A Study to Evaluate Atumelnant in Adults with Congenital Adrenal Hyperplasia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Crinetics Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Hormonal diseases [C19]

Trial duration

26 Feb 2026 → ongoing

Decision date (initial)

2025-12-08

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Crinetics Pharmaceuticals, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Safety, Efficacy

To evaluate efficacy of atumelnant, compared with placebo, in reducing daily GC dosage while maintaining adrenal androgen control at the end of the 32-week Treatment Period

Secondary objectives 1

1. To evaluate efficacy of atumelnant, compared with placebo, in reducing adrenal steroid levels and other CAH disease burden at Week 2. To evaluate efficacy of atumelnant, compared with placebo, in reducing adrenal steroid levels and other CAH disease burden at the end of the 32-week Treatment Period. To evaluate efficacy of atumelnant, compared with placebo, in reducing daily GC dosage while maintaining adrenal androgen control at the end of the 32-week Treatment Period. To evaluate efficacy of atumelnant, compared with placebo, in reducing daily GC dosage while maintaining adrenal androgen control at the end of the 32-week Treatment Period and reducing adrenal androgens over time.

Conditions and MedDRA coding

Classic Congenital Adrenal Hyperplasia

Study design 1 period

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, Swedish Medical Products Agency, Medicines Evaluation Board

EMA paediatric investigation plan (PIP)

EMEA-000023-PIP34-37

Plan to share IPD

No

IPD plan description

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 1. Male or female, between ≥18 to <75 years of age at the time of signing the ICF.
2. 2. Willing and able to understand and adhere to the study procedures as specified in the protocol and comply with the study treatment.
3. 3. Have classic CAH due to 21-OHD confirmed by the Investigator and approved by the Medical Monitor.
4. 4. Participants with levels of morning serum A4 as follows: A4 >ULN and treated with <11 mg/m2/day (physiologic) GC doses in hydrocortisone equivalents OR normal A4 (above mid-range to ≤ULN) and treated with ≥15 mg/m2/day GC doses in hydrocortisone equivalents OR A4 >ULN and treated with ≥11 mg/m2 /day GC doses in hydrocortisone equivalents.
5. 5. On a stable (defined as no dose change of >X mg/day hydrocortisone equivalent within 2 months prior to Screening) regimen of GC replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone, dexamethasone) at the time of informed consent.
6. 6. If treated with mineralocorticoids (fludrocortisone), the dose should be stable for at least 2 months prior to Screening without orthostatic hypotension, and with serum sodium and potassium in the normal range.
7. 7. If on estrogen therapy (any route), the dose must be stable for at least 3 months prior to Screening.
8. 8. Female participants who engage in heterosexual intercourse must: a. Be of nonchildbearing potential, defined as either surgically sterile (ie, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), OR b. Agree to use a highly effective or a clinically acceptable method of contraception from the beginning of Screening until at least 2 weeks after the last dose of study drug. Contraceptive use by men and women also should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Periodic abstinence (ie, calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. c. See Section 12.1.4 for additional contraception guidance.
9. 9. Male participants who engage in heterosexual intercourse must: a. Agree to use a condom when sexually active with a female partner of childbearing potential from Screening until at least 2 weeks after the last dose of study drug (or be surgically sterile [ie, vasectomy with a confirmed absence of sperm in ejaculate]) OR b. Agree to remain abstinent on a long-term and persistent basis during the study and until at least 2 weeks after the last dose of study drug. c. Agree to not donate sperm for the duration of the study and until at least 2 weeks after the last dose of study drug. d. See Section 12.1.4 for additional contraception guidance.

Exclusion criteria 1

1. Refer to section 5.3 of the Protocol for the full exclusion criteria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of participants with morning post-GC A4 ≤ULN who are on physiologic GC replacement at Week 32

Secondary endpoints 1

1. Percent change from baseline of morning A4 at Week 2 Percent change from baseline of morning 17-OHP at Week 32  Proportion of participants with morning pre-GC A4 ≤ULN who are on physiologic GC replacement at Week 32  Percent change from baseline in GC daily dose when morning post-GC A4 ≤ULN at week 32

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Crinetics Pharmaceuticals Inc.

Sponsor organisation

Crinetics Pharmaceuticals Inc.

Address

6055 Lusk Boulevard

City

San Diego

Postcode

92121-2700

Country

United States

Scientific contact point

Organisation

Crinetics Pharmaceuticals Inc.

Contact name

Clinical Medical Lead

Public contact point

Organisation

Crinetics Pharmaceuticals Inc.

Contact name

Clinical Medical Lead

Third parties 15

Locations

7 EU/EEA countries · 27 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 92 files

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

Application history

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