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

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2016 by Millendo Therapeutics, Inc.
Sponsor:
Information provided by (Responsible Party):
Millendo Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT02804178
First received: June 1, 2016
Last updated: December 20, 2016
Last verified: December 2016
  Purpose
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.

Condition Intervention Phase
Congenital Adrenal Hyperplasia
Drug: ATR-101
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

Resource links provided by NLM:


Further study details as provided by Millendo Therapeutics, Inc.:

Primary Outcome Measures:
  • 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. ]

Secondary Outcome Measures:
  • 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. ]

Estimated Enrollment: 17
Study Start Date: May 2016
Estimated Study Completion Date: September 2017
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth BID up to 1000 mg BID.
Drug: ATR-101

  Eligibility

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

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02804178

Contacts
Contact: Marianne Plaunt, PhD 734-249-6030 plaunt@millendo.com
Contact: Marian Ijzerman, PhD 734-845-9300 ijzerman@millendo.com

Locations
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21287
Contact: Oyinkan Kusemiju    410-955-2812    okusemi1@jhmi.edu   
Principal Investigator: Roberto Salvatori, MD         
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: Elizabeth Joyal    301-496-8542    ejoyal@mail.nih.gov   
Principal Investigator: Deborah Merke, MD         
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Timothy Muth    734-615-8914    tmuth@ed.umich.edu   
Principal Investigator: Richard Auchus, MD         
United States, Minnesota
Mayo Clinic - Rochester Recruiting
Rochester, Minnesota, United States, 55905
Contact: Samantha McAllister    507-266-3912    McAllister.Samantha@mayo.edu   
Principal Investigator: Alice Chang, MD         
United States, Oklahoma
The University of Oklahoma - Tulsa Schusterman Center Recruiting
Tulsa, Oklahoma, United States, 74135
Contact: Sharnella Goudeau    918-619-4803    sharnella-goudeau@ouhsc.edu   
Principal Investigator: Laura Chalmers, MD         
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 10021
Contact: Michelle Pitt    215-590-3174    pittm@email.chop.edu   
Principal Investigator: Maria Vogiatzi, MD         
Sponsors and Collaborators
Millendo Therapeutics, Inc.
  More Information

Responsible Party: Millendo Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02804178     History of Changes
Other Study ID Numbers: ATR-101-201
Study First Received: June 1, 2016
Last Updated: December 20, 2016
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Millendo Therapeutics, Inc.:
ATR-101
classic congenital adrenal hyperplasia
21-hydroxylase deficiency
CAH

Additional relevant MeSH terms:
Hyperplasia
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
Epinephrine
Racepinephrine
Epinephryl borate
Adrenergic alpha-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Adrenergic beta-Agonists
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Anti-Asthmatic Agents
Respiratory System Agents

ClinicalTrials.gov processed this record on May 25, 2017