A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

January 4, 2024 updated by: Spruce Biosciences

A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects With Classic Congenital Adrenal Hyperplasia

An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a study that will test the efficacy and safety of Tildacerfont. The first 12-weeks will be a double-blind, placebo controlled, dose ranging study. The following 58-weeks will assess the long term safety of Tildacerfont. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.

Study Type

Interventional

Enrollment (Actual)

96

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
      • Brisbane, Australia
        • Spruce Study Site
      • Elizabeth Vale, Australia
        • Spruce Study Site
      • Melbourne, Australia
        • Spruce Study Site
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Spruce Study Site
  • Brazil
      • Brasília, Brazil
        • Spruce Study Site
      • São Paulo, Brazil
        • Spruce Study Site
  • Canada
      • Ottawa, Canada, K1H7W9
        • Spruce Study Site
      • Sherbrooke, Canada, J1H 5N4
        • Spruce Study Site
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Canada
        • Spruce Study Site
    • Ontario
      • London, Ontario, Canada
        • Spruce Study Site
  • Denmark
      • Aarhus, Denmark
        • Spruce Study Site
      • Copenhagen, Denmark
        • Spruce Study Site
  • Estonia
      • Tallinn, Estonia
        • Spruce Study Site
      • Tartu, Estonia
        • Spruce Study Site
  • Germany
      • Munich, Germany
        • Spruce Study Site
  • Ireland
      • Dublin, Ireland
        • Spruce Study Site
  • Italy
      • Milan, Italy
        • Spruce Study Site
      • Napoli, Italy
        • Spruce Study Site
      • Rome, Italy
        • Spruce Study Site
      • Torino, Italy
        • Spruce Study Site
  • Korea, Republic of
      • Seoul, Korea, Republic of
        • Spruce Study Site
  • Latvia
      • Riga, Latvia
        • Spruce Study Site
  • Lithuania
      • Kaunas, Lithuania
        • Spruce Study Site
  • Netherlands
      • Nijmegen, Netherlands
        • Spruce Study Site
  • Poland
      • Kraków, Poland
        • Spruce Study Site
      • Warsaw, Poland
        • Spruce Study Site
  • Romania
      • Bucharest, Romania
        • Spruce Study Site
  • Spain
      • Barcelona, Spain
        • Spruce Study Site
      • Madrid, Spain
        • Spruce Study Site
      • Sevilla, Spain
        • Spruce Study Site
      • Tarragona, Spain
        • Spruce Study Site
  • Sweden
      • Falun, Sweden
        • Spruce Study Site
      • Stockholm, Sweden
        • Spruce Study Site
  • Switzerland
      • Saint Gallen, Switzerland
        • Spruce Study Site
      • Zürich, Switzerland
        • Spruce Study Site
  • Turkey
      • Istanbul, Turkey
        • Spruce Study Site
  • United Kingdom
      • Birmingham, United Kingdom
        • Spruce Study Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Spruce Study Site
    • California
      • Los Angeles, California, United States, 90027
        • Spruce Study Site
      • Orange, California, United States, 92868
        • Spruce Clinical Site
      • Sacramento, California, United States, 95817
        • Spruce Study Site
    • Colorado
      • Englewood, Colorado, United States, 80113
        • Spruce Study Site
    • Florida
      • Tampa, Florida, United States, 33612
        • Spruce Clinical Site
      • West Palm Beach, Florida, United States, 33401
        • Spruce Study Site
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Spruce Study Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Spruce Clinical Site
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Spruce Study Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Spruce Clinical Site
      • Rochester, Minnesota, United States, 55905
        • Spruce Study Site
    • Nevada
      • Las Vegas, Nevada, United States, 89148
        • Spruce Clinical Site
    • North Carolina
      • Hickory, North Carolina, United States, 28601
        • Spruce Study Site
    • Ohio
      • Canton, Ohio, United States, 44718
        • Spruce Study Site
      • Cincinnati, Ohio, United States, 45219
        • Spruce Study Site
      • Cleveland, Ohio, United States, 44195
        • Spruce Study Site
      • Columbus, Ohio, United States, 43210
        • Spruce Study Site
    • Oregon
      • Bend, Oregon, United States, 97702
        • Spruce Clinical Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Spruce Study Site
      • Philadelphia, Pennsylvania, United States, 19107
        • Spruce Study Site
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Spruce Study Site
    • South Carolina
      • Columbia, South Carolina, United States, 28203
        • Spruce Study Site
    • Tennessee
      • Memphis, Tennessee, United States, 38163
        • Spruce Clinical Site
    • Texas
      • Austin, Texas, United States, 78731
        • Spruce Study Site
      • Dallas, Texas, United States, 75093
        • Spruce Study Site
      • Edinburg, Texas, United States, 78539
        • Spruce Clinical Site
      • Fort Worth, Texas, United States, 76104
        • Spruce Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male and female subjects over 18 years old, inclusive
  • Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
  • Has been on a stable supraphysiologic dose of GC replacement ≥15 mg/day and ≤60 mg/day in HC equivalents
  • For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening

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. Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (eg, HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥1 month before screening.
  • Shows clinical signs or symptoms of adrenal insufficiency

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Tildacerfont Group 1
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Names:
  • SPR001
Experimental: Tildacerfont Group 2
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Names:
  • SPR001
Experimental: Tildacerfont Group 3
Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Names:
  • SPR001
Placebo Comparator: Placebo
Placebo administered daily via oral tablet for 12 weeks.
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Names:
  • SPR001

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in androstenedione
Time Frame: 12 weeks
Percent change of androstenedione
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of subjects who achieve reduction A4 levels
Time Frame: 12 weeks
Proportion of subjects who achieve A4 ≤ ULN
12 weeks
Proportion of subjects who achieve reduction in 17-OHP
Time Frame: 12 weeks
Proportion of subjects who achieve 17-OHP≤ 1200ng/dL
12 weeks
Effectiveness in reducing TART(s) in Male CAH subjects
Time Frame: 12 weeks
Change in lesion volume of TART(s) from baseline
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Spruce Biosciences

Investigators

  • Principal Investigator: Kyriakie Sarafoglou, M.D, Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 26, 2020

Primary Completion (Estimated)

March 1, 2024

Study Completion (Estimated)

November 1, 2029

Study Registration Dates

First Submitted

June 25, 2020

First Submitted That Met QC Criteria

June 30, 2020

First Posted (Actual)

July 7, 2020

Study Record Updates

Last Update Posted (Estimated)

January 8, 2024

Last Update Submitted That Met QC Criteria

January 4, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SPR001-203
  • CAHmelia 203 (Other Identifier: Spruce Biosciences)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Congenital Adrenal Hyperplasia

Clinical Trials on Tildacerfont/Placebo

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Burkina Faso

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe