Tildacerfont for Congenital Adrenal Hyperplasia

No longer recruiting at 83 trial locations

Overseen By Pamela Wedel

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Spruce Biosciences

Must be taking: Glucocorticoids, Mineralocorticoids

Disqualifiers: Other CAH, Adrenalectomy, Hypopituitarism, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if Tildacerfont can help individuals with classic Congenital Adrenal Hyperplasia (CAH) reduce their long-term need for high doses of steroids. Participants will take either Tildacerfont or a placebo (a pill with no active medication) for 24 weeks, followed by Tildacerfont for an additional 52 weeks. The trial seeks participants with a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency who have been on steady, high doses of glucocorticoids (a type of steroid medication) for at least a month. As a Phase 2 trial, this research measures Tildacerfont's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant medical advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it mentions that participants should have been on a stable dose of glucocorticoid and mineralocorticoid replacements for at least one month before screening. This suggests you may continue your current medications.

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

Research has shown that tildacerfont is generally safe for people with congenital adrenal hyperplasia (CAH). In one study, 10 out of 11 participants completed the trial, with only one stopping due to a side effect, suggesting that most handled the treatment well.

Another study supports the safety of tildacerfont, showing it can safely lower certain hormone levels in the body. These findings indicate that tildacerfont doesn't cause serious problems for most patients.

Since this trial is in phase 2, tildacerfont has already passed initial safety tests in humans. While more research is needed, the current data provides a positive outlook on its safety for those considering joining a trial. ^{1 2 3 4 5}

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

Unlike the standard treatments for congenital adrenal hyperplasia, which often involve corticosteroids, Tildacerfont acts on a different pathway by targeting the CRF1 receptor. This mechanism helps to reduce the overproduction of androgens without the side effects typically associated with steroid use. Researchers are excited about Tildacerfont because it offers the potential for effective management of the condition with fewer side effects, potentially improving quality of life for patients.

What evidence suggests that Tildacerfont might be an effective treatment for congenital adrenal hyperplasia?

Research has shown that tildacerfont, which participants in this trial may receive, may help people with Congenital Adrenal Hyperplasia (CAH) by balancing hormone levels. One study found that patients taking tildacerfont had lower levels of certain hormones, indicating better balance. Another study found that taking 200mg of tildacerfont daily reduced the need for glucocorticoids (a type of steroid hormone) compared to a placebo. This suggests that tildacerfont might help lower the need for high doses of glucocorticoids, often used to manage CAH symptoms. Overall, these findings suggest that tildacerfont could be an effective treatment option for managing CAH. ^{1 2 3 4 5}

Who Is on the Research Team?

Ron Newfield, M.D

Principal Investigator

Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.

Are You a Good Fit for This Trial?

This trial is for adults over 18 with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. It's not open to those with other forms of CAH, a history of bilateral adrenalectomy, or hypopituitarism.

Inclusion Criteria

I am over 18 years old.

Exclusion Criteria

I have a form of CAH that is not due to 21-hydroxylase deficiency.

You have had surgery to remove both adrenal glands or you have a condition called hypopituitarism.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Double-blind, placebo-controlled treatment with Tildacerfont or placebo for 24 weeks

24 weeks

Regular visits as per trial protocol

Open-label Treatment

All subjects receive open-label Tildacerfont for 52 weeks to continue reducing steroid dose and observe long-term safety

52 weeks

Regular visits as per trial protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label Extension (optional)

Participants may opt into continuation of treatment long-term for up to 240 weeks

Up to 240 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Tildacerfont

Trial Overview The study tests if Tildacerfont can reduce the need for high doses of glucocorticoids in CAH patients. Participants will receive either Tildacerfont or a placebo and may continue treatment up to 240 weeks.

How Is the Trial Designed?

Group I: Tildacerfont Group Experimental Treatment 1 Intervention

Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks

Group II: Placebo Placebo Group 1 Intervention

Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 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 ... Phase 2b and Phase 3 clinical trials of CRF1 antagonists involving Crinecerfont demonstrated consistent results supporting its efficacy and safety.

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 ...