Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03257462
• Other study ID #: SPR001-201
• Status: Completed
• Phase: Phase 2
• Start date: July 26, 2017
• Est. completion date: May 28, 2019

Study information

• Verified date: June 2018
• Source: Spruce Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: May 28, 2019
• Est. primary completion date: April 15, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• CAH - Congenital Adrenal Hyperplasia
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• SPR001
SPR001 Capsules

Locations

Country	Name	City	State
United States	Spruce Biosciences Clinical Site	Ann Arbor	Michigan
United States	Spruce Biosciences Clinical Site	Atlanta	Georgia
United States	Spruce Biosciences Clinical Site	Indianapolis	Indiana
United States	Spruce Biosciences Clinical Site	Las Vegas	Nevada
United States	Spruce Biosciences Clinical Site	Melbourne	Florida
United States	Spruce Biosciences Clinical Site	Minneapolis	Minnesota
United States	Spruce Biosciences Clinical Site	Orange	California
United States	Spruce Biosciences Clinical Site	Philadelphia	Pennsylvania
United States	Spruce Biosciences Clinical Site	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Spruce Biosciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of SPR001 in patients with CAH	Incidence of treatment-emergent adverse events; changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters	6 weeks
Primary	Change in 17-hydroxyprogesterone	Change in 17-hydroxyprogesterone from Baseline to End-of-study	6 weeks
Secondary	Changes in pharmacodynamic (PD) markers	Changes in ACTH and androgens from Baseline to End-of-study	6 weeks
Secondary	Maximum plasma concentration (Cmax)	To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH	6 weeks
Secondary	Area under the concentration-time curve (AUC)	To evaluate the PK parameter of area under the concentration-time curve (AUC) of SPR001 in patients with CAH	6 weeks
Secondary	PK/PD relationships	To explore the potential relationships between pharmacokinetics and pharmacodynamics	6 weeks

External Links

•   On-line clinical study-related information for patients, including pre-screening questionnaire.