NL-OMON49059已完成不适用
First in Human Study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of PPSGG (PN-1007) in anti-MAG neuropathy patients. - PN-1007-001
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
研究概览
简要总结
Trial ended prematurely
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •- Written informed consent.
- •- Age between 18 and 80 years, male and female.
- •- Patient with a confirmed diagnosis of monoclonal IgM associated with
- •monoclonal gammopathy of undetermined significance (MGUS) with anti-MAG
- •activity (titer of > 10*000 Bühlmann Titer units [BTU]) and demyelinating
- •neuropathy defined by electrophysiological criteria according to European
- •Federation of Neurological Societies/Peripheral Nervous System paraproteinemic
- •demyelinating neuropathy (EFNS/PNS PDN) guideline, 2010.
- •- Clear clinical signs of disability: with at least ONLS * 2 in lower
- •extremities.
- •- Inflammatory Neuropathy Cause and Treatment sensory sum score (ISS) * 2.
- •- Patients must have adequate hepatic function as evidenced by total bilirubin
- •< 26 µmol/l (1.5 mg/dL), and alkaline phosphatase and aspartate
- •transaminase/alanine aminotransferase < 2X the upper limit of normal (ULN).
- •- Absence of cause of neuropathy independent from anti- MAG activity: e.g.
- •diabetes, hypothyroidism, past or current dependence on alcohol, past or
- •current treatment with neurotoxic drugs.
- •- Patients must have adequate renal function as evidenced by serum creatinine
- •<2 mg/dL or calculated creatinine clearance of *60 mL/min within 28 days before
- •the first investigational medicinal product (IMP) administration using the
- •Modification of Diet in Renal Disease (MDRD) formula.
- •- Capability to meet the requirements of the study.
排除标准
- •- Patients with total serum IgM levels >30 g.
- •- Hematological malignancy (e.g. known multiple myeloma or confirmed
- •Waldenström's macroglobulinemia based on bone marrow analysis).
- •- Patients with any history of malignancy of any organ system (other than
- •localized basal cell carcinoma of the skin), treated or untreated, within the
- •past 5 years, regardless of whether there is evidence of local recurrence or
- •metastases.
- •- Previous immunosuppressive treatment with intravenous immunoglobulin (IVIG)
- •or apheresis/plasmapheresis in the preceeding 3 months, and/or cyclophosphamide
- •and biologicals (e.g. rituximab): in the preceeding 6 months prior to enrolment.
- •- Other neurological, neuromuscular, rheumatologic or orthopedic conditions
- •with significant impact on the capability of walking preventing evaluation of
- •neurological scores.
- •- Anti-MAG neuropathy patients with persistent clinically significant
- •laboratory abnormalities not related to the anti-MAG neuropathy, such as
- •significant renal dysfunction, hepatic dysfunction, cardiac disease or other
- •significant neurological disorder.
- •- Anti-MAG neuropathy patients with a modified Rankin Scale (mRS) score > 4.
- •- Participation in another interventional clinical trial.
- •- Any other significant finding that would increase, according to the
- •Investigator, the risk of having an adverse outcome from participating in the
- •- Any other medical condition, including mental illness or substance abuse
- •deemed by the investigator(s) to likely interfere with the patient's ability to
- •sign informed consent, cooperate and participate in the study, or interfere
- •with the interpretation of the results.
- •- Patients who have undergone major surgery * 2 weeks prior to starting study
- •drug or who have not recovered from the side-effects of surgery.
- •- A history of clinically significant ECG abnormalities, or any of the
- •following ECG abnormalities at screening:
- •PR > 200 msec.; QRS complex > 120 msec.; QTcF > 450 msec (males); QTcF > 460
- •msec (females); History of familial long QT syndrome or known family history of
- •Torsades de Pointes; Use of agents known to prolong the QT interval unless they
- •can be permanently discontinued for the duration of the study.
- •- Sexually active males must use a condom during intercourse after the start of
- •the IMP administration and for at least one week after stopping study
- •medication and should not father a child in this period after completion of the
- •study medication (SAD and MAD phases). A condom is required to be used also by
- •vasectomized men in order to prevent delivery of the drug via seminal fluid. In
- •addition, male participants should not donate sperm for the time period
- •specified above.
- •- Use of other investigational drugs at the time of enrolment, or within 5
- •half-lives of enrolment, or within 30 days, whichever is longer; or longer if
- •required by local regulations.
- •- Women of child-bearing potential, defined as all women physiologically
- •capable of becoming pregnant, unless they are using highly effective methods of
- •contraception during dosing and for 1 week after discontinuation of the
- •investigational drug.
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