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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03669549
Recruitment Status : Terminated (Following an interim data review, further investment in nevanimibe has been discontinued)
First Posted : September 13, 2018
Last Update Posted : July 16, 2020
Sponsor:
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 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
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 : July 11, 2018
Actual Primary Completion Date : June 3, 2020
Actual Study Completion Date : July 12, 2020


Arm Intervention/treatment
Experimental: Nevanimibe hydrochloride
Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID
Drug: Nevanimibe hydrochloride
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.




Primary Outcome Measures :
  1. Percentage of subjects achieving serum 17-OHP targets [ Time Frame: Through Day 113 ]
    <= 2x ULN



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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
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

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 ClinicalTrials.gov identifier (NCT number): NCT03669549


Locations
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Brazil
University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto
Ribeirão Preto, Brazil, 14051-140
Universidade Federal de São Paulo, Escola Paulista de Medicina
São Paulo, Brazil, 04037-002
Hospital das Clínicas da FMUSP - Prédio do Instituto Central
São Paulo, Brazil, 05403-900
Czechia
Institute of Endocrinology
Praha 1, Czechia
France
Hospital Pitié-Salpetrière
Paris, France
Israel
Bnai Zion Medical Center
Haifa, Israel
Beilinson Hospital
Petach Tikva, Israel
Tel-Aviv-Sourasky Medical Center
Tel Aviv, Israel
Spain
Hospital General Universitario Gregorio Marañón
Madrid, Spain
Hospital Universitario Virgen del Rocío
Sevilla, Spain
University Hospital La Fe
Valencia, Spain
Sponsors and Collaborators
Millendo Therapeutics US, Inc.
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Responsible Party: Millendo Therapeutics US, Inc.
ClinicalTrials.gov Identifier: NCT03669549    
Other Study ID Numbers: ATR-101-202
First Posted: September 13, 2018    Key Record Dates
Last Update Posted: July 16, 2020
Last Verified: March 2020
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. ):
CAH
nevanimibe
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Hyperplasia
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