Efgartigimod for Stiff Person Syndrome (ESPS)
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Distribution of Stiffness Index (DSI)
研究概览
简要总结
The goal of this clinical trial is to examine the proportion of patients with stiff person syndrome with no worsening in stiffness and/or spasms while on efgartigimod-hyaluronidase (EFD) when compared to their current treatment. The main questions it aims to answer are:
- proportion of patients with stiff person syndrome with no worsening in DSI and HSS scores
- whether there are changes in laboratory values, vital signs, electrocardiogram, or depression, and suicide risk during the trial, as well as the incidence of any adverse events.
Participants will come to the clinic up to 18 times and have up to 5 phone call visits during the course of the trial. During these visits a variety of measures will be collected, including blood samples, neurological and physical exams, patient reported outcomes on stiffness, spasms, depression, and suicide risk.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18-80, men and women, capable of giving informed consent.
- •Must have unexplained stiffness of limb and axial (trunk) muscles and involuntary striated muscle spasms.
- •DSI and HHS must be >=2 (to allow for detection of improvement).
- •Presence of serum (or CSF) anti-GAD antibodies regardless of titers.
- •Stable dosage for at least two months before screening visit with antispasmodics (muscle relaxants, benzodiazepines, baclofen).
- •Women with children bearing potential must be willing to use highly effective contraception method during and for a minimum of 90 days after the last dose of IP.
排除标准
- •Exclusionary conditions:
- •1a. type 1 DM
- •1b. Cancer, except basal cell skin cancer
- •1c. Infections: HIV, Hepatitis B or C, clinically significant active infection not sufficiently resolved in the investigator's opinion
- •1d. Serum IgG (<6 g/L) at screening
- •1e. pregnancy/breast feeding.
- •Exclusionary medications: rituximab within 6 months of screening.
- •IVIG or PLEX treatment 4 weeks prior to screening.
- •History of any type of solid organ or bone marrow transplant.
研究组 & 干预措施
subcutaneous Efgartigimod-hyaluronidase (EFG)
Participants with GAD65-antibody positive SPS will be given subcutaneous Efgartigimod-hyaluronidase (EFG).
干预措施: Efgartigimod (Biological)
结局指标
主要结局
Distribution of Stiffness Index (DSI)
时间窗: From enrollment to the end of treatment at week 13
DSI is a validated indicator or stiffness. Scores range from 0 to 6 and reflect the extent of stiffness. One point is given for stiffness in each of the following areas: lower trunk, upper trunk, legs, arms, face and abdomen.
heightened sensitivity score (HSS)
时间窗: From enrollment to end of treatment at week 13
HSS measures changes in the frequency of spasms. Scores range from 1 to 7; one point given for each source or type of spasm: unexpected noises, visual stimuli, somatosensory stimuli, voluntary activities, emotional upset or stress, no specific stimuli, or nocturnal spasms.
次要结局
- Proportion of patients with SPS who are treatment responders(enrollment to end of 13 weeks)
- Modified Rankin Scale(From enrollment to end of treatment at week 13)
- The duration of time patients maintain responder status(from enrollment to end of treatment at week 13)