A Phase 2 interventional study of Tildacerfont/Placebo in Congenital Adrenal Hyperplasia, sponsored by Spruce Biosciences. Terminated at 43 sites in 16 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-01.
Sponsored by Spruce Biosciences · Phase 2, Interventional, and Treatment
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic Congenital adrenal hyperplasia (CAH) subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
This is a study that evaluated the ability of tildacerfont to reduce the glucocorticoid steroid dose used by adult subjects with CAH. The first 24-weeks were a double-blind, placebo controlled, comparison of tildacerfont vs placebo. The following 52-weeks allowed all subjects to move to open label tildacerfont to continue to reduce steroid dose where appropriate, and observe long term safety. Subjects were offered a long term open label extension up to 240 weeks.
101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.
This study's enrollment of 100 is above 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:
Has had a unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to:
Psychiatric conditions, including but not limited to bipolar disorder, schizophrenia, or schizoaffective disorders that are not effectively controlled on medication and may have an adverse impact on study compliance. Symptoms including hallucinations, delusions, and psychosis are exclusionary. Additionally:
Increased risk of suicide based on the Investigator's judgment or the results of the C-SSRS conducted at screening and baseline (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months) b. Hospital Anxiety and Depression Scale (HADS) score >12 for either depression or anxiety at screening or baseline
Has clinically significant abnormal or clinical laboratory results. Abnormal results that must be reviewed and discussed with the Medical Monitor to determine eligibility for this study include but are not limited to:
Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before the start of the Treatment Period to the end of the study:
i. Moderate to strong inhibitors and/or inducers of ii. Sensitive substrates or narrow-therapeutic-range substrates of CYP3A4 (except hormonal containing ≤35 μg ethinyl estradiol) iii. Sensitive substrates or narrow-therapeutic-range substrates of BCRP (except those that can be administered QD in the morning, separated by approximately 10 hours from evening administration of study drug)
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.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
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.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
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.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks
Drug: Tildacerfont/Placebo
Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 weeks
Drug: Tildacerfont/Placebo
Tablet, administered daily
Also known as: SPR001
Change in Total Daily GC Dose in Subjects With Classic CAH Over the 24-week, Double Blind, Placebo-Controlled Treatment Period
Absolute change from baseline (Day 1) in GC dose in HCe at Week 24. The analysis of absolute change in total daily GC dose in HCe will include data from Weeks 3, 6, 12, 18 and 24 in a mixed model
Time frame: 24 Weeks
Effect of Tildacrfont in Reducing GC Use to Near-physiologic Levels While Maintaining Androgen Control in Subjects With CAH
Proportion of subjects with GC dose ≤11mg/m2/day in HCe and A4 ≤1.2x baseline or A4 ≤ ULN at Week 24
Time frame: 24 weeks
Effect of Tildacrfont in Reducing GC Use to Near-physiologic Levels While Maintaining Androgen Control in Subjects With CAH
Proportion of subjects with baseline GC dose ≤ 35mg HCe who achieve GC dose ≤11 mg/m2/day in HCe and A4 ≤ 1.2x baseline or ≤ ULN at Week 24
Time frame: 24 Weeks
Effectiveness in Reducing Cardiovascular Risk in Subjects With CAH
Proportion of subjects with improvement in at least one cardiovascular risk factor at Week 24
Time frame: 24 Weeks
This study randomized approximately 100 participants with classic congenital adrenal hyperplasia (CAH) currently receiving glucocorticoid (GC) at a supraphysiologic dose
| Milestone | Tildacerfont Group | Placebo |
|---|---|---|
| Started | 48 | 52 |
| Completed | 44 | 50 |
| Not completed | 4 | 2 |
| Withdrew: Withdrawal by subject | 1 | 2 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 |
| Withdrew: Protocol violation | 1 | 0 |
| Milestone | Tildacerfont Group | Placebo |
|---|---|---|
| Started | 42 | 50 |
| Completed | 20 | 24 |
| Not completed | 22 | 26 |
| Withdrew: Study terminated | 15 | 19 |
| Withdrew: Withdrawal by subject | 6 | 5 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Physician decision | 1 | 0 |
| Withdrew: Other | 0 | 1 |
Absolute change from baseline (Day 1) in GC dose in HCe at Week 24. The analysis of absolute change in total daily GC dose in HCe will include data from Weeks 3, 6, 12, 18 and 24 in a mixed model
| mg | Tildacerfont Group | Placebo |
|---|---|---|
| Change in Total Daily GC Dose in Subjects With Classic CAH Over the 24-week, Double Blind, Placebo-Controlled Treatment Period | -5.0 ± 8.91 | -4.3 ± 9.75 |
Proportion of subjects with GC dose ≤11mg/m2/day in HCe and A4 ≤1.2x baseline or A4 ≤ ULN at Week 24
| Participants | Tildacerfont Group | Placebo |
|---|---|---|
| Effect of Tildacrfont in Reducing GC Use to Near-physiologic Levels While Maintaining Androgen Control in Subjects With CAH | 4 | 5 |
Proportion of subjects with baseline GC dose ≤ 35mg HCe who achieve GC dose ≤11 mg/m2/day in HCe and A4 ≤ 1.2x baseline or ≤ ULN at Week 24
| Participants | Tildacerfont Group | Placebo |
|---|---|---|
| Effect of Tildacrfont in Reducing GC Use to Near-physiologic Levels While Maintaining Androgen Control in Subjects With CAH | 2 | 4 |
Proportion of subjects with improvement in at least one cardiovascular risk factor at Week 24
| Participants | Tildacerfont Group | Placebo |
|---|---|---|
| Effectiveness in Reducing Cardiovascular Risk in Subjects With CAH | 26 | 19 |
Collected over The study period was expected to be up to 240 weeks in duration (double-blind treatment [24 weeks], open-label treatment [52 weeks], and optional open-label extension [up to the 240th week]), but due to early study termination, the total study period was 205 weeks. No subject entered the optional open label extension. The final safety follow-up was assessed at 30 days after the last dose of study drug for each participant.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Tildacerfont Group | 0/48 (0%) | 0/48 (0%) | 11/48 (22.9%) |
| Placebo | 0/52 (0%) | 0/52 (0%) | 16/52 (30.8%) |
| Open Label Extension | 0/92 (0%) | 2/92 (2.2%) | 77/92 (83.7%) |
| Event | Tildacerfont Group | Placebo | Open Label Extension |
|---|---|---|---|
| adrenocortical insufficiency acuteEndocrine disorders | 0/48 | 0/52 | 1/92 |
| intervertebral disc protrusionMusculoskeletal and connective tissue disorders | 0/48 | 0/52 | 1/92 |
| Event | Tildacerfont Group | Placebo | Open Label Extension |
|---|---|---|---|
| NasopharyngitisInfections and infestations | 5/48 | 2/52 | 17/92 |
| FatigueGeneral disorders | 6/48 | 8/52 | 16/92 |
| HeadacheNervous system disorders | 7/48 | 9/52 | 9/92 |
| COVID-19Infections and infestations | 6/48 | 5/52 | 9/92 |
| VomitingGastrointestinal disorders | 3/48 | 6/52 | 11/92 |
| Upper respiratory tract infectionInfections and infestations | 3/48 | 4/52 | 10/92 |
| DiarrheaGastrointestinal disorders | 5/48 | 3/52 | 6/92 |
| GastroenteritisInfections and infestations | 1/48 | 0/52 | 9/92 |
| NauseaGastrointestinal disorders | 4/48 | 5/52 | 7/92 |
| Back painMusculoskeletal and connective tissue disorders | 2/48 | 4/52 | 5/92 |
| Age, Continuous(years) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Mean | 33.0 ± 11.52 | 32.0 ± 12.08 | 32.5 ± 11.77 |
| Sex: Female, Male(Participants) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Female | 25 | 28 | 53 |
| Male | 23 | 24 | 47 |
| Race/Ethnicity, Customized(Participants) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 7 | 6 | 13 |
| Not Hispanic or Latino | 41 | 44 | 85 |
| Unknown | 0 | 2 | 2 |
| Asian | 6 | 8 | 14 |
| Black or African American | 3 | 0 | 3 |
| White | 38 | 43 | 81 |
| Other | 1 | 1 | 2 |
| Females with Child-Bearing Potential(Participants) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Count of participants | 22 | 26 | 48 |
| Time Since Congenital adrenal hyperplasia (CAH) Diagnosis(years) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Mean | 31.2 ± 11.58 | 28.0 ± 12.99 | 29.5 ± 12.37 |
| Type of CAH Diagnosis(Participants) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Salt-wasting | 36 | 35 | 71 |
| Simple Virilizing | 12 | 17 | 29 |
| 17-hydroxyprogesterone (17-OHP)(ng/dL) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Mean | 5642.5 ± 7481.57 | 5656.6 ± 6995.03 | 5649.8 ± 7195.84 |
| Androstenedione (A4)(ng/dL) | Tildacerfont Group | Placebo | Total |
|---|---|---|---|
| Mean | 227.2 ± 247.10 | 222.2 ± 203.28 | 224.6 ± 224.24 |
10 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Feb 2021 to primary completion in Oct 2024, 44 months.
Results were first posted in Oct 2025, 11 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
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Adrenal Hyperplasia, Congenital →
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