PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism): A Registry for Patients With Chronic Hypoparathyroidism
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 1,339
- 试验地点
- 199
- 主要终点
- Change from Baseline in Serum Magnesium
研究概览
简要总结
The main aim of this study is to find out the long-term safety and effectiveness profile of recombinant human parathyroid hormone (1-84) (rhPTH[1-84]) treatment in participants with chronic hypoparathyroidism under conditions of routine clinical practice.
Participants will be treated according to their clinic's standard practice determined by the treating doctors. Each participant will fill out a study questionnaire during a routine doctor visit.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants diagnosed with chronic hypoparathyroidism, that is, hypoparathyroidism with a duration of longer than 6 months, including:
- •Adult participants (greater than or equal to [>=] 18 years of age) who are receiving for chronic hypoparathyroidism any of the following options: standard therapy, standard therapy plus rhPTH(1-84), or rhPTH(1-84) therapy alone.
- •Pediatric participants (less than [<] 18 years of age) who are receiving for chronic hypoparathyroidism any of the following options: standard therapy, standard therapy plus rhPTH(1-84), or rhPTH(1-84) therapy alone.
排除标准
- •Participants or legally acceptable representatives unable to provide informed consent.
- •Participants using rhPTH(1-34) or who used rhPTH(1-34) for more than 2 years are excluded. Participants who had been treated with rhPTH(1-34) within 3 months of enrollment are also excluded as are participants currently using rhPTH(1-34).
- •FOR US SITES ONLY: Participants treated with rhPTH(1-84) prior to the US recall may use rhPTH(1-34) only while rhPTH(1-84) is unavailable due to the recall.
- •Participants currently enrolled in an interventional clinical study (whether or not the study is related to hypoparathyroidism); note that this does not include participants enrolled in other observational registries.
- •History of hypoparathyroidism resulting from impaired responsiveness to PTH (pseudohypoparathyroidism).
研究组 & 干预措施
Natpar(a)
Participants receiving parathyroid hormone (rhPTH(1-84) (Natpar[a]) for treatment of chronic hypoparathyroidism as per standard clinical practice will be enrolled and evaluated during the study period.
干预措施: No intervention (Other)
Conventional Therapy
Participants receiving conventional therapy/standard of care (including calcium supplements, active vitamin D, vitamin D) for treatment of chronic hypoparathyroidism as per standard clinical practice will be enrolled and evaluated during the study period.
干预措施: No intervention (Other)
结局指标
主要结局
Change from Baseline in Serum Magnesium
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in serum magnesium will be evaluated.
Change from Baseline in 25-Hydroxy (25-OH) Vitamin D results
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in 25-OH vitamin D will be evaluated.
Change from Baseline in Serum Creatinine
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in serum creatinine will be evaluated.
Incidence Rate of the Renal Events
时间窗: Baseline up to 10 years (follow-up)
Incidence rate of the renal events will be recorded for nephrolithiasis, nephrocalcinosis, hospitalization/emergency room visits for renal events.
Change from Baseline in 24-Hour Urine Calcium
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in 24-hour urine calcium will be evaluated.
Change from Baseline in Serum Albumin
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in serum albumin will be evaluated.
Change from Baseline in Serum Phosphate
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in serum phosphate will be evaluated
Change from Baseline in Estimated Glomerular Filtration Rate (eGFR; calculated)
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in eGFR; calculated will be evaluated.
Incidence Rate of the Bone Fractures (site)
时间窗: Baseline up to 10 years (follow-up)
Incidence rate of the bone fractures (site) will be recorded.
Change from Baseline in Serum Calcium
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in serum calcium will be evaluated.
Change from Baseline in Serum Ionized Calcium
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in serum ionized calcium will be evaluated.
Change from Baseline in 24-Hour Urine Protein
时间窗: Baseline up to 10 years (follow-up)
Change from Baseline in 24-hour urine protein will be evaluated.
Change from Baseline in Albumin-Corrected Total Calcium
时间窗: Baseline up to 10 years (follow-up)
Change from baseline in albumin-corrected total calcium will be evaluated.
Incidence Rate of the Soft Tissue Calcifications (site)
时间窗: Baseline up to 10 years (follow-up)
Incidence rate of the soft tissue calcifications (site) will be recorded.
Incidence Rate of the Cardiovascular Events
时间窗: Baseline up to 10 years (follow-up)
Incidence rate of the cardiovascular events will be calculated for this clinical outcome. Cardiovascular events include myocardial infarction, stroke, arrhythmia.
Incidence Rate of the Cataract
时间窗: Baseline up to 10 years (follow-up)
Incidence rate of the cataract will be recorded by questionnaire (present/not present).
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Baseline up to 10 years (follow-up)
An AE is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, a new disease or worsening in severity or frequency of a concomitant disease, temporally associated with the use of a medicinal product, whether or not the event is considered causally related to the use of the product. An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event.
次要结局
- Health-related Quality of Life (HRQoL)(Baseline up to 10 years (follow-up))
- Rate of Hospitalization/Emergency Room (ER) Visits(Baseline up to 10 years (follow-up))
- Disease-specific Patient-reported Outcome Measures(Baseline up to 10 years (follow-up))