Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Overview

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

Full Title of Study: “A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: March 10, 2023

Interventions

  • Drug: Crinecerfont
    • CRF1-receptor antagonist
  • Drug: Placebo
    • Non-active dosage form

Arms, Groups and Cohorts

  • Experimental: Crinecerfont
    • Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.
  • Placebo Comparator: Placebo
    • Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.

Clinical Trial Outcome Measures

Primary Measures

  • Change from Baseline in Serum Androstenedione (A4) at Week 4
    • Time Frame: Baseline to Week 4

Secondary Measures

  • Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
    • Time Frame: Baseline to Week 4
  • Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28
    • Time Frame: Baseline to Week 28
  • Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28
    • Time Frame: Baseline to Week 28
  • Change from baseline in body mass index at Week 28
    • Time Frame: Baseline to Week 28
  • Change from baseline in salivary 17-OHP at Week 28
    • Time Frame: Baseline to Week 28
  • Change in bone age advancement at Week 28
    • Time Frame: Baseline to Week 28
  • Change from baseline in predicted adult height at Week 52
    • Time Frame: Baseline to Week 52

Participating in This Clinical Trial

Inclusion Criteria

  • Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit. – Have a medically confirmed diagnosis of 21-hydroxylase deficiency CAH. – Be on a stable regimen of steroidal treatment for CAH. – Have elevated androgen levels. – Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study. Exclusion Criteria:

  • Have a diagnosis of any of the other forms of classic CAH. – Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy. – Have a clinically significant unstable medical condition or chronic disease other than CAH. – Have a history of cancer unless considered to be cured. – Have a known history of clinically significant arrhythmia or abnormalities on ECG. – Have a known hypersensitivity to any corticotropin-releasing hormone antagonist. – Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study. – Have current substance dependence or substance (drug) or alcohol abuse. – Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.

Gender Eligibility: All

Minimum Age: 2 Years

Maximum Age: 17 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Neurocrine Biosciences
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Clinical Development Lead, Study Director, Neurocrine Biosciences

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