CTRI/2023/12/060825尚未招募2 期
A 12-Week, Phase 2 Open-Label, sequential dose cohort study to evaluate the safety, efficacy and pharmacokinetics of CRN04894 treatment in participants with Congenital Adrenal Hyperplasia. - NI
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •1.Willing and able to understand and adhere to the study procedures.
- •2.Classic 21-hydroxylase deficiency confirmed by the Investigator and approved by the
- •Medical Monitor.
- •3.The A4 and 17-OHP values taken before morning glucocorticoid (GC) dose during
- •Screening Visit 2 (at Week-2), should meet these criteria:
- •a.A4 more than 1.5 X upper limit of normal (ULN) and
- •b.17-OHP more than or equal to 1000 ng/dL (30.3 nmol/L); historical results from within the last 2 years will be accepted if the participant has been on the current dose of GC since that time.
- •Participants who failed screening based on findings the Investigator believes are
- •temporary and not reflective of the usual state of the participant (eg, normal A4 levels
- •when the participant usually is well above this value) can be considered for rescreening.
- •These cases should be discussed with the Medical Monitor.
- •4.On a stable (defined as no dose change of more than 5 mg/day hydrocortisone equivalent within 6 months prior to Screening) regimen of glucocorticoid replacement (eg, hydrocortisone, prednisolone, prednisone, methylprednisolone).
- •5.Compliance, as judged per principal investigator (PI) discretion, with glucocorticoid
- •replacement and mineralocorticoid replacement (if applicable) regimen documented
- •during the Screening Period.
- •6.Minimum total daily dose of more than or equal to 15 mg hydrocortisone (or equivalent).
- •7.If on estrogen therapy (any route), dose must be stable for at least 3 months prior to
- •8.Female participants who engage in heterosexual intercourse must be of nonchildbearing
- •potential, defined as either surgically sterile (ie, hysterectomy, bilateral salpingectomy,
- •tubal ligation 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 of a follicle stimulating hormone (FSH) more than or equal to 30 IU/L is required to confirm menopause. Participants not using hormone replacement therapy must 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 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.
- •9.Male participants: The participant should 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.
排除标准
- •1. Diagnosis of any other form of CAH other than classic 21-hydroxylase deficiency
- •2. Dexamethasone use within 30 days of screening
- •3. History of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic
- •glucocorticoid therapy
- •4. Night shift workers or any other reason for abnormal sleep/wake cycles
- •5. Clinically significant unstable medical condition or chronic disease other than CAH
- •6. History of major surgery/surgical therapy for any cause within 4 weeks prior to Screening
- •7. Diabetes mellitus treated with insulin for less than 6 weeks prior to Screening, or with
- •change in total daily insulin dose by more than 15 percent within 6 weeks prior to Screening
- •8. Poorly controlled diabetes mellitus defined as having a hemoglobin A1c (HbA1c) more than or equal to 8.5 percent (more than or equal to 69 mmol/mL), or estimated HbA1c based on fructosamine if HbA1c is not evaluable (eg, due to hemoglobinopathies)
- •9. 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.
- •10. History of unstable angina or acute myocardial infarction within 12 weeks prior to
- •Screening or other clinically significant cardiac disease at the time of Screening as judged
- •by the Investigator
- •11. Concomitant 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
- •12. History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or
- •cervical carcinoma in situ
- •13. Pregnant or lactating
- •14. Known history of illicit drug or alcohol abuse within the last year
- •15. Use of antiandrogen therapy in the past 3 months (eg, spironolactone, finasteride,
- •cyproterone acetate, flutamide)
- •16. Use of testosterone, androgen-containing supplements, aromatase inhibitors, or growth
- •17. Use of medications that are strong inducers of cytochrome P450 (CYP) 3A4 within
- •30 days prior to Day 1 of the study. These include but are not limited to apalutamide,
- •carbamazepine, enzalutamide, mitotane, phenytoin, rifampin, or St. John’s wort.
- •18. Use of medications (all routes of administration ie, oral, topical and inhaled) or ingestion
- •of food that are strong or moderate inhibitors of CYP3A4 within 14 days prior to Day 1
- •of the study. These include but are not limited to boceprevir, cobicistat, danoprevir and
- •ritonavir, elvitegravir and ritonavir, grapefruit juice (more than 8 ounces daily), indinavir
- •and ritonavir, itraconazole, ketoconazole, lopinavir and ritonavir, nirmatrelvir and
- •ritonavir, paritaprevir and ritonavir and (ombitasvir and/or dasabuvir), posaconazole,
- •ritonavir, saquinavir and ritonavir, telaprevir, tipranavir and ritonavir, telithromycin,
- •troleandomycin, voriconazole, clarithromycin, idelalisib, nefazodone, nelfinavir,
- •aprepitant, ciprofloxacin, conivaptan, crizotinib, cyclosporine, diltiazem, dronedarone,
- •erythromycin, fluconazole, fluvoxamine, imatinib, tofisopam, verapamil.
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