A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The change in Headache Impact Scale (HIT-6)
研究概览
简要总结
Patients receiving intravenous immunoglobulin (IVIG) therapy for primary immunodeficiency and neurologic conditions may experience adverse drug reactions (ADRs). The mechanism of the ADR is unknown. Currently, the standard practice for these patients is to change from IV to subcutaneous IG (SCIG) but because of the need of immunomodulation or patient preference, SCIG may not be an option. Data has shown that some levels of complement decrease from pre- to post-infusion of IVIG. This study is to determine if replacing this complement protein may ameliorate ADRs.
详细描述
This is a single-site, pilot study conducted in the US to determine the benefit of human C1-esterase inhibitor [recombinant] (C1-INH-R) therapy to ameliorate ADRs in subjects receiving IVIG therapy experience ADRs post-infusion. Subjects who are currently receiving IVIG for immunodeficiency or neurologic conditions and experience ADRs will be enrolled.
In part 1, subjects will continue to receive IVIG for two infusions. Complement proteins will be measured pre- and post-infusion. Quality of life and other questionnaires will be administered.
In part 2, subjects will receive C1-INH-R infusion prior to receiving IVIG infusion for three infusions. Complement proteins will be measured pre- and post- infusion. Quality of life and other questionnaires will be administered.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
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
- •Age 18 years and older experiencing ADRs related to IVIG infusions
- •Stable dose of IVIG for 3 months
- •Willing to comply with all aspects of the protocol, including blood draws
- •Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception. Acceptable forms of contraception are defined as those with a failure rate < 1% when properly applied and include: a combination oral pill, some intra-uterine devices, and a sterilized partner in a stable relationship. Female patients must not be pregnant, planning to become pregnant, or be actively breastfeeding through the entire study period.
排除标准
- •Receiving treatment for HAE, either prophylactic or acute therapy
- •Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
- •Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant.
- •Patients who, in the investigator's opinion, might not be suitable for the trial
研究组 & 干预措施
C1-esterase inhibitor [recombinant] (C1-INH-R)
Single-site, open-label arm to evaluate the benefit of C1-INH-R in subjects on IVIG therapy who experience ADRs. The study will have 2 periods:
- 6 - 8 weeks - subjects will receive 2 infusions of IVIG
- 9 - 12 weeks - subjects will receive 3 infusions of C1-INH-R prior to IVIG infusion
干预措施: C1-esterase inhibitor [recombinant] (C1-INH-R) (Biological)
结局指标
主要结局
The change in Headache Impact Scale (HIT-6)
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated measurement of the impact headaches have on the ability to function on the job, at school, at home and in social situations. 6 item questionnaire rated from never (6 points each), rarely (8 points each), sometimes (10 points each), very often (11 points each), always (13 points each). The higher number the more the impact.
The change in Modified Fatigue Impact Scale (MFIS)
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated 24 item questionnaire measuring the impact of fatigue rated 0 - 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always)
The change in Migraine Disability Assessment (MIDAS)
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Headache severity measurement of number of days affected after infusion
The change in Energy Sliding Scale
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated10 point scale measuring energy level with 1 being the worst (Lowest) and 10 being the best (highest)
The change in Perceived Deficits Questionnaire - cognitive assessment
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated 20 item questionnaire measuring memory, attention and concentration rated 0 to 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always) with the lower score being least impact.
The change in Modified Fatigue Severity Scale (mFSS)
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated Rasch-built 7-item modified fatigue survey scale. Scale rated 0 - 3 (3 = agree, 0 = less agree)
The change in Activities of Daily Living Sliding Scale
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated 10 point scale measuring level of activity with 1 being the worst (least) and 10 being the best (greatest)
The change in Activities of Daily Living Questionnaire
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Calculates the number of days missed from work, school/daycare/activities, housework, and regular exercise
The change in Infection Questionnaire
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Subject-rated 7 item infection questionnaire measured from 1 to 10 with 1 being the least affected and 10 being the most affected.
The change in 36 item short form survey (SF-36)
时间窗: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Health Survey asks 36 questions to measure functional health and well-being from the patient's point of view.
Change in the number of ADRs
时间窗: Measured at each infusion (every 3 - 4 weeks)
Adverse reactions to infusions
次要结局
- Change in levels of C1-INH pre- and post-infusion(Measurement at each infusion (every 3 - 4 weeks))