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

Sponsor

Spruce Biosciences (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04457336

Collaborator

(none)

Enrollment

Locations

Arms

50.2

Anticipated Duration (Months)

1.3

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Congenital Adrenal Hyperplasia	Drug: Tildacerfont/Placebo	Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

72 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Subjects will be randomized in a 1:1:1:1 manner to one of three doses of Tildacerfont or Placebo.Subjects will be randomized in a 1:1:1:1 manner to one of three doses of Tildacerfont or Placebo.

Masking:

Triple (Participant, Care Provider, Investigator)

Masking Description:

Double-Blind

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Aug 26, 2020

Anticipated Primary Completion Date :

Dec 1, 2023

Anticipated Study Completion Date :

Nov 1, 2024

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

Change in androstenedione [12 weeks]
Percent change of androstenedione

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 supraphsiologic 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Spruce Study Site	Birmingham	Alabama	United States	35294
2	Spruce Clinical Site	Orange	California	United States	92868
3	Spruce Clinical Site	Sacramento	California	United States	95821
4	Spruce Study Site	Englewood	Colorado	United States	80113
5	Spruce Clinical Site	Tampa	Florida	United States	33612
6	Spruce Study Site	Chicago	Illinois	United States	60611
7	Spruce Clinical Site	Indianapolis	Indiana	United States	46202
8	Spruce Study Site	Kansas City	Kansas	United States	66160
9	Spruce Study Site	Jefferson	Louisiana	United States	70121
10	Spruce Study Site	Baltimore	Maryland	United States	21287
11	Spruce Study Site	Camp Springs	Maryland	United States	20746
12	Spruce Study Site	Boston	Massachusetts	United States	02111
13	Spruce Clinical Site	Minneapolis	Minnesota	United States	55454
14	Spruce Study Site	Rochester	Minnesota	United States	55905
15	Spruce Study Site	Jackson	Mississippi	United States	39216
16	Spruce Clinical Site	Las Vegas	Nevada	United States	89148
17	Spruce Study Site	Reno	Nevada	United States	89557
18	Spruce Study Site	New Brunswick	New Jersey	United States	08901
19	Spruce Study Site	Bronx	New York	United States	10467
20	Spruce Study Site	Williamsville	New York	United States	14221
21	Spruce Study Site	Hickory	North Carolina	United States	28601
22	Spruce Study Site	Canton	Ohio	United States	44718
23	Spruce Study Site	Cincinnati	Ohio	United States	45219
24	Spruce Study Site	Cleveland	Ohio	United States	44195
25	Spruce Study Site	Columbus	Ohio	United States	43210
26	Spruce Clinical Site	Edmond	Oklahoma	United States	73034
27	Spruce Clinical Site	Bend	Oregon	United States	97702
28	Spruce Study Site	Philadelphia	Pennsylvania	United States	19104
29	Spruce Study Site	Philadelphia	Pennsylvania	United States	19107
30	Spruce Study Site	Philadelphia	Pennsylvania	United States	19140
31	Spruce Study Site	Providence	Rhode Island	United States	02903
32	Spruce Study Site	Columbia	South Carolina	United States	29203
33	Spruce Clinical Site	Memphis	Tennessee	United States	38163
34	Spruce Study Site	Dallas	Texas	United States	75093
35	Spruce Clinical Site	Edinburg	Texas	United States	78539
36	Spruce Clinical Site	Fort Worth	Texas	United States	76104
37	Spruce Study Site	Richmond	Virginia	United States	23298
38	Spruce Clinical Site	Seattle	Washington	United States	98105
39	Spruce Study Site	Nedlands	Western Australia	Australia	6009
40	Spruce Study Site	Melbourne		Australia
41	Spruce Study Site	Sydney		Australia
42	Spruce Study Site	London	Ontario	Canada
43	Spruce Study Site	Sherbrooke		Canada	J1H 5N4
44	Spruce Study Site	St. John's		Canada
45	Spruce Study Site	Aarhus		Denmark
46	Spruce Study Site	Copenhagen		Denmark
47	Spruce Study Site	Munich		Germany
48	Spruce Study Site	Rome		Italy
49	Spruce Study Site	Nijmegen		Netherlands
50	Spruce Study Site	Kraków		Poland
51	Spruce Study Site	Barcelona		Spain
52	Spruce Study Site	Madrid		Spain
53	Spruce Study Site	Sevilla		Spain
54	Spruce Study Site	Falun		Sweden
55	Spruce Study Site	Birmingham		United Kingdom
56	Spruce Study Site	Liverpool		United Kingdom
57	Spruce Study Site	London		United Kingdom