Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 14
- 主要终点
- Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement
研究概览
简要总结
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi. Data on safety and on measures of efficacy are collected.
详细描述
Patients with ADA-SCID who require ERT will receive Revcovi on a dosage and schedule determined by the treating physician. They will be followed for safety throughout the study, and will be monitored for the efficacy markers of adenosine deaminase (ADA) activity and deoxyadenosine nucleotide (dAXP) concentration according to a suggested schedule. Some subjects will be new to ERT; some will have transitioned from Adagen, which was the ERT available before Revcovi; and some will have previously participated in an earlier Phase 3 trial of Revcovi (study STP-2279-002). Patients will be followed either until they are able to successfully undergo a stem cell transplant or stem cell gene therapy and thus no longer require ERT treatment, or until all ongoing participants have received a minimum of 24 months of Revcovi treatment.
Note: Due to the nature of this study, all analyses are descriptive and no statistical hypotheses will be tested.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 0 Months 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, aged newborn to adult
- •In need of ERT treatment due to one of the following circumstances:
- •Waiting to receive a stem cell transplant
- •Previously declined, had been found to be ineligible for, or did not respond to a stem cell transplant
- •Waiting to enrol in a clinical trial on gene therapy, or had been found to be ineligible for or had failed such a trial
- •One of the following histories of ERT treatment:
- •Revcovi only
- •Previously on Adagen but had transitioned to Revcovi
- •Not yet on any ERT but about to start on Revcovi
排除标准
- •Any condition that, in the opinion of the Investigator, makes the patient unsuitable for the study.
研究组 & 干预措施
Patients with ADA-SCID in need of ERT treatment
All participants will receive Revcovi. For analysis purposes, there will be three groups, who differ with respect to ERT history:
- Adagen-naïve: Subjects starting on ERT for the first time
- Adagen-transitioning: Subjects switching from Adagen to Revcovi
- STP-2279-002 participants: Subjects who had taken part in an earlier Phase III trial of Revcovi (study STP-2279-002) in which they had been switched from Adagen to Revcovi. This last group differs from the Adagen-transitioning group in that they will have been on Revcovi the longest.
干预措施: elapegademase-lvlr (Biological)
结局指标
主要结局
Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement
时间窗: From enrollment to end of treatment up to Month 24
A deficiency in the ADA enzyme results in a build-up of dAXP nucleotides, which are toxic to lymphocytes and lead to impairment of immune function. A detoxified level of dAXP concentration is defined as a trough value of 0.02 millimoles per liter (mmol/L) or lower. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Number of Subjects Meeting the Optimal Threshold for ADA Activity at the Last Measurement
时间窗: From enrollment to end of treatment up to Month 24
An optimal level of ADA plasma activity is defined as a trough value of 30 millimoles per hour per liter (mmol/h/L) or greater. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Safety of Revcovi
时间窗: From enrollment to end of treatment up to Month 24
The number of subjects reporting adverse events (AEs). Due to the nature of ADA-SCID, only AEs deemed to be at least possibly related to Revcovi treatment were documented.
次要结局
未报告次要终点