A Long-term Open-label Study Investigating the Safety and Tolerability of NPSP558, a Recombinant Human Parathyroid Hormone (rhPTH[1-84]), for the Treatment of Adults With Hypoparathyroidism - A Clinical Extension Study (RACE)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 51
- 试验地点
- 14
- 主要终点
- Number of Responders With Calcium Source at Week 52
研究概览
简要总结
This study is a long-term, open-label study using NPSP558 for the treatment of adult patients with Hypoparathyroidism.
详细描述
Patients with a history of Hypoparathyroidism will be enrolled to receive study drug for up to 80 months, which will be injected daily in either thigh. During that time they will be monitored for safety (specifically calcium levels in blood or urine). In addition, the patients' intake of Vitamin D and Calcium will be measured.
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
- •Previously completed the rhPTH[1-84] RELAY study (8 weeks of active therapy) and/or previously completed the rhPTH[1-84] REPLACE study (Visit 18).
- •Able to perform daily SC self-injections of study medication (or have a designee perform injection).
- •Women who are (1) postmenopausal; (2) surgically sterilized; or, (3) of childbearing potential with a negative pregnancy test and who consent to use two acceptable methods of contraception for the duration of the study.
- •Males who have female partners of childbearing potential must use two acceptable forms of contraception for the duration of the study.
- •Serum creatinine <1.5 mg/dL at enrollment.
- •Total serum calcium less than or equal to upper limit of normal (ULN) based on local laboratory result prior to enrollment.
- •Serum 25 hydroxy (OH) vitamin D less than or equal to 1.5 times the ULN within approximately 16 weeks prior to enrollment.
排除标准
- •Any condition that, in the investigator's opinion after consultation with the sponsor, would preclude the safe use of parathyroid hormone (PTH).
- •Pregnant or lactating women.
- •Any disease or condition which has a high probability of precluding the subject from completing the study or where the subject cannot or will not appropriately comply with study requirements.
研究组 & 干预措施
NPSP558
titration of 25, 50, 75 or 100 μg
干预措施: NPSP558 (Drug)
结局指标
主要结局
Number of Responders With Calcium Source at Week 52
时间窗: Week 52
A responder was defined as a participant who met all of the following 3 criteria at each (1) a greater than (\>) 50% reduction from baseline or less than (\<) 500 milligram (mg) of daily calcium supplementation. (2) a \>50% reduction from baseline or \<0.25 microgram (mcg) of daily calcitriol supplementation. (3) an albumin-corrected total serum calcium concentration that was normalized or maintained compared to the baseline greater than or equal to (\>=) 1.875 millimoles per liter (mmol/L) and not exceeding the Upper Limit of Normal (ULN) values (2.15 to 2.55 mmol/L). End of Treatment (EOT) was defined as the last determination of response or last available measurement during the treatment period. Number of responders with calcium source for citrate and carbonate at week 52 was reported here.
Number of Participants With Treatment Emergent Adverse Event (TEAE) and Treatment Emergent Serious Adverse Event (TESAE)
时间窗: From start of study drug administration up to follow-up (82 months)
SAE is an adverse event (AE) that results in death, life threatening, persistent or significant incapacity or substantial disruption of ability to conduct normal life functions, hospitalization or prolongation of existing hospitalization, congenital anomaly or birth defect, important medical events that may not result in death, be life threatening, or require hospitalization. An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical or medicinal product. Treatment emergent adverse events (TEAEs) were defined as AEs whose onset occurs, severity worsens or intensity increases after receiving the study medication of this study and \<= 30 days after last dose of study drug.
Number of Responders With Calcium Source at End Of Treatment (EOT) (Up to 82 Months)
时间窗: EOT (up to 82 months)
A responder was defined as a participant who met all of the following 3 criteria at each (1) a greater than (\>) 50% reduction from baseline or less than (\<) 500 milligram (mg) of daily calcium supplementation. (2) a \>50% reduction from baseline or \<0.25 microgram (mcg) of daily calcitriol supplementation. (3) an albumin-corrected total serum calcium concentration that was normalized or maintained compared to the baseline greater than or equal to (\>=) 1.875 millimoles per liter (mmol/L) and not exceeding the Upper Limit of Normal (ULN) values (2.15 to 2.55 mmol/L). End of Treatment (EOT) was defined as the last determination of response or last available measurement during the treatment period. Number of responders with calcium source for citrate and carbonate at EOT was reported here.
次要结局
- Percent Change From Baseline in Albumin Corrected Total Serum Calcium (ACSC) at EOT (Up to 82 Months)(Baseline, EOT (up to 82 months))
- Change From Baseline in Bone Mineral Density (BMD) at Week 52 and EOT (Up to 82 Months)(Baseline, Week 52 and EOT (up to 82 months))
- Percent Change From Baseline in Oral Calcium Supplementation at Week 52 and EOT (Up to 82 Months)(Baseline, Week 52 and EOT (up to 82 months))
- Percent Change From Baseline in Oral Calcitriol Supplementation at Week 52 and EOT (Up to 82 Months)(Baseline, Week 52 and EOT (up to 82 months))
- Change From Baseline in 24-Hour Urine Calcium Excretion Through EOT (Up to 82 Months)(Baseline, EOT (up to 82 months))
- Change From Baseline in Bone Turnover Markers at EOT (Up to 82 Months)(Baseline, EOT (up to 82 months))
- Change From Baseline in Serum Calcium Concentration in Participants Who Used and Calcium Sparing Diuretics at EOT (Upto 82 Months)(Baseline, EOT (upto 82 months))
- Change From Baseline in Serum Phosphate at Month 72 and EOT (Upto 82 Months)(Baseline, Month 72, EOT (upto 82 months))
- Change From Baseline in 24-hour Urine Calcium Excretion in Participants Who Used Calcium-Sparing Diuretics Through EOT (Up to 82 Months)(Baseline, EOT (up to 82 months))
- Number of Participants Who Maintained a Calcium Phosphate Product in A Normal Range at EOT (Up to 82 Months)(EOT (up to 82 months))
- Change From Baseline in Serum Carboxy Terminal Telopeptide of Type I Collagen (s-CTx) Bone Turnover Marker at EOT (Up to 82 Months)(Baseline, EOT (up to 82 months))