Randomized, Single-blind, Cross-over Study Investigating the Non-inferiority of Efficacy and Safety of HyQvia in Comparison With Conventional Subcutaneous Ig Therapy in Multifocal Motor Neuropathy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Changes in isometric muscle strength
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of HyQvia (Immunoglobulin 10% with recombinant hyaluronidase) with conventional subcutaneous immunoglobulin treatment in patients with Multifocal Motor Neuropathy (MMN).
详细描述
Subcutaneous immunoglobulin (SCIG) therapy for MMN is equally efficacious to intravenous immunoglobulin (IGIV), may be self-induced and may induce fewer systemic adverse reactions. Limited SC infusion volumes and reduced bioavailability, however, necessitate multiple infusion sites, more frequent treatment, and dose adjustment to achieve pharmacokinetic equivalence. This is an issue in particular in MMN where relatively high and frequent doses are necessary to maintain long-term improvement of muscle strength. Recombinant human hyaluronidase (rHuPH20) increases subcutaneous tissue permeability and facilitates dispersion and absorption, enabling subcutaneous administration of higher (monthly) doses of Ig. If treatment with HyQvia is at least equally effective and safe as compared with conventional Ig treatment, HyQvia could become the preferred treatment option for patients with MMN as it may have attractive benefits for patients by its mode of administration.
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age at onset 18 - 65 years.
- •The presence of asymmetrical limb weakness at onset or motor involvement having a motor nerve distribution in at least two peripheral nerve distributions, predominant upper limb involvement, disabling weakness MRC grade 4 or less in at least one muscle.
- •Decreased or absent tendon reflexes in affected limbs.
- •Electrophysiological evidence of one site with definite motor conduction block or one site with probable conduction block according to previously defined criteria.
- •Response to SCIG according to criteria that were described in previous studies
- •On SCIG maintenance treatment for more than 3 months preceding the study.
- •Patients have given written informed consent, prior to the study, with the understanding that consent may be withdrawn at any time without prejudice.
排除标准
- •Bulbar signs or symptoms.
- •Upper motor neuron signs (spasticity, hyperreflexia, extensor plantar response).
- •Sensory symptoms and signs with sensory deficits on examination (except for vibration sense) and abnormal results of sensory nerve conduction studies
- •Other neuropathies (e.g. diabetic, lead, porphyric or vasculitic neuropathy, chronic inflammatory demyelinating polyneuropathy, Lyme neuroborreliosis, post radiation neuropathy, hereditary neuropathy with liability to pressure palsies, Charcot-Marie-Tooth neuropathies, meningeal carcinomatosis).
- •Treatment with other immunosuppressive drugs (cyclophosphamide, azathioprine, cyclosporin) in the 6 months preceding the study.
- •Female patient who is pregnant or breast-feeding or of childbearing potential.
- •Confirmation that the patient is not pregnant will be established by a negative b-HCG test within a 7-day period before inclusion in the study. Lack of childbearing potential is met by a) being post-menopausal, b) being surgically sterile, c) practising contraception with an oral contraceptive, intra-uterine device, diaphragm or condom with spermicide or d) being sexually inactive.
- •Age < 18 years
研究组 & 干预措施
Group A
24 weeks of treatment with conventional subcutaneous immunoglobulin (Subcuvia) followed by 24 weeks of treatment with HyQvia
干预措施: HyQvia (Drug)
Group A
24 weeks of treatment with conventional subcutaneous immunoglobulin (Subcuvia) followed by 24 weeks of treatment with HyQvia
干预措施: Subcuvia (Drug)
Group B
24 weeks of treatment with HyQvia followed by 24 weeks of treatment with conventional subcutaneous immunoglobulin (Subcuvia)
干预措施: HyQvia (Drug)
Group B
24 weeks of treatment with HyQvia followed by 24 weeks of treatment with conventional subcutaneous immunoglobulin (Subcuvia)
干预措施: Subcuvia (Drug)
结局指标
主要结局
Changes in isometric muscle strength
时间窗: Evaluation at week 0, 12, 24, 36, 48
Measurement of isometric muscle strength of four involved muscle groups
次要结局
- Changes in grip strength(Evaluation at week 0, 12, 24, 36, 48)
- Changes in gait performance(Evaluation at week 0, 12, 24, 36, 48)
- Changes in disability score(Evaluation at week 0, 12, 24, 36, 48)
- Development of Headache and Nausea(During the entire study period)
- Development of hemolytic anemia(Evaluation at week 0, 12, 24, 36, 48)
- Changes in clinical evaluation of muscle strength(Evaluation at week 0, 12, 24, 36, 48)
- Development of antibody against hyaluronidase(Evaluation at week 0, 12, 24, 36, 48)
- Patient satisfaction(Evaluation at week: 6, 12, 18, 24, 30, 36, 42, 48)
- Changes in hand/finger function(Evaluation at week 0, 12, 24, 36, 48)
研究者
Johannes Jakobsen
Professor, DMSc
Rigshospitalet, Denmark