Cushing SyndromePhase 2NCT03053271
A Study of ATR-101 for the Treatment of Endogenous Cushing's Syndrome
A Phase 2 study testing ATR-101 in people with Cushing Syndrome, run by Millendo Therapeutics US, Inc. at 4 sites in 2 countries.
Trial summary
This is a Phase 2 multicenter, randomized, double-blind, placebo controlled study of ATR-101 to evaluate the efficacy and safety of orally-administered ATR-101 in adults with endogenous Cushing's syndrome. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Cushing Syndrome
- What is being tested?
- ATR-101
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 13 Apr 2017to 12 Aug 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of endogenous Cushing's syndrome
- Baseline UFC 1.3 to 10 × upper limit of normal (ULN)
- If previous pituitary surgery, participants must be at least 3 months since surgery at the time of screening
- BMI between 18 and 60 kg/m2, inclusive
Who cannot join
- Pseudo-Cushing's syndrome, cyclic Cushing's syndrome or current iatrogenic Cushing's syndrome
- Candidates for surgical treatment of Cushing's syndrome, unless surgery is not anticipated to occur during the study
- Normal late night salivary cortisol or 24-hr urine free cortisol
- Radiotherapy of the pituitary within 6 months
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States3 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Medical College of Wisconsin
Milwaukee, Wisconsin, 53051
United Kingdom1 site
The James Cook University Hospital
Middlesbrough, TS4 3 BW
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ATR-101 for the Treatment of Cushing's Syndrome
This is a Phase 2 multicenter, randomized, double-blind, placebo controlled study of ATR-101 to evaluate the efficacy and safety of orally-administered ATR-101 in adults with endogenous Cushing's syndrome. Following wash-out (if needed), all eligible subjects will enter an open-label intra-subject dose-escalation period of 8 weeks' duration, followed either by a double-blind randomized withdrawal period of 4 weeks' duration (if the subject meets randomization criteria) or by an additional open label dosing period of 4 weeks' duration (if the subject does not meet randomization criteria).It is anticipated that the overall duration of the study per subject will range from approximately 16-22 weeks.
- Conditions
- Cushing Syndrome
- Being tested
- ATR-101, Placebo
- Keywords
- Endogenous Cushing's Syndrome, ATR-101, CS
- Medical subject headings
- Cushing Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATR-101Drug
Also called nevanimibe HCl
During the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks at the same dose level being used at the completion of the open-label dose-escalation period.
PlaceboDrug
During the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks with placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period.
| Study group | Role | What they receive |
|---|---|---|
| ATR-101 | Experimental | During the 4-week randomized withdrawal period, eligible subjects will receive ATR-101 at the same dose level being used at the completion of the open-label dose-escalation period. Receives: Drug: ATR-101 |
| Placebo | Placebo comparator | During the 4-week randomized withdrawal period, eligible subjects will receive a placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
The number of subjects meeting the criterion was divided by the total number of subjects.
Measured: Through Day 85
Secondary outcomes (4)
The Proportion of Subjects With a Normal 24-hr UFC
The number of subjects meeting the criterion was divided by the total number of subjects.
Measured: Through Day 85
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
The number of subjects meeting the criterion was divided by the total number of subjects.
Measured: Through Day 85
The Proportion of Subjects With a Normal 24-hr UFC
The number of subjects meeting the criterion was divided by the total number of subjects.
Measured: Through Day 57 and Day 85
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
The number of subjects meeting the criterion was divided by the total number of subjects.
Measured: Through Day 57 and Day 85
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2017
- Study start13 Apr 2017
- Primary completion17 Jun 2019
- Study completion12 Aug 2019
Why it stopped: Slow enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cushing 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Millendo Therapeutics US, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- James Findling, MD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03053271
- Sponsor's protocol ID
- ATR-101-301
- Status last verified
- Feb 2021
- First submitted
- 6 Feb 2017
- Last updated
- 2 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ATR-101 for the Treatment of Cushing's Syndrome
- ClinicalTrials.gov ID
- NCT03053271
- Acronym / sponsor ID
- ATR-101-301
- Conditions
- Cushing Syndrome
- Keywords
- Endogenous Cushing's Syndrome, ATR-101, CS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow enrollment
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Millendo Therapeutics US, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Apr 2017 (actual)
- Primary completion
- 17 Jun 2019 (actual)
- Study completion
- 12 Aug 2019 (actual)
- First posted
- 15 Feb 2017 (actual)
- Results
- Posted 2 Mar 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2 multicenter, randomized, double-blind, placebo controlled study of ATR-101 to evaluate the efficacy and safety of orally-administered ATR-101 in adults with endogenous Cushing's syndrome. Following wash-out (if needed), all eligible subjects will enter an open-label intra-subject dose-escalation period of 8 weeks' duration, followed either by a double-blind randomized withdrawal period of 4 weeks' duration (if the subject meets randomization criteria) or by an additional open label dosing period of 4 weeks' duration (if the subject does not meet randomization criteria).It is anticipated that the overall duration of the study per subject will range from approximately 16-22 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ATR-101 | Experimental | Drug: ATR-101During the 4-week randomized withdrawal period, eligible subjects will receive ATR-101 at the same dose level being used at the completion of the open-label dose-escalation period. |
| Placebo | Placebo comparator | Drug: PlaceboDuring the 4-week randomized withdrawal period, eligible subjects will receive a placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of endogenous Cushing's syndrome
- Baseline UFC 1.3 to 10 × upper limit of normal (ULN)
- If previous pituitary surgery, participants must be at least 3 months since surgery at the time of screening
- BMI between 18 and 60 kg/m2, inclusive
Key exclusion criteria (4 listed)
- Pseudo-Cushing's syndrome, cyclic Cushing's syndrome or current iatrogenic Cushing's syndrome
- Candidates for surgical treatment of Cushing's syndrome, unless surgery is not anticipated to occur during the study
- Normal late night salivary cortisol or 24-hr urine free cortisol
- Radiotherapy of the pituitary within 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.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ATR-101Also: nevanimibe HClDuring the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks at the same dose level being used at the completion of the open-label dose-escalation period. | Drug | ATR-101 |
| PlaceboDuring the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks with placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period. | Drug | Placebo |
Primary outcome
The Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
Time frame: Through Day 85
The number of subjects meeting the criterion was divided by the total number of subjects.
4 secondary outcomes
The Proportion of Subjects With a Normal 24-hr UFC
Time frame: Through Day 85
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
Time frame: Through Day 85
The Proportion of Subjects With a Normal 24-hr UFC
Time frame: Through Day 57 and Day 85
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
Time frame: Through Day 57 and Day 85
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
James Findling, MD principal investigator
Medical College of Wisconsin
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ATR-101 in people with Cushing Syndrome, run by Millendo Therapeutics US, Inc. at 4 sites in 2 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 4 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: stopped early, as of the sponsor's last update on 2 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, United Kingdom. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value (time frame: Through Day 85). 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 2 Mar 2021.