Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

Sponsor

Spruce Biosciences (Industry)

Overall Status

Completed

CT.gov ID

NCT03257462

Collaborator

(none)

Enrollment

Locations

Arms

Actual Duration (Months)

2.9

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).

Condition or Disease	Intervention/Treatment	Phase
Congenital Adrenal Hyperplasia CAH - Congenital Adrenal Hyperplasia	Drug: SPR001	Phase 2

Detailed Description

This is a 6-week, multiple-dose, dose escalation study of SPR001 for the treatment of adults with classic CAH. After screening, eligible patients will be enrolled into a 6-week treatment period followed by a 4-week washout/safety follow-up period.

It is initially planned that up to approximately 18 patients in 2 dose cohorts will be enrolled. Additional patients or dose groups may be considered based upon specific safety, PK/PD, and/or efficacy findings, or if an active dose has not yet been reached.

SPR001 will be administered as an oral daily dose. Patients will undergo titration of SPR001 through three escalating dosage strengths at 2-week intervals. Patients will have overnight PK/PD assessments performed at baseline, which include an pre-dose overnight assessment and a post-dose overnight assessment for PK/PD following administration of the first dose. At the end of each 2-week dosing period, patients will return for single overnight visits for steady-state PK/PD assessments.

A follow-up outpatient visit will occur 30 days after their last dose.

Study Design

Study Type:

Interventional

Actual Enrollment :

26 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH)

Actual Study Start Date :

Jul 26, 2017

Actual Primary Completion Date :

Apr 15, 2019

Actual Study Completion Date :

May 28, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort A The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks.	Drug: SPR001 SPR001 Capsules
Experimental: Cohort B Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A.	Drug: SPR001 SPR001 Capsules
Experimental: Cohort C Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B.	Drug: SPR001 SPR001 Capsules

Arm

Intervention/Treatment

Experimental: Cohort A

The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks.

Drug: SPR001

SPR001 Capsules

Experimental: Cohort B

Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A.

Drug: SPR001

SPR001 Capsules

Experimental: Cohort C

Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B.

Drug: SPR001

SPR001 Capsules

Outcome Measures

Primary Outcome Measures

Safety of SPR001 in patients with CAH [6 weeks]
Incidence of treatment-emergent adverse events; changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters
Change in 17-hydroxyprogesterone [6 weeks]
Change in 17-hydroxyprogesterone from Baseline to End-of-study

Secondary Outcome Measures

Changes in pharmacodynamic (PD) markers [6 weeks]
Changes in ACTH and androgens from Baseline to End-of-study
Maximum plasma concentration (Cmax) [6 weeks]
To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH
Area under the concentration-time curve (AUC) [6 weeks]
To evaluate the PK parameter of area under the concentration-time curve (AUC) of SPR001 in patients with CAH
PK/PD relationships [6 weeks]
To explore the potential relationships between pharmacokinetics and pharmacodynamics

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male and female patients age 18 or older.
Documented diagnosis of classic CAH due to 21-hydroxylase deficiency
Elevated 17-OHP at screening
On a stable glucocorticoid replacement regimen for a minimum of 30 days

Exclusion Criteria:

Clinically significant unstable medical condition, illness, or chronic disease
Clinically significant psychiatric disorder.
Clinically significant abnormal laboratory finding or assessment
History of bilateral adrenalectomy or hypopituitarism
Pregnant or nursing females
Use of any other investigational drug within 30 days
Unable to understand and comply with the study procedures, understand the risks, and/or unwilling to provide written informed consent.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Spruce Biosciences Clinical Site	Orange	California	United States	92123
2	Spruce Biosciences Clinical Site	San Diego	California	United States	92123
3	Spruce Biosciences Clinical Site	Melbourne	Florida	United States	32935
4	Spruce Biosciences Clinical Site	Atlanta	Georgia	United States	30046
5	Spruce Biosciences Clinical Site	Indianapolis	Indiana	United States	46202
6	Spruce Biosciences Clinical Site	Ann Arbor	Michigan	United States	48109
7	Spruce Biosciences Clinical Site	Minneapolis	Minnesota	United States	55414
8	Spruce Biosciences Clinical Site	Las Vegas	Nevada	United States	89148
9	Spruce Biosciences Clinical Site	Philadelphia	Pennsylvania	United States	19104