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Nevanimibe HCl for the Treatment of Classic CAH

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03669549
Recruitment Status : Recruiting
First Posted : September 13, 2018
Last Update Posted : May 3, 2019
Information provided by (Responsible Party):
Millendo Therapeutics, Inc. ( Millendo Therapeutics US, Inc. )

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 12-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 20-38 weeks.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Drug: Nevanimibe hydrochloride Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia
Actual Study Start Date : August 1, 2018
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : March 2020

Arm Intervention/treatment
Experimental: Nevanimibe hydrochloride
Ascending dose level of oral nevanimibe hydrochloride beginning with 1000 mg BID up to 2000 mg BID
Drug: Nevanimibe hydrochloride
During the 12-week treatment period, all subjects will begin dosing with nevanimibe HCl 1000 mg BID and be dose titrated to 1500 mg BID and then 2000 mg BID based upon the 17-OHP response that will be assessed every 4 weeks.

Primary Outcome Measures :
  1. Percentage of subjects achieving serum 17-OHP targets [ Time Frame: Through Day 85 ]
    <= 2x ULN

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03669549

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Contact: Marian Ijzerman, PhD 734-845-9300
Contact: Eric Henricks, BS 734-845-9423

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Institute of Endocrinology Recruiting
Praha 1, Czechia
Contact: Katerina Lunakova    +420 224 905 357   
Principal Investigator: Jana Vrbikova, MD, PhD         
Hospital Cardiologique Louis Pradel, Groupement Hospitalier Est Not yet recruiting
Bron, France
Contact: Farida Smail   
Principal Investigator: Aude Brac de la Perrière, MD         
Hospital Pitié-Salpetrière Recruiting
Paris, France
Contact: Jerome Dulon    +33 142 16 02 11   
Principal Investigator: Philippe Touraine, MD         
Bnai Zion Medical Center Recruiting
Haifa, Israel
Contact: Michal Yeiches    +972-4-8359484   
Principal Investigator: Mohammad Sheikh-Ahmad, MD         
Beilinson Hospital Recruiting
Petach Tikva, Israel
Contact: Liat Rot    +972-39211403   
Principal Investigator: Ilan Shimon, MD         
Tel-Aviv-Sourasky Medical Center Recruiting
Tel Aviv, Israel
Contact: Miri Margaliot    +972-52-6110243   
Principal Investigator: Naftali Stern, MD         
Hospital General Universitario Gregorio Marañón Recruiting
Madrid, Spain
Contact: Sara Calvo    +34 915 86 81 12   
Principal Investigator: Rogelio Garcia Centeno, MD         
Hospital Universitario Virgen del Rocío Recruiting
Sevilla, Spain
Contact: Cristina Muñoz Gómez    +34 955 01 37 24   
Principal Investigator: Miguel Ángel Mangas Cruz, MD         
University Hospital La Fe Recruiting
Valencia, Spain
Contact: Ana I Catalá    +34 961 24 55 54   
Principal Investigator: Juan Francisco Merino-Torres, MD         
Sponsors and Collaborators
Millendo Therapeutics US, Inc.

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Responsible Party: Millendo Therapeutics US, Inc. Identifier: NCT03669549     History of Changes
Other Study ID Numbers: ATR-101-202
First Posted: September 13, 2018    Key Record Dates
Last Update Posted: May 3, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Millendo Therapeutics, Inc. ( Millendo Therapeutics US, Inc. ):

Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders