NCT03831152撤回3 期
A Phase 3B Open-Label Extension Of Study B23CS (ARENA 2) Evaluating The Continued Safety And Efficacy Of ADV7103 In Subjects With Primary Distal Renal Tubular Acidosis
Advicenne Pharma1 个研究点 分布在 1 个国家开始时间: 2023年12月20日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- The Safety of ADV7103 will be assessed by evaluating the frequency of Treatment-Emergent Adverse events as compared to placebo
研究概览
简要总结
Open-label study involving longitudinal assessment of the continued safety, tolerability, and efficacy of ADV7103 in maintaining targeted serum bicarbonate levels, preventing metabolic acidosis, and preventing hypokalemia in the following groups of subjects with primary dRTA:
- subjects who participated in Study B23CS and were adherent to the protocol;
- subjects ≥ 6 months of age who are living in Europe and did not participate in Study B23CS; and
- infants younger than 6 months of age
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
- 4 Months 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who participated in Study B23CS must meet all of the following criteria to be registered into this study:
- •1. Female or male subjects who participated in Study B23CS and were adherent to the protocol;
- •Subjects who did not participate in Study B23CS must meet all of the following criteria to be registered into this study:
- •Female or male subjects ≤ 65 years of age at time of consent with the following stipulations:
- •Subjects ≥ 6 months of age must reside in Europe
- •Subjects < 6 months of age must reside in the United States, Canada, or Europe;
- •Subjects < 6 months of age must be able to swallow (not suck) solid food without difficulty;
- •Subject presents with a previous diagnosis of primary dRTA;
- •Subject ≥ 6 months of age requires ≥ 0.9 mEq/kg/day of alkali therapy to maintain serum bicarbonate levels above the LLN;
- •Urine pH > 5.5 and serum bicarbonate > 18 mEq/L for subjects ≥ 4 years old or > 17 mEq/L for subjects < 4 years old on alkali therapy and potassium supplementation (if indicated) on at least one occasion for each within 6 months;
- •European subjects must be included in a social health insurance system and/or in compliance with the recommendations of the national law in force relating to biomedical research;
- •Subject or parent/guardian is willing and able to understand and sign informed consent and willing to comply with protocol instructions; child assent when appropriate; and
排除标准
- •Subjects who participated in Study B23CS and meet any of the following criteria will be considered ineligible for registration into this study:
- •Female subject who is pregnant or lactating or has plans for pregnancy during the study; or
- •Subject requires therapy with potassium sparing diuretics, angiotensin converting enzyme inhibitors, angiotensin II receptor blockers, trimethoprim, drospirenone and other progestins, nephrotoxic antibiotics, penicillins, tacrolimus, or medications known to delay gastric emptying or otherwise interfere with absorption of study product.
- •Subjects who did not participate in Study B23CS and meet any of the following criteria will be considered ineligible for registration into this study:
- •Female subject who is pregnant or lactating or has plans for pregnancy during the study;
- •Subject has evidence of proximal tubule dysfunction unless the subject is < 6 months of age;
- •Subject presents with another diagnosed condition as a potential etiology for her/his dRTA ;
- •Subject requires therapy with potassium sparing diuretics, angiotensin converting enzyme inhibitors, angiotensin II receptor blockers, trimethoprim, drospirenone and other progestins, nephrotoxic antibiotics, penicillins, tacrolimus, or medications known to delay gastric emptying or otherwise interfere with absorption of study product;
- •Subject has evidence of obstructive uropathy or other findings on renal ultrasound expected to require intervention during the course of the study;
- •Subject has any of the following laboratory abnormalities:
- •AST and/or ALT > 1.5x upper limit of normal (ULN)
- •Serum potassium > 5.0 mEq/L or hypokalemia accompanied by clinical symptoms (eg, muscle cramps) or significant ECG changes (eg, T wave depression, U wave elevation)
- •Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 (according to the updated Schwartz formula for children and Chronic Kidney Disease - Epidemiology Collaboration [CKD-EPI] formula for adults)
- •Total bilirubin > ULN, except with known Gilbert's disease.
- •Subject has been hospitalized or had outpatient surgery (other than minor skin and dRTA disease-related procedures or ear tube placement) in the past 6 months or is planning surgery in the next 6 months.
研究组 & 干预措施
Experimental ADV7103
Experimental
All patients receive ADV7103 at their individualized dose
干预措施: ADV7103 (Drug)
结局指标
主要结局
The Safety of ADV7103 will be assessed by evaluating the frequency of Treatment-Emergent Adverse events as compared to placebo
时间窗: To 30 months
Number/proportion of subjects presenting with ADV7103 treatment-related adverse events (AEs) during the study, by severity grade.
次要结局
未报告次要终点
研究者
研究点 (1)
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