NCT06712823招募中2 期
An Open-label, Long-term Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia (CALM2-CAH)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 15
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) leading to discontinuation
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of atumelnant (CRN04894).
详细描述
This single-arm, long-term, open-label, study is designed to evaluate the safety, tolerability, and efficacy of atumelnant (CRN04894) in participants with congenital adrenal hyperplasia (CAH). Enrollment will be limited to individuals who completed a parent Crinetics atumelnant CAH study, and in the opinion of the Investigator had an acceptable benefit-risk assessment in the completed study and would benefit from continued dosing in this Open-Label Extension (OLE) study.
A total of approximately 150 - 200 participants may be enrolled in the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 74 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants are eligible to be included in the study only if all the following criteria apply:
- •Participants with CAH who have completed the Treatment Period in a Crinetics parent atumelnant CAH study, and in the opinion of the Investigator had an acceptable benefit-risk assessment in the completed study and would benefit from continued dosing in this extension study.
- •Group 1: Participants meeting the above criteria and did not have study drug administration interrupted between End of Trial (EOT) of the parent study and the commencement of the OLE study.
- •Group 2: Participants meeting the above criteria but had study drug administration interrupted between EOT of the parent study and the commencement of the OLE study.
- •Female participants who engage in heterosexual intercourse must:
- •Be of nonchildbearing potential, defined as either surgically sterile (ie, hysterectomy, bilateral salpingectomy for at least 3 months, or bilateral oophorectomy), OR
- •Be postmenopausal with at least 1 year of amenorrhea. In participants with less than 1 year of amenorrhea, confirmation is required with 2 follicle-stimulating hormone (FSH) measurements. A documented, historical test result measured prior to Screening may be used as 1 of the 2 measurements. The FSH value should be ≥30 IU/L to confirm menopausal status, OR
- •Agree to use a highly effective 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.
- •Male participants 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 agree to remain abstinent on a long-term and persistent basis). Male participants should also agree to not donate sperm for the duration of the study and until at least 2 weeks after the last dose of study drug.
- •Participants are willing and able to give signed informed consent, including compliance with the requirements and restrictions listed in the Informed Consent Form (ICF).
- •Participants are willing and able to comply with the study procedures as specified in the protocol and comply with the study treatment.
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Any medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardize the participant's safety or ability to complete the study.
- •Participants have known history of (that is within the past 12 months), or current alcohol or drug abuse.
- •Participants have any mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study, and/or evidence of poor compliance with medical instructions.
- •Participants have a known allergy or hypersensitivity to any of the test materials or related compounds, including being at high risk of adrenal insufficiency as judged by the Investigator.
- •Women who are pregnant or lactating or, if of childbearing potential, who are unwilling to use highly effective contraception as described in this study. Male participants who are unwilling to use highly effective contraception as described in this study.
- •Participant is an employee or immediate family member of an employee of Crinetics.
- •Participants who have been dosed with an investigational drug (other than atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to informed consent or plan to use an investigational drug in another study.
- •Participants who have had an active malignant disease within the last 5 years prior to Screening excluding dermal squamous or basal cell carcinoma of the skin with complete local excision or resected cervical carcinoma in situ.
- •Participants who are committed to an institution by virtue of an order issued either by the judicial or the administrative authorities.
- •Specific for Participants Not Currently Receiving Atumelnant
- •Participants with any clinically significant abnormal laboratory test during Screening or clinically significant concomitant disease other than CAH including but not limited to cardiovascular disease; moderate or severe renal insufficiency (estimated glomerular filtration rate <30 mL/min/1.73 m2 using Chronic Kidney Epidemiology Collaboration [CKD-EPI] formula) at Screening; or Significant liver disease or alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >3× upper limit of normal (ULN), and/or total bilirubin >1.5×ULN during Screening. Participants with previously diagnosed Gilbert's syndrome not accompanied by other hepatobiliary disorders and associated with total bilirubin <3.5 mg/dL (<51.3 μmol/L) will be permitted.
- •Participants with a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
- •Participants with a history of major surgery/surgical therapy for any cause within 4 weeks prior to Screening.
- •Participants with poorly controlled diabetes mellitus defined as having a hemoglobin A1c (HbA1c) ≥8.5% (≥69 mmol/mL).
- •Participants with hypothyroidism who are not receiving adequate hormone replacement therapy based on thyroid hormone levels measured at the time of Screening, as determined by the Investigator.
- •Participant has an average (of 3 electrocardiograms [ECGs]) Fridericia's corrected QT (QTcF) interval >450 milliseconds (msec) (men) or >470 msec (women), time interval between P and R waves (PR interval) >220 msec, time interval of the QRS complex (QRS) interval >120 msec, second- or third-degree atrioventricular block, left bundle branch block, or hemiblock at Screening.
研究组 & 干预措施
Treatment
Experimental
Open-label treatment period (up to 2 years). The maximum atumelnant dose permitted is not to exceed the highest atumelnant dose explored in a parent study for the indication.
干预措施: atumelnant (CRN04894) (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) leading to discontinuation
时间窗: Week 108
Incidence of glucocorticoid (GC) deficiency / adrenal insufficiency and adrenal crisis
时间窗: Week 108
Incidence of hospitalizations related to congenital adrenal hyperplasia (CAH)
时间窗: Week 108
Change from baseline in morning (before 11:00 AM) serum androstenedione (A4) over time
时间窗: Week 108
Incidence of treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (SAEs), adverse events of special interest (AESI [adrenal insufficiency]) and any adverse events (AEs) leading to discontinuation
时间窗: Week 108
次要结局
- Change from baseline in morning (before 11:00 AM) serum 17-hydroxyprogesterone (17-OHP) over time(Week 108)
- Change from baseline in daily glucocorticoid (GC) dose (hydrocortisone [HC] mg equivalents) over time(Week 108)
- Change from baseline in daily glucocorticoid (GC) dose (hydrocortisone [HC] mg equivalents body surface area [BSA] adjusted) over time(Week 108)
研究者
研究点 (15)
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