Subcutaneous Immunoglobulin With rHuPH20 in Multifocal Motor Neuropathy
试验速览
- 阶段
- 不适用
- 发起方
- UMC Utrecht
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Safety measured by anamnesis
研究概览
简要总结
Subcutaneous immunoglobulin (SCIG) and recombinant human hyaluronidase (rHuPH20) will be tested in patient witch MMN and currently under maintenance treatment with IVIG for safety, tolerability and efficacy.
详细描述
Multifocal motor neuropathy (MMN) is a chronic immune mediated neuropathy, which affects patients at a relatively young age and necessitates treatment with immunoglobulins (Ig) to improve and maintain muscle strength. Subcutaneous immunoglobulin (SCIG) therapy for MMN is equally efficacious to intravenous immunoglobulin (IVIG), may be self-administered and may induce fewer systemic adverse reactions. However, limited subcutaneous infusion volumes and reduced bioavailability, 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.
Objective:
The primary objective is to study the safety and tolerability of HyQvia in patients with
MMN as compared with conventional intravenous, by documenting:
- Side effects by using a diary and a 10 cm visual analogue scale (VAS) scales to measure headache and nausea.
- Biochemical tests (Hemoglobin, haptoglobin, reticulocyte counts, lactate dehydrogenase, bilirubin, DAT) for haemolytic anemia 3-7 days after the last IVIG/SCIG infusion, during the enrolment period and 3-7 days after the last HyQvia infusion during the treatment period.
- Anti- hyaluronidase antibody titer before and after/during the HyQvia treatment. Together with the above mentioned test parameters in point 2, each time 30ml (equal to three tubes) will be drawn.
- Patient satisfaction of product on a 10 cm VAS scale.
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 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age at onset of MMN, 18 - 99 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 IVIG according to criteria that were described in previous studies.
- •Stable on IVIG maintenance treatment in the year 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:
- •being post-menopausal,
- •being surgically sterile,
- •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
HyQvia
human immunoglobulin and one vial of recombinant human hyaluronidase (rHuPH20
干预措施: rHuPH20 (Drug)
结局指标
主要结局
Safety measured by anamnesis
时间窗: 1 year
presence of headache, gastrointestinal complaints, fatigue, flue-like symptoms
次要结局
- Stability of muscle strength(1 year)
- Safety measured by questionnaire(1 year)
- Quality of life (QoL) measured by a VAS questionnaire(1 year)
- Safety measured by blood test(1 year)