NCT07706764招募中3 期
A Phase 3, Multicenter, Open-Label Single-Arm Clinical Trial to Assess the Safety, Tolerability, Pharmacokinetics, and Efficacy of Palopegteriparatide Administered Subcutaneously Daily in the Adolescent Population (12 Years to Less Than 18 Years of Age) With Chronic Hypoparathyroidism
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- Percentage of participants meeting the multicomponent efficacy endpoint at Week 26
研究概览
简要总结
This trial will enroll adolescents between ages of ≥12 and <18 years with clinically diagnosed hypoparathyroidism . The purpose of the study is to see how well treatment with once-daily palopegteriparatide works and how safe it is. At least 12 participants will receive palopegteriparatide for 234 weeks. This trial will be conducted in Europe.
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
- 12 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Males and females, 12 to less than 18 years of age
- •2. Participants with postsurgical chronic hypoparathyroidism, or auto-immune, genetic, or idiopathic hypoparathyroidism for at least 26 weeks
- •3. Normal levels of serum 25(OH) vitamin D and magnesium
- •4. Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73
- •5. Able to perform daily subcutaneous self-injections of palopegteriparatide (or have a caregiver to perform injections)
- •6. Body mass index (BMI) Z-score greater than -2 SDS and below + 3 SDS
- •7. Written, signed informed consent
排除标准
- •1. Impaired responsiveness to PTH which is characterized as PTH-resistance, with elevated PTH levels in the setting of hypocalcemia
- •2. Any disease that might affect calcium metabolism or calcium-phosphate homeostasis or PTH levels other than hypoparathyroidism, such as active hyperthyroidism
- •3. Use of loop diuretics, phosphate binders (other than calcium supplements), digoxin, lithium, methotrexate, biotin >30 µg/day, or systemic corticosteroids (other than as replacement therapy). Short course use of steroids (≤2 weeks/year) equivalent to prednisone ≤60 mg/day is permitted
- •4. Use of thiazide diuretic
- •5. Use of PTH-like drugs
- •6. Use of other drugs known to influence calcium and bone metabolism, such as calcitonin, fluoride tablets (>0.5 mg/day), strontium, or cinacalcet hydrochloride, within 12 weeks prior to Screening
- •7. Use of osteoporosis therapies known to influence calcium and bone metabolism, i.e., bisphosphonate (oral or intravenous [IV]), denosumab, raloxifene, or romosozumab therapies within 2 years prior to Screening
- •8. Non-hypocalcemic seizure disorder with occurrence of a seizure within 26 weeks prior to Screening
- •9. Increased risk for osteosarcoma
- •10. Female participants who are pregnant, intend to become pregnant, or are lactating
- •11. Diagnosed drug or alcohol dependence within 3 years prior to Screening
研究组 & 干预措施
Palopegteriparatide, once daily
Experimental
Participants will receive palopegteriparatide by subcutaneous injection for 234 weeks
干预措施: Palopegteriparatide (Combination Product)
结局指标
主要结局
Percentage of participants meeting the multicomponent efficacy endpoint at Week 26
时间窗: 26 weeks
The multi-component endpoint is the percentage of participants who met the following criteria at Week 26: 1) albumin-adjusted serum calcium within the normal range ; 2) independence from active vitamin D, and 3) independence from therapeutic doses of calcium
次要结局
- Percentage of participants meeting the multicomponent efficacy endpoint through Week 234(234 weeks)
- Serum biochemistries(234 weeks)
- Renal calcifications(234 weeks)
- Hospitalizations/emergency room (ER)/urgent care visits(234 weeks)
- Adverse events(234 weeks)
- Bone mineral density(234 weeks)
- Bone turnover marker(234 weeks)
- HPES-Symptom score(234 weeks)
- HPES-Impact score(234 weeks)
- Urine biochemistries(234 weeks)
研究者
研究点 (3)
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