NCT07080385招募中2 期
A Phase 2/3, Multicenter, Single-Arm, Open-Label Study Evaluating the Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Calcilytix Therapeutics, Inc., a BridgeBio company6 个研究点 分布在 2 个国家目标入组 28 人开始时间: 2026年1月30日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 28
- 试验地点
- 6
- 主要终点
- Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3
研究概览
简要总结
The overall objective of this study is to evaluate the pharmacokinetics (PK), efficacy, and safety of encaleret in pediatric participants from birth to 17 years of age with ADH1.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provide written informed consent (if legally permitted), or have written informed consent from a parent/legal guardian and provide assent (where required and as appropriate per local requirements)
- •Have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance of the calcium-sensing receptor (CASR), associated with biochemical findings of hypoparathyroidism at screening or a documented history of hypoparathyroidism as manifested by hypocalcemia and intact parathyroid hormone (PTH) <40 picogram per milliliter (pg/mL) (4.2 picomoles per liter [pmol/L])
- •Have at least 1 symptom or sign of hypoparathyroidism at screening or a documented history of symptoms or signs of hypoparathyroidism
- •Be on ADH1 treatment for at least 6 months before screening for cohorts 1 to 3, or for at least 3 months before screening for cohort 4
排除标准
- •History of thyroid or parathyroid surgery
- •History of renal transplantation
- •History of cancer (except thyroid cancer, basal cell skin cancer, or squamous cell skin cancer), skeletal malignancies, bone metastases, irradiation (radiotherapy) to the skeleton, chemotherapy with alkylating agents, Paget disease, fibrous dysplasia, chronic osteomyelitis, bone infarcts, benign bone tumors with curettage and bone grafts, retinoblastoma, or Li-Fraumeni syndrome within 5 years before screening
- •Received any investigational medicinal product within 30 days or 5 half-lives before Day 1, whichever is longer, or is in follow-up for another interventional clinical study during screening
- •Treatment with a strong P-glycoprotein (P-gp) inhibitor within 300 days before screening for amiodarone or within 30 days before screening for any other strong P-gp inhibitor
- •Treatment with cardiac glycosides, or is being breastfed while the participant's nursing mother is treated with cardiac glycosides, within 30 days before screening
- •Presence or history of any disease or condition (eg, drug or alcohol dependence) that would affect the participant's safety, treatment compliance, or ability to complete the study, in the opinion of the investigator
- •Other protocol defined inclusion/exclusion criteria apply.
研究组 & 干预措施
Encaleret
Experimental
Participants will receive encaleret dosing in Period 1 (6 days), Period 2 (20 weeks) and Period 3 (4 weeks). Following completion of Period 3, participants will have the option to enter a long-term extension (LTE) to continue encaleret for an additional approximately 24 months or until the sponsor decides to end the study, whichever occurs first.
干预措施: Encaleret (Drug)
结局指标
主要结局
Period 1: Maximum Plasma Concentration (Cmax) of Encaleret and Metabolites M1, and M3
时间窗: 5 days
Period 1: Area Under the Plasma Concentration-time Curve (AUC) of Encaleret and Metabolites M1, and M3
时间窗: 5 days
Period 3: Number of Participants with Albumin-corrected Blood Calcium (cCa) and Urinary Calcium (UCa) Excretion Response
时间窗: Week 25
次要结局
- Period 1: Change from Baseline in Blood cCa(Baseline up to Day 5)
- Period 1: Change from Baseline in Blood Intact Parathyroid Hormone (iPTH) Concentration(Baseline up to Day 5)
- Period 1: Change from Baseline in Blood Phosphate Concentration(Baseline up to Day 5)
- Period 1: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration(Baseline up to Day 5)
- Period 1: Change from Baseline in Blood Magnesium Concentration(Baseline up to Day 5)
- Period 1: Change from Baseline in 24-hour UCa in Toilet Trained Participants(Baseline up to Day 5)
- Period 1: Change from Baseline in Spot Ratio of UCa/Urinary Creatinine (UCr) In Non-toilet Trained Participants(Baseline up to Day 5)
- Period 3: Number of Participants with Blood iPTH Within the Reference Range(Week 25 (post-dose))
- Period 3: Number of Participants with Blood Phosphate Within the Reference Range(Week 25)
- Period 3: Number of Participants with Blood Magnesium Within the Reference Range(Week 25)
- Period 3: Number of Participants with Blood 1,25-(OH)2 Vitamin D Within the Reference Range(Week 25)
- Periods 1, 2, and 3: Change from Baseline in Short Form-10 Health Survey for Children (SF-10) in Participants Aged ≥6 Years(Baseline up to Week 25)
- Periods 1, 2, and 3: Dose of Calcium Supplements and/or Active Vitamin D Analogs Used as Rescue Therapy(25 weeks)
- Periods 1, 2, and 3: Dosing Frequency of Calcium Supplements and/or Active Vitamin D Analogs Used as Rescue Therapy(25 weeks)
- Periods 1, 2, 3, and LTE: Number of Participants with Adverse Events (AEs) and Serious AEs (SAEs)(145 weeks)
- LTE: Change from Baseline in Blood cCa(Baseline up to 145 weeks)
- LTE: Change from Baseline in Blood iPTH Concentration(Baseline up to 145 weeks)
- LTE: Change from Baseline in Blood Phosphate Concentration(Baseline up to 145 weeks)
- LTE: Change from Baseline in Blood 1,25-(OH)2 Vitamin D Concentration(Baseline up to 145 weeks)
- LTE: Change from Baseline in Blood Magnesium Concentration(Baseline up to 145 weeks)
- LTE: Change from Baseline in 24-hour UCa In Toilet Trained Participants(Baseline up to 145 weeks)
- LTE: Change from Baseline in Spot Ratio of UCa/UCr In Non-toilet Trained Participants(Baseline up to 145 weeks)
- LTE: Change from Baseline in SF-10 in Participants Aged ≥6 Years(Baseline up to 145 weeks)
研究者
研究点 (6)
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