BridgeMD
This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 70 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baseline.
EU CTIS ID: 2023-503770-21-00
What this study is testing
To evaluate the effect of tildacerfont in reducing androstenedione in subjects with congenital adrenal hyperplasia over 12 weeks.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1.Male and female subjects ≥ 18 years old at screening.
- 2.Has a known childhood diagnosis of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-hydroxyprogesterone and currently treated with hydrocortisone, hydrocortisone acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned glucocorticoids).
- 3. Has been on a stable, supraphysiologic dose of glucocorticoid replacement for ≥1 month before screening.
- 4. Has androstenedione >upper limit of normal at both screening and Week 4 if daily glucocorticoid dose <30 mg OR has androstenedione >2.5x upper limit of normal at both screening and Week 4.
- 5.For subjects with the salt-wasting form of congenital adrenal hyperplasia, the subject is 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.
You likely can't join if
- 1.Has a known or suspected diagnosis of any other known form of classic congenital adrenal hyperplasia (not due to 21-hydroxylase deficiency).
- 7.c.Current or history of liver disease (with the exception of Gilbert’s syndrome).
- 7.d.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.
- 7.e.Active hepatitis B, hepatitis C, or human immunodeficiency virus at screening.
- 7.f. Subjects who plan to undergo bariatric surgery during the study are excluded.
- 7.g.Any other condition that would impact subject safety or confound interpretation of study results.
See the full eligibility criteria
Who can join
- 1.Male and female subjects ≥ 18 years old at screening.
- 2.Has a known childhood diagnosis of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-hydroxyprogesterone and currently treated with hydrocortisone, hydrocortisone acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned glucocorticoids).
- 3. Has been on a stable, supraphysiologic dose of glucocorticoid replacement for ≥1 month before screening.
- 4. Has androstenedione >upper limit of normal at both screening and Week 4 if daily glucocorticoid dose <30 mg OR has androstenedione >2.5x upper limit of normal at both screening and Week 4.
- 5.For subjects with the salt-wasting form of congenital adrenal hyperplasia, the subject is 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.
What rules you out
- 1.Has a known or suspected diagnosis of any other known form of classic congenital adrenal hyperplasia (not due to 21-hydroxylase deficiency).
- 7.c.Current or history of liver disease (with the exception of Gilbert’s syndrome).
- 7.d.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.
- 7.e.Active hepatitis B, hepatitis C, or human immunodeficiency virus at screening.
- 7.f. Subjects who plan to undergo bariatric surgery during the study are excluded.
- 7.g.Any other condition that would impact subject safety or confound interpretation of study results.
- 8.Psychiatric conditions, including but not limited to depression, bipolar disorder, schizophrenia or schizoaffective disorder that are not effectively controlled on medication and may have an adverse impact on study compliance. Symptoms including hallucinations, delusions and psychosis are exlcusionary.
- 8.a.Increased risk of suicide based on the Investigator's judgment or the results of the Columbia–Suicide Severity Rating Scale (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)
- 8.b.Hospital Anxiety and Depression Scale score >12 for either depression or anxiety at screening or Week 6.
- 9. Has clinically significant abnormal ECG or clinical laboratory results.
- 10. Routinely works overnight shifts.
- 2.Has a history that includes bilateral adrenalectomy or hypopituitarism.
- 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:
- 14.a.rosiglitazone, aromatase inhibitors, testosterone, or growth hormones or any other medication or supplement that could impact subject safety or confound interpretation of study results.
- 14.b.drugs listed in the study protocol.
- 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.
- 3.Has a history of allergy or hypersensitivity to tildacerfont, or any of its excipients or any other CRF1 receptor antagonist.
- 4.Current treatment with dexamethasone as glucocorticoid therapy for congenital adrenal hyperplasia.
- 5.Is not adherent to glucocorticoid 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:
- 7.a. An ongoing malignancy or <3 years of remission history from any malignancy, other than successfully treated localized skin cancer.
- 7.b. Estimated glomerular filtration rate of <45 mL/min/1.73 m2.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- New Zealand
- Brazil
- United Kingdom
- Mexico
- Korea, Republic of
- Switzerland
- Australia
- Canada
- Turkey
- Argentina
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; New Zealand; Brazil; United Kingdom; Mexico; Korea, Republic of and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.