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

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: NCT02804178
Recruitment Status : Completed
First Posted : June 17, 2016
Last Update Posted : March 21, 2018
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE June 1, 2016
First Posted Date  ICMJE June 17, 2016
Last Update Posted Date March 21, 2018
Actual Study Start Date  ICMJE May 18, 2016
Actual Primary Completion Date August 17, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 14, 2016)
Reduction of 17-OHP to </= 2X ULN [ Time Frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels. ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02804178 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 14, 2016)
  • Number of participants with abnormal laboratory values and/or adverse events that are related to treatment [ Time Frame: Baseline, Day 1 and Day 15 of each dose level. Each subject will have up to 5 dose levels. ]
    Safety evaluations will include adverse events, vital signs, physical examinations, laboratory measures and ECGs.
  • Pharmacokinetics: Area under the curve [ Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4 hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
  • Pharmacokinetics: Maximum plasma concentration [ Time Frame: Evaluated at Day 1 (predose, 1, 2, 4, hours post-dose and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
  • Pharmacokinetics: Time to maximum concentration [ Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4, hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Official Title  ICMJE A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Congenital Adrenal Hyperplasia
Intervention  ICMJE Drug: ATR-101
Study Arms  ICMJE Experimental: ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth BID up to 1000 mg BID.
Intervention: Drug: ATR-101
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 21, 2017)
15
Original Estimated Enrollment  ICMJE
 (submitted: June 14, 2016)
17
Actual Study Completion Date  ICMJE August 17, 2017
Actual Primary Completion Date August 17, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

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

Exclusion Criteria:

  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02804178
Other Study ID Numbers  ICMJE ATR-101-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Millendo Therapeutics, Inc.
Study Sponsor  ICMJE Millendo Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Millendo Therapeutics, Inc.
Verification Date March 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP