Trial Outcomes & Findings for Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia (NCT NCT03687242)

NCT ID: NCT03687242

Last Updated: 2025-04-01

Results Overview

Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 11 participants
• Primary outcome timeframe: Over 12 weeks
• Results posted on: 2025-04-01

Participant Flow

Participant milestones

Measure	SPR001 SPR001 at Dose A SPR001: Open-label SPR001
Overall Study STARTED	11
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	SPR001 SPR001 at Dose A SPR001: Open-label SPR001
Overall Study Adverse Event	1

Baseline Characteristics

Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia

Baseline characteristics by cohort

Measure	SPR001 n=11 Participants SPR001 at Dose A SPR001: Open-label SPR001
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants
Body mass index (kg/m2)	33.6 kg/m^2 STANDARD_DEVIATION 11.31 • n=5 Participants

PRIMARY outcome

Timeframe: Over 12 weeks

Population: Participants who received at least one dose of study drug.

Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.

Outcome measures

Measure	SPR001 n=11 Participants SPR001 at Dose A SPR001: Open label SPR001
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH Subjects experiencing adverse events	9 Participants
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH Subjects who did not experience adverse events	2 Participants

SECONDARY outcome

Timeframe: Week 12

Population: One subject was withdrawn prematurely on Day 24 due to adverse events and is not included in the efficacy analyses.

Change from Baseline to Week 12 in 17-OHP following dosing of SPR001 in subjects with CAH. 17-OHP is measured in patient serum sample. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.

Outcome measures

Measure	SPR001 n=10 Participants SPR001 at Dose A SPR001: Open label SPR001
Change From Baseline in 17-hydroxyprogesterone (17-OHP)	-43.4 percentage of change from baseline Standard Deviation 47.83

SECONDARY outcome

Timeframe: Week 12

Population: One subject was withdrawn prematurely on Day 24 due to adverse events and is not included in the efficacy analyses.

Change from Baseline to Week 12 in androstenedione (A4) following dosing of SPR001 in subjects with CAH. A4 is measured in patient serum. Results are expressed as mean percent change from baseline. Reductions in A4 are indicators of better disease control.

Outcome measures

Measure	SPR001 n=10 Participants SPR001 at Dose A SPR001: Open label SPR001
Change From Baseline in Androstenedione (A4)	34.0 percentage of mean change from baseline Standard Deviation 238.20

SECONDARY outcome

Timeframe: Week 12

Population: One subject was withdrawn prematurely on Day 24 due to adverse events and is not included in the efficacy analyses.

Change from Baseline to Week 12 in adrenocorticotropic hormone (ACTH) following dosing of SPR001 in subjects with CAH. ACTH is measured in patient serum. Results are expressed as mean percent change from baseline. Reductions in ACTH are indicators of better disease control.

Outcome measures

Measure	SPR001 n=10 Participants SPR001 at Dose A SPR001: Open label SPR001
Change From Baseline in Adrenocorticotropic Hormone (ACTH)	-45.64 percentage of mean change from baseline Standard Deviation 6.244

Adverse Events

SPR001

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	SPR001 n=11 participants at risk SPR001 at Dose A SPR001: Open-label SPR001
Endocrine disorders Hypothyroidism	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Eye disorders Lacrimation increased	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Gastrointestinal disorders Abdominal pain upper	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Gastrointestinal disorders Diarrhoea	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Gastrointestinal disorders Nausea	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Gastrointestinal disorders Vomiting	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Infections and infestations Upper respiratory tract infection	18.2% 2/11 • Number of events 2 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Infections and infestations Nasopharyngitis	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Injury, poisoning and procedural complications Contusion	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Investigations Glycosylated haemoglobin increased	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Investigations Hepatic enzyme increased	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Musculoskeletal and connective tissue disorders Back pain	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Nervous system disorders Dysgeusia	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Nervous system disorders Headache	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Psychiatric disorders Insomnia	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Skin and subcutaneous tissue disorders Acne	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Skin and subcutaneous tissue disorders Pruritis	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks
Skin and subcutaneous tissue disorders Pruritis generalized	9.1% 1/11 • Number of events 1 • During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks

Additional Information

Spruce CMO, MD

Spruce BioSciences Inc.

Phone: 415-655-4169

Email: clinicaltrials@sprucebio.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place