Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome
试验速览
- 阶段
- 不适用
- 入组人数
- 22
- 主要终点
- A >50% change from baseline on the Heightened Sensitivity scores (scales from 1-7, each item adds one) after 12 weeks of treatment.
研究概览
简要总结
This is a pilot, proof-of concept investigator-initiated trial planned for 22 patients with the diagnosis of Stiff Person Syndrome (SPS). The study will compare efficacy of treatment using subcutaneous immunoglobulin therapy (SCIg) compared to intravenous immunoglobulin (IVIg) therapy. The majority of IVIg naïve subjects (those not already receiving IVIg) are typically managed with non-immunotherapy mostly Gamma Aminobutyric Acid (GABA) -enhancing drugs such as Baclofen or Diazepam.
详细描述
Study Design:This is a proof of concept observational prospective, open label, study on the safety, efficacy and convenience of treatment with SCIg study of 22 patients at Thomas Jefferson University Hospital. Two cohorts of patients within the total of 22 will be included; half of them (11 patients) currently receiving and responding to IVIg and the other half starting de novo on SCIg. Patients diagnosed with SPS according to defined sets of symptoms will be eligible to enroll.
The primary clinical outcome will be based on clinical efficacy measures, as used before for the IVIg trial, based on changes in the Stiffness Index and Heightened Sensitivity scores, using the validated scales that the investigators have had previously utilized and validated (Dalakas et al 2001; see attached at the end of the protocol). These same measurements will be applied while on IVIg (weeks 0, 4, 8, 12) and will be compared to the measurements obtained during SCIg (weeks 16, 20, 24, 28). The secondary outcome will be Quality of Life (QoL) responses and patient preference for each treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men or women aged >18 years
- •Diagnosis of SPS based on standard criteria
- •IVIg Group: Receiving the equivalent of 1-2 g/kg IVIg every 4 weeks with dependence* on IVIg to maintain clinical response *Dependence is clinically determined either by symptomatic worsening of condition at the end of the inter-dose interval or by worsening after dose reduction or discontinuation within the previous 3 months.
- •IVIg-Naïve Group: Patients with symptomatic SPS and never treated with IVIg (IVIg-naïve group), poorly controlled with standard therapy
排除标准
- •Pregnancy, planned pregnancy, breast-feeding or unwillingness to practice contraception
- •Severe concurrent medical conditions, which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
- •Initiation of immunomodulatory treatment other than IVIg in the past 3 months
- •Participation in a trial of an investigational medicinal product in the past 12 weeks
- •Presence of any medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study.
结局指标
主要结局
A >50% change from baseline on the Heightened Sensitivity scores (scales from 1-7, each item adds one) after 12 weeks of treatment.
时间窗: 24 months
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
A >50% change from baseline on the Stiffness Index scores ( scale from 0-6; each item adds one) after 12 weeks of treatment.
时间窗: 24 MONTHS
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
次要结局
- A meaningful change on Quality of Life (QoL) measures after 12 weeks of SCIg based on 6 sets of QoL Questionnaires (mobility, self-care, usual activities, pain, anxiety/depression, health state)(24 MONTHS)
- If SCIg reduces the anti-GAD antibody titers measured in the patient's blood samples by more than 30%(24 MONTHS)
- If >50% of patients prefer either SCIg or IVIg after 12 weeks of treatment based on a YES/NO questionnaire .(24 MONTHS)