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

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3-47
SponsorNeurocrine Switzerland GmbH

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Crinecerfont

    Drug

    Oral solution

Treatment groups

20 Participants
are divided into 1 treatment group
Group A: CrinecerfontExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants With Treatment-emergent Adverse Events (TEAE)

Time frame
Day 1 up to 28 weeks

Secondary outcomes

Other outcomes

Sponsors and contacts

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