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

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Tildacerfont/Placebo
Sponsored by
Spruce Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Spruce Study SiteRecruiting
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  • Spruce Biosciences Clinical SiteRecruiting
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  • Spruce Site
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Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Tildacerfont Group

Placebo

Arm Description

Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks

Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 weeks

Outcomes

Primary Outcome Measures

Proportion of subjects who can reduce GC dose at Week 24
Proportion of subjects with at least a 5 mg/day HCe reduction from baseline in GC dose and A4 ≤ULN at Week 24

Secondary Outcome Measures

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

Full Information

First Posted
August 30, 2020
Last Updated
October 9, 2023
Sponsor
Spruce Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT04544410
Brief Title
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 29, 2020 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
September 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Spruce Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia
Keywords
CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Subjects will be randomized in a 1:1 manner to either Tildacerfont or Placebo for 24 weeks followed by 52 weeks open label Tildacerfont
Masking
ParticipantCare ProviderInvestigator
Masking Description
Double-Blind for first 24 weeks, then open label
Allocation
Randomized
Enrollment
90 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Tildacerfont Group
Arm Type
Experimental
Arm Description
Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 weeks
Intervention Type
Drug
Intervention Name(s)
Tildacerfont/Placebo
Other Intervention Name(s)
SPR001
Intervention Description
Tablet, administered daily
Primary Outcome Measure Information:
Title
Proportion of subjects who can reduce GC dose at Week 24
Description
Proportion of subjects with at least a 5 mg/day HCe reduction from baseline in GC dose and A4 ≤ULN at Week 24
Time Frame
24 Weeks
Secondary Outcome Measure Information:
Title
Percentage change in GC use in subjects with CAH
Description
Percent change from baseline in GC dose at week 24
Time Frame
24 weeks
Title
Change in the median cumulative HCe dose in subjects with CAH
Description
Median total cumulative GC dose in HCe at Week 24
Time Frame
24 Weeks
Title
Effectiveness in reducing cardiovascular risk in subjects with CAH
Description
Proportion of subjects with improvement in at least one cardiovascular risk factor at week 24
Time Frame
24 Weeks
Title
Effectiveness in improving HOMA-IR in subjects with CAH
Description
Change from baseline in the HOMA-IR at Week 24
Time Frame
24 Weeks
Title
Effect on body weight in subjects with CAH
Description
Percent change from baseline in body weight after 24 weeks of tildacerfont treatment
Time Frame
24 weeks
Title
Effect on body weight in subjects with CAH
Description
Percent change from baseline in body weight after 52 weeks of tildacerfont treatment
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Clinical Trials
Phone
415-655-4169
Email
CAHmelia@sprucebiosciences.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ron Newfield, M.D
Organizational Affiliation
Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
Official's Role
Principal Investigator
Facility Information:
Facility Name
Spruce Study Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Individual Site Status
Withdrawn
Facility Name
Spruce Study Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Clinical Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95821
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Jefferson
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Biosciences Clinical Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Site
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Individual Site Status
Withdrawn
Facility Name
Spruce Study Site
City
Columbia
State/Province
Missouri
ZIP/Postal Code
65201
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89148
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Syracuse
State/Province
New York
ZIP/Postal Code
13057
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Canton
State/Province
Ohio
ZIP/Postal Code
44718
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Bend
State/Province
Oregon
ZIP/Postal Code
99702
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17003
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Spruce Study Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19140
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02903
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29203
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38163
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Edinburg
State/Province
Texas
ZIP/Postal Code
78539
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Brisbane
ZIP/Postal Code
4029
Country
Australia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Camperdown
Country
Australia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Melbourne
Country
Australia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Sydney
Country
Australia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
São Paulo
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Saint Johns
State/Province
Newfoundland and Labrador
Country
Canada
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
London
State/Province
Ontario
Country
Canada
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Ottawa
State/Province
Ontario
Country
Canada
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Sherbrooke
State/Province
Quebec
ZIP/Postal Code
J1H 5N4
Country
Canada
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Aarhus
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Copenhagen
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Tallinn
Country
Estonia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Tartu
Country
Estonia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Munich
Country
Germany
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Dublin
Country
Ireland
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Milan
Country
Italy
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Roma
Country
Italy
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Seoul
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Riga
Country
Latvia
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Kaunas
Country
Lithuania
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Nijmegen
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Kraków
Country
Poland
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Warsaw
Country
Poland
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Bucharest
Country
Romania
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Bucuresti
Country
Romania
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Madrid
Country
Spain
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Sevilla
Country
Spain
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Tarragona
Country
Spain
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Falun
Country
Sweden
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Stockholm
Country
Sweden
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Saint Gallen
Country
Switzerland
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Zürich
Country
Switzerland
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Istanbul
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Birmingham
ZIP/Postal Code
B15 2GW
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
Liverpool
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Spruce Study Site
City
London
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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