CAH - 21-Hydroxylase Deficiency, CAH - Congenital Adrenal Hyperplasia, Congenital Adrenal Hyperplasia
Conditions
Brief summary
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
Detailed description
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects. To be eligible for this study, an individual must either have completed Study SPR001-201 or meet eligibility criteria for SPR001-naïve subjects. The expected duration of study participation for each subject is up to approximately 5 months. This includes a screening period of ≤30 days, a treatment period of 12 weeks, and a safety follow-up period of 30 days.
Interventions
DRUGSPR001
Open label SPR001
Sponsors
Spruce Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Is approved by the Sponsor's Medical Monitor * Is on a stable regimen of glucocorticoid replacement for ≥30 days before baseline that is expected to remain stable throughout the study * If screening for this study occurs \>3 months after the subject's final follow-up visit in Study SPR001-201, the subject will have serum 17-OHP measured at screening. * Agrees to follow contraception guidelines * Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol
Exclusion criteria
* Experienced a clinically significant AE considered at least possibly related to SPR001 in Study SPR001-201 * If screening for this study occurs \>3 months after the subject's final follow-up visit in Study SPR001-201, the subject will be screened for any clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening * Is at increased risk of suicide * Clinically significant depression or anxiety at screening or baseline * Clinically significant abnormal clinical or laboratory assessments must be discussed with the Medical Monitor to determine eligibility for this study. * Subjects who routinely work overnight shifts require Medical Monitor approval for enrollment * Females who are pregnant or lactating * Use of any other investigational drug within 30 days or 5 half-lives before screening * Use of prohibited concomitant medications (including rosiglitazone, testosterone, and strong inhibitors and/or inducers of CYP3A4) within 30 days or 5 half-lives of baseline. Medications metabolized by CYP3A4, 2C8, 2C9, or 2C19, especially those that are sensitive substrates or substrates with narrow therapeutic ranges should be discussed on a case-by-case basis with the Medical Monitor.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH | Over 12 weeks | Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in 17-hydroxyprogesterone (17-OHP) | Week 12 | 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. |
| Change From Baseline in Androstenedione (A4) | Week 12 | 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. |
| Change From Baseline in Adrenocorticotropic Hormone (ACTH) | Week 12 | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| SPR001 SPR001 at Dose A
SPR001: Open-label SPR001 | 11 |
| Total | 11 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
Baseline characteristics
| Characteristic | SPR001 |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants |
| Body mass index (kg/m2) | 33.6 kg/m^2 STANDARD_DEVIATION 11.31 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 9 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 10 Participants |
| Region of Enrollment United States | 11 participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 11 |
| other Total, other adverse events | 9 / 11 |
| serious Total, serious adverse events | 0 / 11 |
Outcome results
Primary
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
Time frame: Over 12 weeks
Population: Participants who received at least one dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| SPR001 | The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH | Subjects experiencing adverse events | 9 Participants |
| SPR001 | The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH | Subjects who did not experience adverse events | 2 Participants |
Secondary
Change From Baseline in 17-hydroxyprogesterone (17-OHP)
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.
Time frame: Week 12
Population: One subject was withdrawn prematurely on Day 24 due to adverse events and is not included in the efficacy analyses.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SPR001 | Change From Baseline in 17-hydroxyprogesterone (17-OHP) | -43.4 percentage of change from baseline | Standard Deviation 47.83 |
Secondary
Change From Baseline in Adrenocorticotropic Hormone (ACTH)
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.
Time frame: Week 12
Population: One subject was withdrawn prematurely on Day 24 due to adverse events and is not included in the efficacy analyses.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SPR001 | Change From Baseline in Adrenocorticotropic Hormone (ACTH) | -45.64 percentage of mean change from baseline | Standard Deviation 6.244 |
Secondary
Change From Baseline in Androstenedione (A4)
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.
Time frame: Week 12
Population: One subject was withdrawn prematurely on Day 24 due to adverse events and is not included in the efficacy analyses.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| SPR001 | Change From Baseline in Androstenedione (A4) | 34.0 percentage of mean change from baseline | Standard Deviation 238.2 |