A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children with CAH
- Registration Number
- NCT05128942
- Lead Sponsor
- Spruce Biosciences
- Brief Summary
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
- Detailed Description
This is a study to evaluate the safety and efficacy of tildacerfont in participants with Congenital Adrenal Hyperplasia (CAH). Treatment will consist of 4 or 12 weeks of continuous dosing followed by safety follow up.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 65
- Male and female subjects aged 2+
- Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
- Stable dose of GC replacement for at least 1 month prior to screening
- History of bilateral adrenalectomy or hypopituitarism
- Clinically significant unstable medical conditions, illness, or chronic diseases
- History of active bleeding disorders
- Females who are pregnant or nursing
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 9: Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 4 consecutive weeks. Cohort 2: Age 11-17 Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 12 consecutive weeks. Cohort 5: Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 4 consecutive weeks. Cohort 7: Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 4 consecutive weeks. Cohort 1: Age 11-17 Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 12 consecutive weeks. Cohort 3: Age 2-10 Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 12 consecutive weeks. Cohort 4: Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 4 consecutive weeks. Cohort 6: Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered twice daily for 4 consecutive weeks. Cohort 8: Treatment with Tildacerfont Tildacerfont Oral Tildacerfont administered daily for 4 consecutive weeks.
- Primary Outcome Measures
Name Time Method Number of participants with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 12 weeks To evaluate safety of tildacerfont in participants with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0
- Secondary Outcome Measures
Name Time Method Tildacerfont pharmacokinetics (PK) 4 weeks or 12 weeks To determine the consistency of preliminary tildacerfont PK in participants with those simulated in a PBPK model measured by comparing tildacerfont plasma concentrations with current PBPK simulation for consistency
Proportion of participants who achieve a reduction in androstenedione (A4) or reduction in glucocorticoid (GC) dosing 4 weeks or 12 weeks To determine the efficacy of tildacerfont on disease control or reduction of GC use in participants with classic CAH as measured by number of subjects who achieve a reduction in A4 or reduction in GC dosing during treatment period
Proportion of participants with elevated baseline A4 who achieve a reduction in A4 4 weeks To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4
Proportion of participants with elevated baseline A4 who achieve a reduction in A4 who achieve A4 normalization 4 weeks or 12 weeks To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4 or week 12
Trial Locations
- Locations (1)
Spruce Study Site
🇺🇸Richmond, Virginia, United States