A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH

January 22, 2024 updated by: Spruce Biosciences

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

An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic CAH subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a study that will evaluate the ability of Tildacerfont to reduce the glucocorticoid steroid dose used by adult CAH subjects. The first 24-weeks will be a double-blind, placebo controlled, comparison of Tildacerfont vs Placebo. The following 52-weeks will allow all subjects to move to open label Tildacerfont to continue to reduce steroid dose where appropriate, and observe long term safety. Subjects will be offered a long term open label extension up to 240 weeks.

Study Type

Interventional

Enrollment (Estimated)

90

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, 4029
        • Spruce Study Site
      • Camperdown, Australia
        • Spruce Study Site
      • Melbourne, Australia
        • Spruce Study Site
      • Sydney, Australia
        • Spruce Study Site
  • Brazil
      • Curitiba, Brazil
        • Spruce Study Site
      • São Paulo, Brazil
        • Spruce Study Site
  • Canada
    • Ontario
      • Ottawa, Ontario, Canada
        • Spruce Study Site
    • Quebec
      • Sherbrooke, Quebec, Canada, J1H 5N4
        • Spruce Study Site
  • Estonia
      • Tallinn, Estonia
        • Spruce Study Site
      • Tartu, Estonia
        • Spruce Study Site
  • Germany
      • Munich, Germany
        • Spruce Study Site
  • Italy
      • Roma, 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
  • Poland
      • Kraków, Poland
        • Spruce Study Site
      • Warsaw, Poland
        • Spruce Study Site
  • Romania
      • Bucharest, Romania
        • Spruce Study Site
      • Bucuresti, 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
      • Stockholm, Sweden
        • Spruce Study Site
  • Turkey
      • Istanbul, Turkey
        • Spruce Study Site
  • United Kingdom
      • Birmingham, United Kingdom, B15 2GW
        • 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
      • San Diego, California, United States, 92123
        • Spruce Study Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Spruce Study Site
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Spruce Study Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Spruce Biosciences Clinical Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Spruce Study Site
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • 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, 99702
        • Spruce Study Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Spruce Study Site
      • Philadelphia, Pennsylvania, United States, 19107
        • Spruce Study Site
      • Philadelphia, Pennsylvania, United States, 19140
        • Spruce Study Site
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Spruce Study Site
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Spruce Study Site
    • Texas
      • Dallas, Texas, United States, 75231
        • Spruce Study Site
      • Fort Worth, Texas, United States, 76104
        • Spruce Study 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 documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treatment with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
  • Has been on a stable, supraphysiologic dose of GC replacement for ≥1 month before screening.
  • 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
  • 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
Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Names:
  • SPR001
Placebo Comparator: Placebo
Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 weeks
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Names:
  • SPR001

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of subjects who can reduce GC dose at Week 24
Time Frame: 24 Weeks
Proportion of subjects with at least a 5 mg/day HCe reduction from baseline in GC dose and A4 ≤ULN at Week 24
24 Weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage change in GC use in subjects with CAH
Time Frame: 24 weeks
Percent change from baseline in GC dose at week 24
24 weeks
Change in the median cumulative HCe dose in subjects with CAH
Time Frame: 24 Weeks
Median total cumulative GC dose in HCe at Week 24
24 Weeks
Effectiveness in reducing cardiovascular risk in subjects with CAH
Time Frame: 24 Weeks
Proportion of subjects with improvement in at least one cardiovascular risk factor at week 24
24 Weeks
Effectiveness in improving HOMA-IR in subjects with CAH
Time Frame: 24 Weeks
Change from baseline in the HOMA-IR at Week 24
24 Weeks
Effect on body weight in subjects with CAH
Time Frame: 24 weeks
Percent change from baseline in body weight after 24 weeks of tildacerfont treatment
24 weeks
Effect on body weight in subjects with CAH
Time Frame: 52 weeks
Percent change from baseline in body weight after 52 weeks of tildacerfont treatment
52 weeks

Collaborators and Investigators

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

Sponsor

Spruce Biosciences

Investigators

  • Principal Investigator: Ron Newfield, M.D, Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.

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)

September 29, 2020

Primary Completion (Estimated)

September 1, 2024

Study Completion (Estimated)

September 1, 2029

Study Registration Dates

First Submitted

August 30, 2020

First Submitted That Met QC Criteria

September 3, 2020

First Posted (Actual)

September 10, 2020

Study Record Updates

Last Update Posted (Estimated)

January 24, 2024

Last Update Submitted That Met QC Criteria

January 22, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SPR001-204
  • CAHmelia 204 (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.

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