A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
E
Erik Imel, MD
Primary Investigator
Administratively Closed
18 years - 100 years
All
Phase
N/A
5 participants needed
2 Locations
Brief description of study
The purposes of this research study are:
• To study the efficacy of crinecerfont, compared with placebo, in reducing daily glucocorticoid dose and adrenal-related hormone levels, as well as other clinical factors related to glucocorticoid use (such as body weight and insulin resistance) in adults with CAH.
• To learn what happens to the crinecerfont in your body after dosing.
• To study the safety of crinecerfont in adults with CAH.
• To study different doses of crinecerfont in participants who have not reduced their glucocorticoid dose to a certain level by Month 12.
Detailed description of study
The Screening period will consist of testing to determine eligibility for the study. If participants qualify for the study starting on Day 1, they will take a 100 mg dose (2 capsules) of crinecerfont or placebo twice a day for 24 weeks with breakfast and evening meals. After the Week 24 visit, participants will take 100 mg crinecerfont (2 capsules, active study drug) twice a day through to at least Month 12.
At Month 12, if participants have reached a certain level of glucocorticoid dose reduction, they will continue with the same dose of active study drug (crinecerfont) 100 mg twice a day through Month 18. If they have not decreased glucocorticoid dose to a certain level by Month 12, participants will be randomly assigned, like the flip of a coin, to take one of the following:
• 50 mg crinecerfont with breakfast and 150 mg with evening meal. There is a 2 in 3 chance of being assigned to this dose.
• continue with 100 mg twice a day. There is a 1 in 3 chance of being assigned to this dose.
Participants will take the assigned dose for the remainder of the study (through Month 18). Everyone who is randomly assigned at Month 12 will receive 2 capsules in the morning and 3 capsules in the evening (including some placebo capsules) so that they will not know which active dose they are on.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: 2008094595, Congenital Adrenal Hyperplasia
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Age: 18 years - 100 years
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Gender: All
Individuals at least 18 years of age with a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH based on standard medically accepted criteria such as elevated 17-OHP level, confirmed CYP21A2 genotype, positive newborn screening with confirmatory second-tier testing, or cosyntropin stimulation.
Women who are pregnant or breastfeeding will not be eligible.
Individuals with a known or suspected diagnosis of any of the other forms of classic CAH including 11-β-hydroxylase deficiency, 17-α-hydroxylase deficiency, 3-β-hydroxysteroid dehydrogenase deficiency, P450 side-chain cleavage deficiency, or P450 oxidoreductase deficiency will not be eligible.
Individuals with a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic therapy with oral, inhaled, or intranasal glucocorticoids will not be eligible.
Individuals with a clinically significant medical condition or chronic disease, history of malignancy, or with a known history of clinically concerning cardiac arrhythmia will not be eligible.
Updated on
29 Aug 2024.
Study ID: 2008094595, ENDO-NEUROCRINE-CAH-3003
