Study to Evaluate the Safety and Efficacy of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Neurocrine Biosciences Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Trial duration

23 Nov 2021 → ongoing

Decision date (initial)

2024-05-29

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Neurocrine Biosciences, Inc.

External identifiers

EU CT number

2023-509170-33-00

EudraCT number

2020-004381-19

ClinicalTrials.gov

NCT04806451

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Pharmacogenetic, Pharmacokinetic, Efficacy

To evaluate the efficacy of crinecerfont, compared with placebo, in reducing adrenal steroid levels during a glucocorticoid-stable period.

Secondary objectives 4

1. To evaluate the efficacy of crinecerfont, compared with placebo, in reducing daily glucocorticoid dosage while maintaining adrenal androgen control.
2. To evaluate the effect of crinecerfont, compared with placebo, on clinical endpoints associated with supraphysiologic glucocorticoid dosing and androgen excess.
3. To evaluate plasma concentrations of crinecerfont and metabolites.
4. To assess the safety and tolerability of crinecerfont.

Conditions and MedDRA coding

Classic Congenital Adrenal Hyperplasia (CAH)

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002700-PIP01-19

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
2. Be a female or male 2 to 17 years of age with a body weight of at least 10 kg.
3. Have a medically confirmed diagnosis of classic CAH due to 21- hydroxylase deficiency.
4. Be on a stable regimen of glucocorticoid treatment for CAH.
5. Have elevated adrenal androgens.
6. If treated with fludrocortisone, dose should be stable for at least 1 month prior to screening. Regardless of fludrocortisone treatment, upright plasma renin activity (PRA) (in the absence of medications that confound interpretation of PRA) during screening should be <3× ULN and >lower limit of normal (LLN) on the subject's usual sodium intake (if PRA >2 × ULN and <3 × ULN, subjects must have normal age-specific systolic blood pressure and heart rate and serum potassium

Exclusion criteria 7

1. Have a diagnosis of any of the other forms of classic CAH.
2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic daily therapy with oral glucocorticoids.
3. Have a clinically significant unstable medical condition or chronic disease other than CAH
4. Have a history of malignancy, unless successfully treated with curative intent and considered to be cured.
5. Have a known history of clinically significant arrhythmia or abnormalities on screening ECG.
6. Have a known hypersensitivity or allergy to any corticotropin-releasing hormone (CRH) receptor antagonist or any component of the study drug.
7. Females who are pregnant or lactating.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in serum androstenedione at Week 4.

Secondary endpoints 2

1. Change from baseline in serum 17-hydroxyprogesterone at Week 4
2. Percent change from baseline in glucocorticoid dose at Week 28

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Neurocrine Biosciences Inc.

Sponsor organisation

Neurocrine Biosciences Inc.

Address

6027 Edgewood Bend Court

City

San Diego

Postcode

92130-8235

Country

United States

Scientific contact point

Organisation

Neurocrine Biosciences Inc.

Contact name

Neurocrine Medical Information Call Center

Public contact point

Organisation

Neurocrine Biosciences Inc.

Contact name

Neurocrine Medical Information Call Center

Third parties 16

Locations

7 EU/EEA countries · 14 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 110 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications