Congenital Adrenal Hyperplasia
Conditions
Keywords
CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia
Brief summary
An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.
Detailed description
This is a study that will test the efficacy and safety of Tildacerfont. The first 12-weeks will be a double-blind, placebo controlled, dose ranging study. The following 58-weeks will assess the long term safety of Tildacerfont. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.
Interventions
Tablet, administered daily
Sponsors
Spruce Biosciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Double-Blind
Intervention model description
Subjects will be randomized in a 1:1:1:1 manner to one of three doses of Tildacerfont or Placebo.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male and female subjects ≥18 years old at screening 2. Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs) 3. Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥15 mg/day and ≤60 mg/day in HCe) for ≥1 month before screening 4. Has A4 \>ULN at both screening and Week 4 (measured before any AM GC dose) if daily GC dose \<30 mg OR has A4 \>2.5x ULN at both screening and Week 4 (measured before any AM GC dose) 5. For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening 6. Agrees to follow contraception guidelines. Male subjects must also agree to refrain from donating sperm throughout the treatment period and for 90 days after the last dose of study drug. 7. 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
1. Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21 hydroxylase deficiency) 2. Has a history that includes bilateral adrenalectomy or hypopituitarism 3. Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist 4. Current treatment with dexamethasone as GC therapy for CAH a. Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (eg, HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥1 month before screening. 5. Is not adherent to GC or study drug dosing regimen during the Run-in Period (defined as taking \<80% of expected doses based on drug accountability) 6. Shows clinical signs or symptoms of adrenal insufficiency 7. Has had a clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to: 1. An ongoing malignancy or \<3 years of remission history from any malignancy, other than successfully treated localized skin cancer 2. eGFR of \<45 mL/min/1.73 m2 3. Current or history of liver disease (with the exception of Gilbert's syndrome). 4. History of alcohol or substance abuse within the last year, or any significant history of alcohol or substance abuse that would likely prevent the subject from reliably participating in the study, based on the opinion of the Investigator 5. Active hepatitis B, hepatitis C, or HIV at screening 6. Subjects who plan to undergo bariatric surgery during the study are excluded. 7. Any other condition that would impact subject safety or confound interpretation of study results 8. 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: 1. Increased risk of suicide based on the Investigator's judgment or the results of the C-SSRS conducted at screening and Week 6 (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months) 2. HADS score \>12 for either depression or anxiety at screening or Week 6 9. Has clinically significant abnormal ECG 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: 1. Any clinically meaningful abnormal ECG results, including QTcF \>450 ms for male participants or \>470 ms for female participants 2. ALT \>2x ULN 3. Total bilirubin \>1.5x ULN 4. Total bile acids \>5x ULN 10. Routinely works overnight shifts 11. Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (\>2 hours) will require Medical Monitor approval for enrollment. 12. Females who are pregnant or nursing 13. Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study 14. Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before Day 1 to the end of the study: 1. Rosiglitazone, aromatase inhibitors, testosterone, growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results 2. The following drugs: i. Moderate to strong inhibitors and/or inducers of CYP3A4 ii. Sensitive substrates or narrow-therapeutic-range substrates of CYP3A4 (except hormonal contraception 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) 15. Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks | Baseline and 12 weeks of treatment (Week 18) | Assessment of dose response for change from baseline in log A4 after 12 weeks on double-blind placebo-controlled treatment (Week 18) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | 12 weeks | Absolute change from baseline in A4 as determined via application of the delta theorem to the log scale analysis results after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean Treatment Effect of Dose vs Placebo. A negative value represents a reduction in absolute concentration compared to baseline. |
| To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 12 weeks | Change from baseline in 17-OHP as assessed on the log scale after 12 weeks on double-blind, placebo-controlled treatment (Week 18). A negative value represents a % reduction from baseline. |
Countries
Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Latvia, Lithuania, Netherlands, Poland, Romania, South Korea, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom, United States
Participant flow
Recruitment details
96 participants participated in Part A , 86 of those participants continued into Part B, and 57 of these participants then entered Part C
Pre-assignment details
96 informed consents were signed when participants enrolled in the Placebo or Part A of the study. The ICF included language for all Parts of the study.
Participants by arm
| Arm | Count |
|---|---|
| Placebo tablet | 24 |
| Tildacerfont 50 mg 50 mg | 24 |
| Tildacerfont 100 mg 100 mg | 24 |
| Tildacerfont 200 mg 200 mg | 24 |
| Total | 96 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 | FG006 | FG007 |
|---|---|---|---|---|---|---|---|---|---|
| Period 2: Part A | Adverse Event | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 |
| Period 2: Part A | Lost to Follow-up | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
| Period 2: Part A | Other | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
| Period 2: Part A | Physician Decision | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Period 2: Part A | Protocol Violation | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
| Period 2: Part A | Study Terminated | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 |
| Period 2: Part A | Withdrawal by Subject | 0 | 1 | 1 | 1 | 0 | 0 | 0 | 0 |
| Period 3: Part B | Study Terminated | 0 | 0 | 0 | 0 | 1 | 10 | 13 | 0 |
| Period 3: Part B | Withdrawal by Subject | 0 | 0 | 0 | 0 | 0 | 1 | 2 | 0 |
| Period 4: Part C | Adverse Event | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 2 |
| Period 4: Part C | Other | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 2 |
| Period 4: Part C | Site Terminated | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 |
| Period 4: Part C | Study Terminated | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 30 |
| Period 4: Part C | Withdrawal by Subject | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 3 |
Baseline characteristics
| Characteristic | Total | Placebo | Tildacerfont 50 mg | Tildacerfont 100 mg | Tildacerfont 200 mg |
|---|---|---|---|---|---|
| Age, Continuous | 32.3 years STANDARD_DEVIATION 10.81 | 33.5 years STANDARD_DEVIATION 12.03 | 29.1 years STANDARD_DEVIATION 11.55 | 31.5 years STANDARD_DEVIATION 8.29 | 35.1 years STANDARD_DEVIATION 11.36 |
| BMI | 30.99 kg/m^2 STANDARD_DEVIATION 7.23 | 31.46 kg/m^2 STANDARD_DEVIATION 9.29 | 31.27 kg/m^2 STANDARD_DEVIATION 7.675 | 30.98 kg/m^2 STANDARD_DEVIATION 7.521 | 30.25 kg/m^2 STANDARD_DEVIATION 4.453 |
| Cortisol | 3.018 ug/dL STANDARD_DEVIATION 3.6998 | 2.63 ug/dL STANDARD_DEVIATION 3.492 | 7.08 ug/dL STANDARD_DEVIATION 9.382 | 1.29 ug/dL STANDARD_DEVIATION 0.854 | 1.07 ug/dL STANDARD_DEVIATION 1.071 |
| Daily dose of Glucocorticoid | 27.025 mg in hydrocortisone equivalent(s) STANDARD_DEVIATION 8.518 | 26.9 mg in hydrocortisone equivalent(s) STANDARD_DEVIATION 7.59 | 29.1 mg in hydrocortisone equivalent(s) STANDARD_DEVIATION 10.83 | 25.4 mg in hydrocortisone equivalent(s) STANDARD_DEVIATION 6.71 | 26.7 mg in hydrocortisone equivalent(s) STANDARD_DEVIATION 8.94 |
| Diastolic blood pressure | 77.225 mmHg STANDARD_DEVIATION 8.715 | 78.1 mmHg STANDARD_DEVIATION 9.15 | 76.4 mmHg STANDARD_DEVIATION 7.03 | 77.6 mmHg STANDARD_DEVIATION 8.41 | 76.8 mmHg STANDARD_DEVIATION 10.27 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 1 Participants | 0 Participants | 0 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 92 Participants | 23 Participants | 24 Participants | 24 Participants | 21 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Fasting glucose | 90.6 mg/dL STANDARD_DEVIATION 9.975 | 89.1 mg/dL STANDARD_DEVIATION 11.94 | 91.1 mg/dL STANDARD_DEVIATION 9.57 | 91.4 mg/dL STANDARD_DEVIATION 8.36 | 90.8 mg/dL STANDARD_DEVIATION 10.03 |
| Fasting insulin | 14.815 uIU/mL STANDARD_DEVIATION 10.57 | 10.32 uIU/mL STANDARD_DEVIATION 5.491 | 16.72 uIU/mL STANDARD_DEVIATION 9.896 | 15.77 uIU/mL STANDARD_DEVIATION 15.89 | 16.45 uIU/mL STANDARD_DEVIATION 11.004 |
| Glucocorticoid BSA Based Dose | 14.08 mg in hydrocortisone equivalent(s)/m^2 STANDARD_DEVIATION 6.424 | 13.97 mg in hydrocortisone equivalent(s)/m^2 STANDARD_DEVIATION 4.198 | 14.62 mg in hydrocortisone equivalent(s)/m^2 STANDARD_DEVIATION 4.88 | 13.52 mg in hydrocortisone equivalent(s)/m^2 STANDARD_DEVIATION 4.117 | 14.21 mg in hydrocortisone equivalent(s)/m^2 STANDARD_DEVIATION 12.5 |
| HbA1c | 5.23 % of Hemoglobin STANDARD_DEVIATION 0.408 | 5.18 % of Hemoglobin STANDARD_DEVIATION 0.215 | 5.14 % of Hemoglobin STANDARD_DEVIATION 0.255 | 5.33 % of Hemoglobin STANDARD_DEVIATION 0.944 | 5.27 % of Hemoglobin STANDARD_DEVIATION 0.218 |
| HDL | 49.85 mg/dL STANDARD_DEVIATION 15.463 | 56.7 mg/dL STANDARD_DEVIATION 18.85 | 44.0 mg/dL STANDARD_DEVIATION 16.34 | 49.4 mg/dL STANDARD_DEVIATION 11.14 | 49.3 mg/dL STANDARD_DEVIATION 15.52 |
| Homeostatic model assessment of insulin resistance (HOMA-IR) | 3.405 units on a scale STANDARD_DEVIATION 2.639 | 2.33 units on a scale STANDARD_DEVIATION 1.376 | 3.84 units on a scale STANDARD_DEVIATION 2.507 | 3.66 units on a scale STANDARD_DEVIATION 3.9 | 3.79 units on a scale STANDARD_DEVIATION 2.774 |
| LDL | 92.725 mg/dL STANDARD_DEVIATION 28.888 | 91.2 mg/dL STANDARD_DEVIATION 29.91 | 88.3 mg/dL STANDARD_DEVIATION 32.22 | 85.8 mg/dL STANDARD_DEVIATION 21.75 | 105.6 mg/dL STANDARD_DEVIATION 31.67 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 10 Participants | 3 Participants | 2 Participants | 1 Participants | 4 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) White | 84 Participants | 21 Participants | 22 Participants | 22 Participants | 19 Participants |
| Region of Enrollment Europe | 42 Participants | 9 Participants | 11 Participants | 16 Participants | 6 Participants |
| Region of Enrollment North America | 34 Participants | 9 Participants | 10 Participants | 6 Participants | 9 Participants |
| Region of Enrollment Rest of World | 20 Participants | 6 Participants | 3 Participants | 2 Participants | 9 Participants |
| Sex: Female, Male Female | 51 Participants | 12 Participants | 11 Participants | 12 Participants | 16 Participants |
| Sex: Female, Male Male | 45 Participants | 12 Participants | 13 Participants | 12 Participants | 8 Participants |
| Systolic blood pressure | 117.8 mmHg STANDARD_DEVIATION 10.946 | 118.3 mmHg STANDARD_DEVIATION 10.08 | 117.5 mmHg STANDARD_DEVIATION 9.54 | 115.0 mmHg STANDARD_DEVIATION 10.46 | 120.4 mmHg STANDARD_DEVIATION 13.71 |
| Total cholesterol | 164.85 mg/dL STANDARD_DEVIATION 38.4 | 169.3 mg/dL STANDARD_DEVIATION 41.62 | 158.6 mg/dL STANDARD_DEVIATION 42.08 | 152.2 mg/dL STANDARD_DEVIATION 29.15 | 179.3 mg/dL STANDARD_DEVIATION 40.75 |
| Triglycerides | 114.2 ng/dL STANDARD_DEVIATION 65.555 | 107.4 ng/dL STANDARD_DEVIATION 52.48 | 131.3 ng/dL STANDARD_DEVIATION 85.37 | 85.1 ng/dL STANDARD_DEVIATION 33.35 | 133.0 ng/dL STANDARD_DEVIATION 91.02 |
| Waist Circumference | 97.968 cm STANDARD_DEVIATION 16.941 | 95.8 cm STANDARD_DEVIATION 17.074 | 98.78 cm STANDARD_DEVIATION 19.135 | 97.98 cm STANDARD_DEVIATION 19.148 | 99.31 cm STANDARD_DEVIATION 12.407 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk |
|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 24 | 0 / 24 | 0 / 24 | 0 / 24 | 0 / 1 | 0 / 44 | 0 / 41 | 0 / 57 |
| other Total, other adverse events | 17 / 24 | 16 / 24 | 20 / 24 | 16 / 24 | 0 / 1 | 26 / 44 | 17 / 41 | 38 / 57 |
| serious Total, serious adverse events | 0 / 24 | 1 / 24 | 1 / 24 | 0 / 24 | 0 / 1 | 2 / 44 | 0 / 41 | 1 / 57 |
Outcome results
Primary
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks
Assessment of dose response for change from baseline in log A4 after 12 weeks on double-blind placebo-controlled treatment (Week 18)
Time frame: Baseline and 12 weeks of treatment (Week 18)
Population: The ITT Analysis Set included all randomized participants regardless of Treatment Period eligibility or completion. The ITT Analysis Set was the basis for demographics, baseline characteristics, and efficacy analyses.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks | 38.7 log(ng/dL) | Standard Deviation 307.38 |
| Tildacerfont 50 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks | -49.0 log(ng/dL) | Standard Deviation 449.02 |
| Tildacerfont 100 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks | 147.2 log(ng/dL) | Standard Deviation 661.2 |
| Tildacerfont 200 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks | -10.4 log(ng/dL) | Standard Deviation 408.68 |
Secondary
To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks
Change from baseline in 17-OHP as assessed on the log scale after 12 weeks on double-blind, placebo-controlled treatment (Week 18). A negative value represents a % reduction from baseline.
Time frame: 12 weeks
Population: The ITT Analysis Set included all randomized participants regardless of Treatment Period eligibility or completion. The ITT Analysis Set was the basis for demographics, baseline characteristics, and efficacy analyses.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 215.6 percent change | Standard Deviation 6165.61 |
| Tildacerfont 50 mg | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 644.2 percent change | Standard Deviation 6580.01 |
| Tildacerfont 100 mg | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 3564.6 percent change | Standard Deviation 7490.8 |
| Tildacerfont 200 mg | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | -753.7 percent change | Standard Deviation 5698.97 |
p-value: 0.8051Mixed Models Analysis
Secondary
To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks
Absolute change from baseline in 17-OHP as determined via application of the delta theorem to the log scale analysis results after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean Treatment Effect of Dose vs Placebo. A negative value represents a reduction in absolute concentration compared to baseline.
Time frame: 12 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 0 ng/dL | Standard Error 0 |
| Tildacerfont 50 mg | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 355.9 ng/dL | Standard Error 2179.63 |
| Tildacerfont 100 mg | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | 22.6 ng/dL | Standard Error 2120.47 |
| Tildacerfont 200 mg | To Evaluate the Effect of Tildacerfont in Reducing 17-OHP in Participants With Classic CAH Over 12 Weeks | -250.8 ng/dL | Standard Error 2153.59 |
Secondary
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks
Absolute change from baseline in A4 as determined via application of the delta theorem to the log scale analysis results after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean Treatment Effect of Dose vs Placebo. A negative value represents a reduction in absolute concentration compared to baseline.
Time frame: 12 weeks
Population: The ITT Analysis Set included all randomized participants regardless of Treatment Period eligibility or completion. The ITT Analysis Set was the basis for demographics, baseline characteristics, and efficacy analyses.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | 0 ng/dL | Standard Error 0 |
| Tildacerfont 50 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | -29.5 ng/dL | Standard Error 106.26 |
| Tildacerfont 100 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | 39.1 ng/dL | Standard Error 108.66 |
| Tildacerfont 200 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | -23.2 ng/dL | Standard Error 107.72 |
Secondary
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks
Change from baseline in A4 as assessed on the log scale after 12 weeks on double-blind, placebo-controlled treatment (Week 18). Results show mean % of Treatment Effect of Dose vs Placebo. A negative value represents a % reduction from baseline.
Time frame: 12 weeks
Population: The ITT Analysis Set included all randomized participants regardless of Treatment Period eligibility or completion. The ITT Analysis Set was the basis for demographics, baseline characteristics, and efficacy analyses.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Placebo | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | 0 percentage |
| Tildacerfont 50 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | -3.3 percentage |
| Tildacerfont 100 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | 4.3 percentage |
| Tildacerfont 200 mg | To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic CAH Over 12 Weeks | -2.6 percentage |