read the detailed description
This study will include Cushing disease patients with persistent or recurrent disease (after pituitary surgery) or who are newly diagnosed but are unsuitable for early surgery or wish to defer surgery. It will also include patients with ectopic ACTH syndrome (either occult, after surgery failure, or inoperable or metastatic) and patients with Cushing's syndrome from adrenal causes.
The ability of metyrapone to normalize urinary free cortisol levels will be assessed during up to 36 weeks (9 months) of treatment. Patients participating in this study and who are controlled or close to the target at the end of a 3-months period may continue with an optional extension period of 6 months in which the long-term efficacy and safety profiles of metyrapone will be assessed. This extension study is intended to provide new findings to consolidate existing efficacy and safety data on metyrapone in the treatment of Cushing's syndrome.
- Conditions
- Cushing's Syndrome
- Being tested
- metyrapone
- Medical subject headings
- Cushing Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
metyraponeDrug
Also called Metopirone
Single arm study
| Study group | Role | What they receive |
|---|---|---|
| Metyrapone | Experimental | Metyrapone will be administered orally in an open-label fashion. Two possible initiation doses will be used depending on the severity of hypercortisolism, dose will then be adjusted (up or down-titrated) during the first month on an individual basis according to clinical tolerance and cortisol levels achieved. Receives: Drug: metyrapone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Normalization of cortisol levels (urinary free cortisol)
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2014 (estimated)
- Study startApr 2015
- Primary completionNov 2019
- Study completion29 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cushing's SyndromeStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- HRA Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02297945
- Sponsor's protocol ID
- HRA112025-002
- Acronym
- PROMPT
- Status last verified
- Sep 2020
- First submitted
- 19 Nov 2014
- Last updated
- 25 Sep 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Single Arm, Open-label, Multicenter, International Study to Assess the Effects of Metyrapone in Patients With Endogenous Cushing's Syndrome During a 12-week Treatment Period Followed by an Extension Period of 24 Weeks
- ClinicalTrials.gov ID
- NCT02297945
- Acronym / sponsor ID
- PROMPT / HRA112025-002
- Conditions
- Cushing's Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- HRA PharmaIndustry (pharma, biotech, devices)
- Study start
- Apr 2015 (actual)
- Primary completion
- Nov 2019 (actual)
- Study completion
- 29 Apr 2020 (actual)
- First posted
- 21 Nov 2014 (estimated)
- 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.
The purpose of this prospective, international phase III/IV study is to assess the efficacy and safety of metyrapone in patients with endogenous Cushing's syndrome during up to 36 weeks of treatment. The ability of metyrapone (250 mg capsules) to normalize urinary free cortisol (UFC) levels will be assessed during up to 36 weeks (9 months) of treatment.
Detailed description
This study will include Cushing disease patients with persistent or recurrent disease (after pituitary surgery) or who are newly diagnosed but are unsuitable for early surgery or wish to defer surgery. It will also include patients with ectopic ACTH syndrome (either occult, after surgery failure, or inoperable or metastatic) and patients with Cushing's syndrome from adrenal causes.
The ability of metyrapone to normalize urinary free cortisol levels will be assessed during up to 36 weeks (9 months) of treatment. Patients participating in this study and who are controlled or close to the target at the end of a 3-months period may continue with an optional extension period of 6 months in which the long-term efficacy and safety profiles of metyrapone will be assessed. This extension study is intended to provide new findings to consolidate existing efficacy and safety data on metyrapone in the treatment of Cushing's syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Metyrapone | Experimental | Drug: metyraponeMetyrapone will be administered orally in an open-label fashion. Two possible initiation doses will be used depending on the severity of hypercortisolism, dose will then be adjusted (up or down-titrated) during the first month on an individual basis according to clinical tolerance and cortisol levels achieved. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Cushing disease patients with persistent or recurrent disease (after pituitary surgery) or who are newly diagnosed but are unsuitable for early surgery or wish to defer surgery;
- Patients with ectopic ACTH syndrome (either occult, after surgery failure, or inoperable or metastatic);
- Patients with Cushing's syndrome from adrenal causes
Key exclusion criteria (11 listed)
- Pseudo Cushing's syndrome
- Cyclic Cushing's syndrome defined by at least one normal UFC value among at least three 24-hour urinary sampling measurements over the previous 2 months
- Advanced adrenocortical carcinoma or ectopic ACTH secretion (EAS) secondary to a small cell lung carcinoma
- Life expectancy less than 3 months
- Pituitary or adrenal surgery or pituitary irradiation or surgery of the ACTH-secreting ectopic tumor or bilateral adrenalectomy planned before the week 12 visit
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| metyraponeAlso: MetopironeSingle arm study | Drug | Metyrapone |
Primary outcome
Normalization of cortisol levels (urinary free cortisol)
Time frame: 3 months
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 25 Sep 2020.
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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.
36 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing metyrapone in people with Cushing's Syndrome, run by HRA Pharma at 36 sites in 8 countries.
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 4 inclusion and 11 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 25 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in Italy, Romania, Hungary, Belgium, Germany and 3 more countries. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Normalization of cortisol levels (urinary free cortisol) (time frame: 3 months). 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 25 Sep 2020.