Clinical trial NCT03257462: Study of SPR001 in Adults with Classic Congenital Adrenal Hyperplasia
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Congenital Adrenal Hyperplasia
CAH - Congenital Adrenal Hyperplasia
Keywords: 17-hydroxyprogesterone
Lead sponsor: Spruce Biosciences
Sponsor class: INDUSTRY
First posted: 2017-08-22
Enrollment: 26
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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).

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Safety of SPR001 in patients with CAH
timeFrame: 6 weeks
measure: Change in 17-hydroxyprogesterone
timeFrame: 6 weeks
Secondary outcomes:
measure: Changes in pharmacodynamic (PD) markers
timeFrame: 6 weeks
measure: Maximum plasma concentration (Cmax)
timeFrame: 6 weeks
measure: Area under the concentration-time curve (AUC)
timeFrame: 6 weeks
measure: PK/PD relationships
timeFrame: 6 weeks
Other outcomes:
Locations:
city: Orange
country: United States
facility: Spruce Biosciences Clinical Site
state: California
city: San Diego
country: United States
facility: Spruce Biosciences Clinical Site
state: California
city: Melbourne
country: United States
facility: Spruce Biosciences Clinical Site
state: Florida
city: Atlanta
country: United States
facility: Spruce Biosciences Clinical Site
state: Georgia
city: Indianapolis
country: United States
facility: Spruce Biosciences Clinical Site
state: Indiana
city: Ann Arbor
country: United States
facility: Spruce Biosciences Clinical Site
state: Michigan
city: Minneapolis
country: United States
facility: Spruce Biosciences Clinical Site
state: Minnesota
city: Las Vegas
country: United States
facility: Spruce Biosciences Clinical Site
state: Nevada
city: Philadelphia
country: United States
facility: Spruce Biosciences Clinical Site
state: Pennsylvania
Study officials:
affiliation: Spruce Biosciences
name: Michael Huang, MD
role: STUDY_DIRECTOR
affiliation: University of Michigan
name: Richard Auchus, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: