CAH - Congenital Adrenal HyperplasiaPhase 2NCT04045145
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
A Phase 2 study testing Crinecerfont in people with CAH - Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 6 sites in United States.
Trial summary
This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia… Read more about this study
- Who is it for?
- Ages 14 to 17 years
- Conditions the study is for
- CAH - Congenital Adrenal Hyperplasia
- What is being tested?
- Crinecerfont
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 6 sitesUnited States
- Study dates
- 12 Dec 2019to 2 Jul 2021
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Be in good general health.
- Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
- Be on a stable regimen of steroidal treatment for CAH that is expected to remain stable throughout the study.
- Participants of childbearing potential must be instructed on the proper use of barrier methods of contraception and agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently from screening until the final study visit or a prespecified window after the last dose of study drug, whichever is longer.
- Participants of childbearing potential must have a negative pregnancy test at screening and negative urine pregnancy test at baseline.
- Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline.
- Be willing and able to adhere to the study regimen and study procedures described in the protocol and informed consent/assent form, including all requirements at the study center and return for the follow-up visit.
Who cannot join
- Have a clinically significant unstable medical condition or chronic disease, or malignancy.
- Had a medically significant illness within 30 days of screening.
- Have a known or suspected differential diagnosis of any of the other known forms of classic CAH.
- Have a history that includes bilateral adrenalectomy, hypopituitarism, or other condition requiring daily therapy with orally administered glucocorticoids.
- Are pregnant or lactating females.
- Have a history of epilepsy or serious head injury.
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- Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
- Have hypersensitivity to any corticotropin releasing hormone antagonists.
- Test positive at screening for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), or have a history of a positive result.
- Have a recent history of alcohol or drug abuse, or current evidence of substance dependence or abuse criteria.
- Used any anticoagulants or antiplatelet therapies within 30 days before screening.
- Have an active bleeding disorder.
- Used any other investigational drug within 30 days before initial screening, or plans to use an investigational drug (other than the study drug) during the study.
- Have a blood loss ≥250 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Neurocrine Clinical Site
Ann Arbor, Michigan, 48109
Neurocrine Clinical Site
Aurora, Colorado, 80045
Neurocrine Clinical Site
Minneapolis, Minnesota, 55454
Neurocrine Clinical Site
Philadelphia, Pennsylvania, 19104
Neurocrine Clinical Site
San Diego, California, 92123
Neurocrine Clinical Site
Seattle, Washington, 98105
About this study
Official title: A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH).
- Conditions
- CAH - Congenital Adrenal Hyperplasia
- Being tested
- Crinecerfont
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CrinecerfontDrug
Also called NBI-74788
Crinecerfont administered orally for 14 consecutive days.
| Study group | Role | What they receive |
|---|---|---|
| Crinecerfont 50 milligrams (mg) Twice Daily (BID) | Experimental | Crinecerfont administered orally for 14 consecutive days. Receives: Drug: Crinecerfont |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)
Percent changes in 17-OHP were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The 2 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Secondary outcomes (4)
Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period)
Percent changes in 17-OHP were assessed through the collection of samples for a 24-hour period prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The average of each participant's serial sampling values at each visit where serial sampling was performed was calculated and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages)
Percent changes in ACTH were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages)
Percent changes in androstenedione were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages)
Percent change in testosterone were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2019
- Study start12 Dec 2019
- Primary completion2 Jul 2021
- Study completion2 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CAH - Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04045145
- Sponsor's protocol ID
- NBI-74788-CAH2008
- Status last verified
- Jul 2024
- First submitted
- 31 Jul 2019
- Last updated
- 18 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Newfield RS, Sarafoglou K, Fechner PY, Nokoff NJ, Auchus RJ, Vogiatzi MG, Jeha GS, Giri N, Roberts E, Sturgeon J, Chan JL, Farber RH. Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2023 Oct 18;108(11):2871-2878. doi: 10.1210/clinem/dgad270.
PubMed 37216921