NCT05462587招募中3 期
A Phase 3, Randomized, Double-blind, Two-period, Crossover, Withdrawal Study to Assess the Efficacy and Safety of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
AUG Therapeutics2 个研究点 分布在 1 个国家目标入组 4 人开始时间: 2022年7月28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
- 招募中
- 发起方
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen
研究概览
简要总结
The primary objective of this study is to evaluate the efficacy and safety of AVTX-803 compared to withdrawal in patients with Leukocyte Adhesion Deficiency, Type II (LAD II).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 6 Months 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must be between 6 months and 75 years old
- •Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
- •Subject has a documented history of Lewis antigen deficiency
- •Subject has a history of recurrent infections, opportunistic infections or infections that did not respond well to standard of care treatment
- •Subject or parent (for subjects under legal age for consent) has provided written informed consent for this study. Additionally, written informed assent has been provided, as appropriate, for minors of older age, per local institutional review board (IRB)/ethics committee (EC) policy and requirements
- •Subject is willing and able to comply with the protocol
- •Women of childbearing potential (WOCBP) meeting the criteria below:
- •Non-lactating and has a negative pregnancy test at screening -AND-
- •Uses an acceptable double-barrier method of contraception as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.
- •Male subjects must agree to use an acceptable double-barrier method of contraception with their partner as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.
排除标准
- •Subject has severe anemia defined as hemoglobin <8.0 g/dL (<4.9 mmol/L)
- •Subject has impaired renal function as defined by an eGFR <90 mL/min
- •Subject has a total absence of fucosylation on red blood cells and the presence of anti-H antigen
- •Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product
- •In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing
- •In the investigator's opinion, subject is not able or not willing to comply with the study requirements.
- •Subject is pregnant
研究组 & 干预措施
Withdrawal
No Intervention
Subject will be in withdrawal for 8 weeks.
AVTX-803
Experimental
Approximately 4 subjects will receive AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day for 8 weeks.
干预措施: AVTX-803 (L-Fucose) (Drug)
结局指标
主要结局
Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen
时间窗: Change from Baseline at Day 56, Change from Baseline at Day 112
To assess the effect of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II) by evaluating Sialyl-Lewis X on leukocytes as a percentage present at the end of each 8 week treatment period.
次要结局
未报告次要终点
研究者
研究点 (2)
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