Congenital Adrenal HyperplasiaPhase 2NCT02804178

A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

A Phase 2 study testing ATR-101 in people with Congenital Adrenal Hyperplasia, run by Millendo Therapeutics, Inc. at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Congenital Adrenal Hyperplasia
What is being tested?: ATR-101
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 10 participants
Where: 6 sitesUnited States
Study dates: 18 May 2016to 17 Aug 2017
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

Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone
Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal
Chronic glucocorticoid replacement therapy for at least 6 consecutive months
Stable glucocorticoid and mineralocorticoid regimen for at least 1 month

Who cannot join

Non-classic CAH
Other causes of adrenal insufficiency
Surgery within the previous 3 months prior to screening or planned surgery during study participation
History of active cancer requiring medical or surgical therapy within the past 6 months

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

United States6 sites

University of Michigan
Ann Arbor, Michigan, 48109
Johns Hopkins University
Baltimore, Maryland, 21287
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 10021
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
The University of Oklahoma - Tulsa Schusterman Center
Tulsa, Oklahoma, 74135

Other trials in United States

About this study

Official title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.

Conditions: Congenital Adrenal Hyperplasia
Being tested: ATR-101
Keywords: ATR-101, classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, CAH
Medical subject headings: Adrenal Hyperplasia, Congenital, Congenital adrenal hyperplasia due to 21 hydroxylase deficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ATR-101Drug
Also called Nevanimibe hydrochloride
125-1000 mg twice per week

Study groups in NCT02804178
Study group	Role	What they receive
ATR-101	Experimental	Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day. Receives: Drug: ATR-101

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
Measured: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 May 201617 Aug 2017

First posted17 Jun 2016 (estimated)
Study start18 May 2016
Primary completion17 Aug 2017
Study completion17 Aug 2017

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Congenital Adrenal HyperplasiaStudies 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: Not applicable
Intervention model: Single group
Model description: Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).
Masking: None (open label)
Enrolment: 10 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02804178
Sponsor's protocol ID: ATR-101-201
Status last verified: Feb 2021
First submitted: 1 Jun 2016
Last updated: 10 Mar 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: No

Publications

El-Maouche D, Merke DP, Vogiatzi MG, Chang AY, Turcu AF, Joyal EG, Lin VH, Weintraub L, Plaunt MR, Mohideen P, Auchus RJ. A Phase 2, Multicenter Study of Nevanimibe for the Treatment of Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2020 Aug 1;105(8):2771-8. doi: 10.1210/clinem/dgaa381.
PubMed 32589738

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
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RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
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RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)

All Congenital Adrenal Hyperplasia trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
ClinicalTrials.gov ID: NCT02804178
Acronym / sponsor ID: ATR-101-201
Conditions: Congenital Adrenal Hyperplasia
Keywords: ATR-101, classic congenital adrenal hyperplasia, 21-hydroxylase deficiency, CAH
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 10 participants (actual)
Lead sponsor: Millendo Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: 18 May 2016 (actual)
Primary completion: 17 Aug 2017 (actual)
Study completion: 17 Aug 2017 (actual)
First posted: 17 Jun 2016 (estimated)
Results: Posted 17 Feb 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).

Arm	Type	Receives
ATR-101	Experimental	Drug: ATR-101Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone
Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal
Chronic glucocorticoid replacement therapy for at least 6 consecutive months
Stable glucocorticoid and mineralocorticoid regimen for at least 1 month

Key exclusion criteria (4 listed)

Non-classic CAH
Other causes of adrenal insufficiency
Surgery within the previous 3 months prior to screening or planned surgery during study participation
History of active cancer requiring medical or surgical therapy within the past 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 hydrochloride125-1000 mg twice per week	Drug	ATR-101

Primary outcome

Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
Time frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.

Results and safety

Results posted to the registry on 17 Feb 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ATR-101	10	9

Primary outcome: Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101

Time frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.. Count of participants. Unit: Participants

Measure	ATR-101 n = 10
Value	2

Adverse events

Time frame: Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ATR-101	1 of 10 (10%)	9 of 10 (90%)	0 of 10

Limitations and caveats (from the sponsor): The small number of subjects analyzed as a result of the rarity of the disease (congenital adrenal hyperplasia).and the stage of the study (phase 2a)

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32589738El-Maouche D, Merke DP, Vogiatzi MG, Chang AY, Turcu AF, Joyal EG, Lin VH, Weintraub L, Plaunt MR, Mohideen P, Auchus RJ. A Phase 2, Multicenter Study of Nevanimibe for the Treatment of Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2020 Aug 1;105(8):2771-8. doi: 10.1210/clinem/dgaa381.
Individual participant dataSharing planned: No

Related trials for Congenital Adrenal Hyperplasia

RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)

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.

6 sites in 1 country: 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 Congenital Adrenal Hyperplasia, run by Millendo Therapeutics, Inc. at 6 sites in United States.

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: finished, as of the sponsor's last update on 10 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 6 sites in United States. 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 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 17 Feb 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?

Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101 (time frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02804178, which also lists 1 linked publication.

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 10 Mar 2021; AzTrials last refreshed its copy on 8 Nov 2026.