A Phase 4, Open-Label, Single-Center Clinical Study of Extended Use of rhPTH(1-84) in Hypoparathyroidism
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 39
- 试验地点
- 1
- 主要终点
- Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 30
研究概览
简要总结
Chronic hypoparathyroidism is a life-long and irreversible disease for which the chronic administration of rhPTH(1-84) is a potential treatment option. The group of participants in the AAAE0544 core study has been taking rhPTH(1-84) for the treatment of hypoparathyroidism for up to 11 years. This study is designed to extend this experience and gain knowledge about how safe and effective rhPTH(1-84) is in participants with hypoparathyroidism over a long-term duration.
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
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
rhPTH(1-84)
Participants will receive 25, 50, 75, and 100 microgram (mcg) of rhPTH(1-84) subcutaneous injection to the thigh via a multidose pen injector device once daily for 36 months. The dose will be individualized based on albumin-corrected serum calcium (ACSC) and 24-hour calcium urinary excretion to achieve a serum calcium level in the lower half of the normal range.
干预措施: rhPTH(1-84) (Drug)
结局指标
主要结局
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 30
时间窗: Baseline (402), Month 30
Baseline (402) was defined as the last available pre-dose value in SHP634-402.
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at End of Treatment (EOT) (up to Month 36)
时间窗: Baseline (402), EOT (up to Month 36)
Baseline (402) was defined as the last available pre-dose value in SHP634-402. EOT was defined as the last determination of response during the treatment period (from the date of first dose to the date of last dose + 1 day), was analyzed in addition to the scheduled visits.
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 6
时间窗: Baseline (402), Month 6
Baseline (402) was defined as the last available pre-dose value in SHP634-402.
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 12
时间窗: Baseline (402), Month 12
Baseline (402) was defined as the last available pre-dose value in SHP634-402.
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 18
时间窗: Baseline (402), Month 18
Baseline (402) was defined as the last available pre-dose value in SHP634-402.
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 24
时间窗: Baseline (402), Month 24
Baseline (402) was defined as the last available pre-dose value in SHP634-402.
Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 36
时间窗: Baseline (402), Month 36
Baseline (402) was defined as the last available pre-dose value in SHP634-402.
次要结局
- Change From Baseline (402) in Urinary Calcium Excretion at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Functional Assessment of Cancer Therapy - Cognitive Function (FACT-Cog) Assessment at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale Total Score at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Hospital Anxiety and Depression Scale (HADS) at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Bone Architecture Evaluated Using Dual-energy X-ray Absorptiometry (DXA) for Total T-scores at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline, At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Concentration of Serum Phosphate Levels at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Active Vitamin D Supplement Dose Based on Investigator-prescribed Data and Diary Data at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Mean Bone Histology (Biopsy) of Bone Formation Rate at Month 12, and EOT (up to Month 36)(At Month 12, and EOT (up to Month 36))
- Percentage of Participants Who Achieved ACSC Concentrations Above, Below, or in the Range of 1.875 mmol/L (7.5 Milligram Per Deciliter [mg/dL]) to Upper Limit of Normal (ULN) at Month 6, 12, 18, 24, 30, 36 and EOT (up to Month 36)(At Month 6, 12, 18, 24, 30, 36 and EOT (up to Month 36))
- Change From Baseline (402) in Hypoparathyroidism Symptoms Diary (HPT-SD) at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Health-related Quality-of-life Measured With 36-item Short Form Health (SF-36) Survey at Month 12, 24, 36, and EOT (up to Month 36)(Baseline (402), At Month 12, 24, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Concentration of Bone Turnover Markers at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Concentration of Bone Turnover Marker for Bone Specific Alkaline Phosphatase and Tartrate-resistant Acid Phosphatase-5b at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Bone Architecture Evaluated Using Dual-energy X-ray Absorptiometry (DXA) for Bone Mineral Density (BMD) at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline, At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Calcium Supplement Doses and Elemental Calcium Based on Investigator-prescribed Data and Diary Data at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline, At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Change From Baseline (402) in Bone Architecture Evaluated Using High-resolution Peripheral Quantitative Computerized Tomography (HRpQCT) for Total Area of Radius and Tibia at Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36)(Baseline (402), At Month 6, 12, 18, 24, 30, 36, and EOT (up to Month 36))
- Mean Bone Histology (Biopsy) of Adjusted Apposition Rate and Cancellous Minimum Apposition Rate at Month 12, and EOT (up to Month 36)(At Month 12, and EOT (up to Month 36))
- Mean Bone Histology (Biopsy) of Cancellous Bone Volume, Cancellous Eroded Surface, Cancellous Mineral Surface, And Cancellous Osteoid Surface at Month 12, and EOT (up to Month 36)(At Month 12, and EOT (up to Month 36))
- Mean Bone Histology (Biopsy) of Cancellous Osteoid Thickness and Cortical Width at Month 12, and EOT (up to Month 36)(At Month 12, and EOT (up to Month 36))