A Phase 2 interventional study of SPR001 in Congenital Adrenal Hyperplasia and CAH - Congenital Adrenal Hyperplasia, sponsored by Spruce Biosciences. Completed at 9 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-22.
Sponsored by Spruce Biosciences · Phase 2, Interventional, and Treatment
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).
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.
101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.
This study's enrollment of 24 is below the median of 37 across 50 interventional studies indexed under Adrenal Hyperplasia, Congenital.
Browse Adrenal Hyperplasia, Congenital studies →Spruce Biosciences is the lead sponsor of 8 studies on the registry; 2 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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
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
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
Time frame: 6 weeks
Change in 17-hydroxyprogesterone
Change in 17-hydroxyprogesterone from Baseline to End-of-study. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.
Time frame: Cohort A: Baseline/2a (Day -1-0), First dose/2b (Day 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41-42). Cohort B and Cohort C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 14-15), Visit 5 (Last dose +30d)
Changes in Pharmacodynamic (PD) Markers
Changes in adrenocorticotropic hormone (ACTH) and androstenedione (A4) from Baseline to End-of-study are measured in patient serum. Results are expressed as a mean percentage change from baseline. A negative change indicates improvement.
Time frame: Cohort A: Visit 2a (Day -1 to 0), Visit 2b (Days 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41 to 42). Cohorts B+C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 27-28), Visit 5 (+30 days after last dose)
Pharmacokinetic Parameter - Maximum Plasma Concentration (Cmax)
To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH.
Time frame: Serial PK sampling was performed at the end of the 2 weeks for all cohorts and dose levels
Pharmacokinetic Parameter - 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
Time frame: For Cohort A, serial blood collections were made for PK measurements on Day 1 for 200 mg SD and at Week 2 for all QD dose levels. In Cohorts B and C, serial blood samples were drawn for PK measurements at the end of the 2-week treatment period.
No participants enrolled in Period 4: Cohort D
| Milestone | Period 1: Cohort A | Period 2: Cohort B | Period 3: Cohort C | Period 4: Cohort D |
|---|---|---|---|---|
| Started | 10 | 0 | 0 | 0 |
| Completed | 9 | 0 | 0 | 0 |
| Not completed | 1 | 0 | 0 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 | 0 | 0 |
| Milestone | Period 1: Cohort A | Period 2: Cohort B | Period 3: Cohort C | Period 4: Cohort D |
|---|---|---|---|---|
| Started | 0 | 9 | 0 | 0 |
| Completed | 0 | 8 | 0 | 0 |
| Not completed | 0 | 1 | 0 | 0 |
| Withdrew: Discontinued due to sponsor decision | 0 | 1 | 0 | 0 |
| Milestone | Period 1: Cohort A | Period 2: Cohort B | Period 3: Cohort C | Period 4: Cohort D |
|---|---|---|---|---|
| Started | 0 | 0 | 7 | 0 |
| Completed | 0 | 0 | 6 | 0 |
| Not completed | 0 | 0 | 1 | 0 |
Incidence of treatment-emergent adverse events, changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters
| Participants | Cohort A | Cohort B | Cohort C |
|---|---|---|---|
| Safety of SPR001 in Patients With CAH | 6 | 5 | 4 |
Change in 17-hydroxyprogesterone from Baseline to End-of-study. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.
| percentage of mean change from baseline | Cohort B | Cohort C | Cohort A - Dose A | Cohort A - Dose B | Cohort A - Dose C |
|---|---|---|---|---|---|
| Change in 17-hydroxyprogesterone | -47.79 ± 63.300 | -33.19 ± 41.604 | 22.16 ± 172.298 | -23.71 ± 64.146 | -13.79 ± 74.742 |
Changes in adrenocorticotropic hormone (ACTH) and androstenedione (A4) from Baseline to End-of-study are measured in patient serum. Results are expressed as a mean percentage change from baseline. A negative change indicates improvement.
| percentage of mean change from baseline | PD Population Cohort B | PD Population Cohort C | PD Population - Cohort A Dose A | PD Population Cohort A - Dose B | PD Population - Cohort A - Dose C |
|---|---|---|---|---|---|
| Adrenocorticotropic hormone (ACTH) | -26.05 ± 54.807 | 16.66 ± 120.211 | -28.53 ± 53.834 | -67.60 ± 32.100 | -36.83 ± 48.510 |
| Androstenedione (A4) | -1.58 ± 101.458 | -30.17 ± 35.055 | -12.12 ± 47.381 | -33.18 ± 36.465 | -28.93 ± 45.167 |
To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH.
| ng/dL | PK Population Cohort B | PK Population Cohort C | PK Population - Cohort A Dose A | PK Population Cohort A - Dose B | PK Population - Cohort A - Dose C | PK Population Cohort A - Dose A (After single dose) |
|---|---|---|---|---|---|---|
| Pharmacokinetic Parameter - Maximum Plasma Concentration (Cmax) | 411.25 ± 130.107 | 200.96 ± 132.262 | 283.69 ± 150.116 | 719.30 ± 325.056 | 892.56 ± 289.272 | 96.35 ± 86.1 |
To evaluate the PK parameter of area under the concentration-time curve (AUC) of SPR001 in patients with CAH
| ng*hr/dL | PK Population Cohort B | PK Population Cohort C | PK Population - Cohort A Dose A | PK Population Cohort A - Dose B | PK Population - Cohort A - Dose C | PK Population Cohort A - Dose A (After single dose) |
|---|---|---|---|---|---|---|
| Pharmacokinetic Parameter - Area Under the Concentration-time Curve (AUC) | 2975.968 ± 970.6 | 1425.582 ± 941.9188 | 1474.162 ± 642.6 | 4449.263 ± 1826.4801 | 6212.983 ± 2051.9077 | 421.539 ± 331.54 |
Collected over 6-week treatment period followed by a 4-week washout/safety follow-up period. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort A - Dose A | 0/10 (0%) | 0/10 (0%) | 6/10 (60%) |
| Cohort A - Dose B | 0/10 (0%) | 0/10 (0%) | 3/10 (30%) |
| Cohort A - Dose C | 0/9 (0%) | 0/10 (0%) | 3/9 (33.3%) |
| Cohort B | 0/9 (0%) | 0/9 (0%) | 5/9 (55.6%) |
| Cohort C | 0/7 (0%) | 0/7 (0%) | 4/7 (57.1%) |
| Event | Cohort A - Dose A | Cohort A - Dose B | Cohort A - Dose C | Cohort B | Cohort C |
|---|---|---|---|---|---|
| HeadacheNervous system disorders | 0/10 | 0/10 | 1/9 | 2/9 | 0/7 |
| Upper respiratory tract infectionInfections and infestations | 0/10 | 2/10 | 0/9 | 0/9 | 0/7 |
| DisorientationPsychiatric disorders | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
| DyspepsiaGastrointestinal disorders | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
| ChillsGeneral disorders | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
| FatigueGeneral disorders | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
| ContusionInjury, poisoning and procedural complications | 0/10 | 0/10 | 0/9 | 1/9 | 1/7 |
| Blood thyroid stimulating hormone increasedInvestigations | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/10 | 0/10 | 0/9 | 0/9 | 1/7 |
24 unique subjects participated in the study; however, two subjects were enrolled in more than one cohort, one subject in Cohorts A and C and one subject in Cohorts A and B. For the subjects enrolled in more than 1 cohort, cohort totals/statistics reflect all baseline values.
| Age, Categorical(Participants) | Cohort A | Cohort B | Cohort C | Cohort D | Total |
|---|---|---|---|---|---|
| Cohort A — <=18 years | 0 | 0 | 0 | 0 | 0 |
| Cohort A — Between 18 and 65 years | 9 | 0 | 0 | 0 | 9 |
| Cohort A — >=65 years | 1 | 0 | 0 | 0 | 1 |
| Cohort B — <=18 years | 0 | 0 | 0 | 0 | 0 |
| Cohort B — Between 18 and 65 years | 0 | 7 | 0 | 0 | 7 |
| Cohort B — >=65 years | 0 | 1 | 0 | 0 | 1 |
| Cohort C — <=18 years | 0 | 0 | 0 | 0 | 0 |
| Cohort C — Between 18 and 65 years | 0 | 0 | 5 | 0 | 5 |
| Cohort C — >=65 years | 0 | 0 | 1 | 0 | 1 |
| Sex: Female, Male(Participants) | Cohort A | Cohort B | Cohort C | Cohort D | Total |
|---|---|---|---|---|---|
| Cohort A — Female | 5 | 0 | 0 | 0 | 5 |
| Cohort A — Male | 5 | 0 | 0 | 0 | 5 |
| Cohort B — Female | 0 | 1 | 0 | 0 | 1 |
| Cohort B — Male | 0 | 7 | 0 | 0 | 7 |
| Cohort C — Female | 0 | 0 | 3 | 0 | 3 |
| Cohort C — Male | 0 | 0 | 3 | 0 | 3 |
| Ethnicity (NIH/OMB)(Participants) | Cohort A | Cohort B | Cohort C | Cohort D | Total |
|---|---|---|---|---|---|
| Cohort A — Hispanic or Latino | 3 | 0 | 0 | 0 | 3 |
| Cohort A — Not Hispanic or Latino | 7 | 0 | 0 | 0 | 7 |
| Cohort A — Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Cohort B — Hispanic or Latino | 0 | 2 | 0 | — | 2 |
| Cohort B — Not Hispanic or Latino | 0 | 6 | 0 | — | 6 |
| Cohort B — Unknown or Not Reported | 0 | 0 | 0 | — | 0 |
| Cohort C — Hispanic or Latino | 0 | 0 | 1 | — | 1 |
| Cohort C — Not Hispanic or Latino | 0 | 0 | 5 | — | 5 |
| Cohort C — Unknown or Not Reported | 0 | 0 | 0 | — | 0 |
| Race (NIH/OMB)(Participants) | Cohort A | Cohort B | Cohort C | Cohort D | Total |
|---|---|---|---|---|---|
| Cohort A — American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Cohort A — Asian | 0 | 0 | 0 | 0 | 0 |
| Cohort A — Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Cohort A — Black or African American | 0 | 0 | 0 | 0 | 0 |
| Cohort A — White | 10 | 0 | 0 | 0 | 10 |
| Cohort A — More than one race | 0 | 0 | 0 | 0 | 0 |
| Cohort A — Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Cohort B — American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Cohort B — Asian | 0 | 0 | 0 | 0 | 0 |
| Cohort B — Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Cohort B — Black or African American | 0 | 0 | 0 | 0 | 0 |
| Cohort B — White | 0 | 8 | 0 | 0 | 8 |
| Cohort B — More than one race | 0 | 0 | 0 | 0 | 0 |
| Cohort B — Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Cohort C — American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 |
| Cohort C — Asian | 0 | 0 | 0 | 0 | 0 |
| Cohort C — Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Cohort C — Black or African American | 0 | 0 | 0 | 0 | 0 |
| Cohort C — White | 0 | 0 | 5 | 0 | 5 |
| Cohort C — More than one race | 0 | 0 | 1 | 0 | 1 |
| Cohort C — Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment(participants) | Cohort A | Cohort B | Cohort C | Cohort D | Total |
|---|---|---|---|---|---|
| United States | 10 | 8 | 6 | — | 24 |
| Body Mass Index (kg/m^2)(kg/m^2) | Cohort A | Cohort B | Cohort C | Cohort D | Total |
|---|---|---|---|---|---|
| Mean | 31.7 ± 11.80 | 30.4 ± 6.31 | 32.3 ± 5.67 | — | 31.4 ± 8.44 |
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Adrenal Hyperplasia, Congenital →
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