A Study to Evaluate the Safety and Effectiveness of SPR001 in Adults with Classic Congenital Adrenal Hyperplasia

Overview

About this study

The purpose of this study is to evaluate the effect of Tildacerfont in reducing androstenedione (hormone, A4) in subjects with Classic Adrenal Hyperplasia (CAH) over 12 weeks.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Male and female subjects.
  • 18 years old at screening.
  • Has a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or elevated 17-OHP.
  • Has A4 > 1.5 x ULN and ACTH > 2 x ULN at both screening and Week 4 (measured before any AM GC dose).
  • Has been on a stable dose of GC replacement ≥ 15 mg/day and ≤ 50 mg/day in HCe that does not vary in total daily dose from day to day for ≥ 1 month before screening without any evidence of non-adherence to the GC regimen during this period (stress dosing is allowed).
  • For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening.
  • Agrees to follow contraception guidelines. Male subjects must also agree to refrain from donating sperm throughout the treatment period and for 90 days after the last dose of study drug.
  • Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol.

Exclusion Criteria:

  • Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency).
  • Has a history that includes bilateral adrenalectomy or hypopituitarism
  • Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist.
  • Current treatment with dexamethasone as GC therapy for CAH a. Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (e.g., HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥ 1 month before screening.
  • Is not adherent to GC or study drug dosing regimen during the Run-in Period (defined as taking 12 for either depression or anxiety at screening or Week 6.
  • Has clinically significant abnormal ECG or clinical laboratory results. Abnormal results that must be reviewed and discussed with the Medical Monitor to determine eligibility for this study include but are not limited to:
    • Any clinically meaningful abnormal ECG results, including QTcF > 450 ms for male participants or > 470 ms for female participants;
    • ALT > 2 x ULN;
    • Total bilirubin >1.5 x ULN (isolated total bilirubin >1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin is 5x ULN.
    • Routinely works overnight shifts
    • Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (> 2 hours) will require Medical Monitor approval for enrollment.
    • Females who are pregnant or nursing.
    • Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study
    • Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before Day 1 to the end of the study:
    • Rosiglitazone, aromatase inhibitors, testosterone, growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results;
    • The drugs listed in Section 13.1, which are:
    • Moderate to strong inhibitors and/or inducers of CYP3A4;
    • Sensitive substrates or narrow-therapeutic-range substrates of CYP3A4 (except hormonal contraception containing ≤ 20 µg ethinyl estradiol);
    • Sensitive substrates or narrow-therapeutic-range substrates of BCRP (except those that can be administered QD in the morning, separated by approximately 10 hours from evening administration of study drug).
  • Use of any anticoagulants or antiplatelet therapies other than aspirin within 30 days before screening.
  • Has a history of an active bleeding disorder.
  • Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study.

Eligibility last updated 31/6/22. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Irina Bancos, M.D.

Open for enrollment

Contact information:

Vanessa Fell

(507) 266-6068

Fell.Vanessa@mayo.edu

More information

Publications

Publications are currently not available
.

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