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Not yet recruiting NCT07536269

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

Phase II Interventional Congenital Adrenal Hyperplasia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Crinecerfont.
Who it may be relevant to
Registry conditions: Congenital Adrenal Hyperplasia. Basic parameters: 3 months — 47 months · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia

Overview

The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to \<4 years of age with CAH.

Interventions

  • Drug Crinecerfont
    Oral solution

Primary outcome measures

  • Number of Participants With Treatment-emergent Adverse Events (TEAE) [Time frame: Day 1 up to 28 weeks]

Eligibility criteria

Inclusion criteria

  • Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD)
  • Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening.
  • Have a body weight of at least 4.5 kilograms (kg) at screening.
  • Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist.

Exclusion criteria

  • Have a known or suspected diagnosis of any of the other forms of classic CAH.
  • Have any condition besides CAH that requires chronic daily therapy with orally administered steroids.
  • Have any other clinically significant medical condition or chronic disease.

Note: Other protocol-defined inclusion and exclusion criteria may apply.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07536269 · NBI-74788-CAH2032

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗