Nevanimibe HCl for the Treatment of Classic CAH

Brief Title

Nevanimibe HCl for the Treatment of Classic CAH

Official Title

A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia

Brief 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 16-week Treatment Period. It is
      anticipated that the overall duration of the study per subject will range from 24-42 weeks.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Percentage of Subjects Achieving Serum 17-OHP Targets


Condition

Congenital Adrenal Hyperplasia

Intervention

Nevanimibe hydrochloride

Study Arms / Comparison Groups

 Nevanimibe hydrochloride
Description:  Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

15

Start Date

July 11, 2018

Completion Date

July 12, 2020

Primary Completion Date

June 3, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Brazil

Location Countries

Brazil

Administrative Informations


NCT ID

NCT03669549

Organization ID

ATR-101-202


Responsible Party

Sponsor

Study Sponsor

Millendo Therapeutics US, Inc.


Study Sponsor

, , 


Verification Date

February 2021