A Phase 2 interventional study of SPR001 in Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia and CAH - 21-Hydroxylase Deficiency, sponsored by Spruce Biosciences. Completed at 8 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-04-01.
Sponsored by Spruce Biosciences · Phase 2, Interventional, and Treatment
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.
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.
101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.
This study's enrollment of 11 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.
Counted across the registry records on this site, refreshed daily.
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.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
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.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
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.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
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.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
SPR001 at Dose A
Drug: SPR001
Open label SPR001
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
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
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
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
| Milestone | SPR001 |
|---|---|
| Started | 11 |
| Completed | 10 |
| Not completed | 1 |
| Withdrew: Adverse event | 1 |
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
| Participants | SPR001 |
|---|---|
| Subjects experiencing adverse events | 9 |
| Subjects who did not experience adverse events | 2 |
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.
| percentage of change from baseline | SPR001 |
|---|---|
| Change From Baseline in 17-hydroxyprogesterone (17-OHP) | -43.4 ± 47.83 |
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.
| percentage of mean change from baseline | SPR001 |
|---|---|
| Change From Baseline in Androstenedione (A4) | 34.0 ± 238.20 |
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.
| percentage of mean change from baseline | SPR001 |
|---|---|
| Change From Baseline in Adrenocorticotropic Hormone (ACTH) | -45.64 ± 6.244 |
Collected over During 12-week treatment period and the 30 days follow-up period, for a total of 16 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| SPR001 | 0/11 (0%) | 0/11 (0%) | 9/11 (81.8%) |
| Event | SPR001 |
|---|---|
| Upper respiratory tract infectionInfections and infestations | 2/11 |
| HypothyroidismEndocrine disorders | 1/11 |
| Lacrimation increasedEye disorders | 1/11 |
| Abdominal pain upperGastrointestinal disorders | 1/11 |
| DiarrhoeaGastrointestinal disorders | 1/11 |
| NauseaGastrointestinal disorders | 1/11 |
| VomitingGastrointestinal disorders | 1/11 |
| NasopharyngitisInfections and infestations | 1/11 |
| ContusionInjury, poisoning and procedural complications | 1/11 |
| Glycosylated haemoglobin increasedInvestigations | 1/11 |
All enrolled subjects who received at least one dose of study drug. This population is the primary analysis set for all efficacy and safety analyses.
| Age, Categorical(Participants) | SPR001 |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 10 |
| >=65 years | 1 |
| Sex: Female, Male(Participants) | SPR001 |
|---|---|
| Female | 7 |
| Male | 4 |
| Ethnicity (NIH/OMB)(Participants) | SPR001 |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 9 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | SPR001 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 10 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | SPR001 |
|---|---|
| United States | 11 |
| Body mass index (kg/m2)(kg/m^2) | SPR001 |
|---|---|
| Mean | 33.6 ± 11.31 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Sep 2018 to primary completion in Jul 2019, 10 months.
Results were first posted in Apr 2025, 5 years 8 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Tildacerfont
Adrenal Hyperplasia, Congenital →
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