Tildacerfont for Congenital Adrenal Hyperplasia

No longer recruiting at 17 trial locations

Overseen By Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Spruce Biosciences

Must be taking: Gc replacement

Disqualifiers: Adrenalectomy, Hypopituitarism, Bleeding disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 4 or 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called tildacerfont to determine its safety and effectiveness for individuals with congenital adrenal hyperplasia (CAH), a condition affecting hormone production. Participants will take tildacerfont either once or twice daily for a set period, depending on their group. Individuals diagnosed with CAH due to 21-hydroxylase deficiency and on a stable dose of glucocorticoid (a type of hormone therapy) for at least a month might be suitable for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires that you have been on a stable dose of glucocorticoid (GC) replacement for at least one month before joining, so you will need to continue taking your current GC medication.

Is there any evidence suggesting that tildacerfont is likely to be safe for humans?

Research has shown that tildacerfont has been tested in people with Congenital Adrenal Hyperplasia (CAH) to assess its safety. In earlier studies, a safety review team examined the data, and tildacerfont appeared well-tolerated. Some side effects were reported, but they were usually mild, causing discomfort without serious harm.

In these studies, adults received tildacerfont, and the results indicated it did not cause serious health problems. This suggests the drug might be safe for people. However, as this is a Phase 2 trial, researchers continue to gather more information to fully understand its safety. Phase 2 trials focus on ensuring a treatment is safe and evaluating its effectiveness.

Overall, tildacerfont has shown potential to be safe for people with CAH, but ongoing research will continue to confirm these findings. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CAH?

Tildacerfont is unique because it targets the CRF1 receptor, which plays a role in the body's stress response, unlike the standard treatments for Congenital Adrenal Hyperplasia (CAH) that focus on replacing deficient hormones with glucocorticoids and mineralocorticoids. This targeted mechanism could lead to better management of the condition with potentially fewer side effects. Researchers are excited about Tildacerfont because it offers a new approach to managing CAH, promising improved quality of life for patients who struggle with the limitations of current therapies.

What evidence suggests that tildacerfont might be an effective treatment for CAH?

Research has shown that tildacerfont can help manage hormone levels in people with Congenital Adrenal Hyperplasia (CAH). One study found that patients who took tildacerfont for up to 12 weeks experienced reduced or normalized important hormone levels. This trial will test tildacerfont in various cohorts, with some participants receiving the treatment for 4 weeks and others for 12 weeks. This approach might help control CAH symptoms by balancing hormones. Additionally, evidence suggests that tildacerfont might allow for a lower dose of daily glucocorticoids (a type of steroid used to manage CAH), potentially reducing side effects. Overall, early findings are promising for those considering this treatment. ^{1 2 3 5 6}

Who Is on the Research Team?

Will Charlton, MD

Principal Investigator

Spruce Biosciences

Are You a Good Fit for This Trial?

This trial is for children aged 2 to 17 with Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. They must have been on a stable dose of glucocorticoid replacement for at least one month. It's not suitable for those without this specific CAH type, who've had both adrenal glands removed, have unstable medical conditions, are pregnant or nursing females, or have bleeding disorders.

Inclusion Criteria

I have been on a stable dose of glucocorticoid replacement for at least 1 month.

I am between 2 and 17 years old.

I have CAH and need ongoing hormone therapy since my diagnosis.

Exclusion Criteria

I do not have any unstable or serious long-term health conditions.

I have had surgery to remove both adrenal glands or have a condition affecting my pituitary gland.

My adrenal hyperplasia is not caused by 21-hydroxylase deficiency.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral Tildacerfont daily for 4 or 12 consecutive weeks

4 or 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tildacerfont

Trial Overview The study tests the safety and effectiveness of Tildacerfont in treating pediatric patients with CAH. This medication could potentially help manage hormone levels in these young patients.

How Is the Trial Designed?

Group I: Cohort 9: Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 4 consecutive weeks.

Group II: Cohort 8: Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 4 consecutive weeks.

Group III: Cohort 7: Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 4 consecutive weeks.

Group IV: Cohort 6: Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered twice daily for 4 consecutive weeks.

Group V: Cohort 5: Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 4 consecutive weeks.

Group VI: Cohort 4: Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 4 consecutive weeks.

Group VII: Cohort 3: Age 2-10 Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 12 consecutive weeks.

Group VIII: Cohort 2: Age 11-17 Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 12 consecutive weeks.

Group IX: Cohort 1: Age 11-17 Treatment with Tildacerfont Experimental Treatment 1 Intervention

Oral Tildacerfont administered daily for 12 consecutive weeks.

Who Is Running the Clinical Trial?

Spruce Biosciences

Lead Sponsor

Trials

6

Recruited

330+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8530725/

Tildacerfont in Adults With Classic Congenital Adrenal ... For patients with 21OHD, up to 12 weeks of oral tildacerfont reduced or maintained key hormone biomarkers toward normal.

2. investors.sprucebio.com investors.sprucebio.com/news-releases/news-release-details/spruce-biosciences-announces-topline-results-cahmelia-204-adult

News Release Details 200mg QD of tildacerfont demonstrated a placebo-adjusted reduction from baseline in daily GC dose of 0.7mg HCe (95% CI: -4.3 to 2.9, p=0.7).

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05128942

NCT05128942 | A Phase 2 Study to Evaluate the Safety ... This is a study to evaluate the safety and efficacy of tildacerfont in participants with Congenital Adrenal Hyperplasia (CAH).

4. sciencedirect.com sciencedirect.com/science/article/pii/S2666396125000330

Review CRF1 receptor antagonists in congenital adrenal ... Results. From Phase 2 clinical trials, pooled efficacy data from studies with CRF1 antagonists resulted in a mean decrease from baseline levels ...

5. hopkinsmedicine.org hopkinsmedicine.org/surgery/specialty-areas/adrenal-center/cah-203

Congenital Adrenal Hyperplasia (CAH) Research Study The purpose of this study is to see if an investigational drug, tildacerfont (SPR001) is safe and effective in reducing the level of certain hormones in the ...

6. investors.sprucebio.com investors.sprucebio.com/node/8316/pdf

Spruce Biosciences Reports Third Quarter 2024 Financial ... At the same time, we will also report topline dose-ranging efficacy and safety data from the. CAHptain-205 study of tildacerfont in adult and ...