Congenital Adrenal HyperplasiaPhase 2NCT03669549
Nevanimibe HCl for the Treatment of Classic CAH
A Phase 2 study testing Nevanimibe hydrochloride in people with Congenital Adrenal Hyperplasia, run by Millendo Therapeutics US, Inc. at 11 sites in 5 countries.
Trial summary
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- Nevanimibe hydrochloride
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 11 Jul 2018to 12 Jul 2020
- 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 and/or historical documentation of elevated 17-OHP
- Chronic glucocorticoid replacement therapy for at least 6 consecutive months prior to screening
- Stable glucocorticoid and mineralocorticoid regimen for at least 4 weeks prior to screening and throughout the treatment period of the study
Who cannot join
- Nonclassic CAH
- Other causes of adrenal insufficiency
- HIV, hepatitis B, or hepatitis C
- AST or ALT >2x ULN, bilirubin or serum creatinine >1.5x ULN
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
Brazil3 sites
University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto
Ribeirão Preto, 14051-140
Universidade Federal de São Paulo, Escola Paulista de Medicina
São Paulo, 04037-002
Hospital das Clínicas da FMUSP - Prédio do Instituto Central
São Paulo, 05403-900
Israel3 sites
Bnai Zion Medical Center
Haifa
Beilinson Hospital
Petah Tikva
Tel-Aviv-Sourasky Medical Center
Tel Aviv
Spain3 sites
Hospital General Universitario Gregorio Marañón
Madrid
Hospital Universitario Virgen del Rocío
Seville
University Hospital La Fe
Valencia
About this study
Official title: A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- Nevanimibe hydrochloride
- Keywords
- CAH, nevanimibe
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nevanimibe hydrochlorideDrug
During the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Nevanimibe hydrochloride | Experimental | Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID Receives: Drug: Nevanimibe hydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Subjects Achieving Serum 17-OHP Targets
The primary efficacy endpoint was the overall response rate within each cohort, defined as the percentage of patients achieving serum 17-OHP targets as follows:
- Men and postmenopausal women: 17-OHP ≤ 2x ULN
- Premenopausal women:
- Follicular phase: 17-OHP ≤ 2x follicular phase ULN
- Luteal phase: 17-OHP ≤ (2x follicular phase ULN + (luteal phase ULN - follicular phase ULN))
Measured: Through Day 113
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2018
- Study start11 Jul 2018
- Primary completion3 Jun 2020
- Study completion12 Jul 2020
Why it stopped: Following an interim data review, further investment in nevanimibe has been discontinued
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
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Millendo Therapeutics US, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03669549
- Sponsor's protocol ID
- ATR-101-202
- Status last verified
- Feb 2021
- First submitted
- 10 Sep 2018
- Last updated
- 4 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
White PC. Emerging treatment for congenital adrenal hyperplasia. Curr Opin Endocrinol Diabetes Obes. 2022 Jun 1;29(3):271-276. doi: 10.1097/MED.0000000000000723.
PubMed 35283460
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT03669549
- Acronym / sponsor ID
- ATR-101-202
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- CAH, nevanimibe
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Following an interim data review, further investment in nevanimibe has been discontinued
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Millendo Therapeutics US, Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Jul 2018 (actual)
- Primary completion
- 3 Jun 2020 (actual)
- Study completion
- 12 Jul 2020 (actual)
- First posted
- 13 Sep 2018 (actual)
- Results
- Posted 4 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 multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nevanimibe hydrochloride | Experimental | Drug: Nevanimibe hydrochlorideAscending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency and/or historical documentation of elevated 17-OHP
- Chronic glucocorticoid replacement therapy for at least 6 consecutive months prior to screening
- Stable glucocorticoid and mineralocorticoid regimen for at least 4 weeks prior to screening and throughout the treatment period of the study
Key exclusion criteria (4 listed)
- Nonclassic CAH
- Other causes of adrenal insufficiency
- HIV, hepatitis B, or hepatitis C
- AST or ALT >2x ULN, bilirubin or serum creatinine >1.5x ULN
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 |
|---|---|---|
| Nevanimibe hydrochlorideDuring the 16-week treatment period, all subjects will begin dosing with nevanimibe HCl 500 mg BID and be dose titrated to 1000 mg BID, 1500 mg BID, and 2000 mg BID as needed based on serum 17-OHP assessments every 4 weeks. | Drug | Nevanimibe hydrochloride |
Primary outcome
Percentage of Subjects Achieving Serum 17-OHP Targets
Time frame: Through Day 113
The primary efficacy endpoint was the overall response rate within each cohort, defined as the percentage of patients achieving serum 17-OHP targets as follows: * Men and postmenopausal women: 17-OHP ≤ 2x ULN * Premenopausal women: * Follicular phase: 17-OHP ≤ 2x follicular phase ULN * Luteal phase: 17-OHP ≤ (2x follicular phase ULN + (luteal phase ULN - follicular phase ULN))
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35283460White PC. Emerging treatment for congenital adrenal hyperplasia. Curr Opin Endocrinol Diabetes Obes. 2022 Jun 1;29(3):271-276. doi: 10.1097/MED.0000000000000723.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Nevanimibe hydrochloride in people with Congenital Adrenal Hyperplasia, run by Millendo Therapeutics US, Inc. at 11 sites in 5 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 3 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 4 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 11 sites in Brazil, Israel, Spain, Czechia, France. 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 4 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?
Percentage of Subjects Achieving Serum 17-OHP Targets (time frame: Through Day 113). 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 NCT03669549, 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 4 Mar 2021.