EUCTR2014-004972-49-FR进行中（未招募）1 期
A randomized, open label, controlled, multiple dose studyto evaluate the clinical efficacy, safety, tolerability,pharmacokinetics and pharmacodynamics of LFG316 inpatients with transplant associated microangiopathy afterhematopoietic precursor cell transplantation - CLFG316X2202
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Written informed consent/assent before any study-specific screening procedures.
- •For pediatric patients, consent will be obtained from parent(s) or legal guardian(s) and the
- •signature of at least 1 parent or guardian will be required. Investigators will also obtain
- •assent of patients according to local, regional or national guidelines.
- •2. Male and female TAM patients = 2 years old at the time of first dose administration.
- •Patients < 12 years old can only be included in the study after first IA has shown that it is
- •safe and well tolerated in patients = 12 years old (Section 3.5).
- •3. The presence of TAM as per below diagnostic criteria at baseline (or screening if baseline visit is skipped). All the criteria have to be met for the patients included in the study: ? Elevated lactate dehydrogenase (any elevation above normal range)
- •? Thrombocytopenia with platelet count < 50x10e9/L or greater than >50% decrease in
- •platelet count from the highest value achieved after transplant
- •? Anemia below lower limit of normal or anemia requiring transfusion support as per
- •center standard
- •? Schistocytes on peripheral blood smear (>2 per HPF) OR histologic evidence of
- •microangiopathy
- •? Absence of coagulopathy (no uncompensated disseminated intravascular coagulation, DIC) at screening
- •4. The presence of TAM high risk features at baseline (or screening if baseline visit is
- •skipped): Patients = 16 years must have a Lansky score of = 70 and patients > 16 must
- •have Karnofsky score = 70% and/or proteinuria (> 30 mg/dL) measured in two urine spot analyses.
- •5. Hypertension, defined for adults by SBP = 160 mmHg and DBP = 100 mmHg at baseline
- •(or screening if baseline visit is skipped), and for pediatric patients by blood pressure
- •greater than the 95th percentile for age, sex, and height (see Table 16-1). Additionally,
- •patients who were started on antihypertensive medication after HSCT or who have received additional antihypertensive medication after HSCT will be eligible, even if they don’t have elevated blood pressure
- •6. Meningococcal vaccine(s) prior to LFG316 treatment if prior vaccination cannot be
- •confirmed. The choice of vaccine(s) should take into account the serotypes prevalent in
- •the geographic areas in which study patients will be enrolled.
- •7. Whenever possible, patients <18 years old should receive vaccination for the prevention
- •of S. pneumoniae and H. influenzae type b prior to LFG316 administration
- •8. Weight of at least 10kg.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 30
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Use of other investigational drugs at the time of enrollment, or within 5 half-lives of
- •enrollment, or until the expected PD effect has returned to baseline, whichever is longer;
- •or even longer if required by local regulations.
- •2. History of hypersensitivity to study drug or to drugs of similar chemical classes.
- •3. Patients with steroid refractory graft versus host disease (SRGvHD) (progression
- •(=increase in overall grade) after 5 days on =1mg/kg methylprednisolone or equivalent OR no improvement (no decrease in overall grade) after 10 days on = 1mg/kg
- •methylprednisolone or equivalent).
- •4. Patients with ALT > 5x ULN.
- •5. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a
- •female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (at screening or baseline).
- •6. 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 45 days after stopping study medication.
- •7. Positive HIV (ELISA and Western blot) test result (checked at screening).
- •8. A positive Hepatitis B surface antigen or Hepatitis C test result at screening.
- •9. Patients with any severe, progressive or uncontrolled acute or chronic medical condition (such as uncontrolled infectious disease or sepsis) or clinical laboratory abnormalities that in the investigator’s opinion would make the patient inappropriate for entry into this study (at screening or baseline).
- •10. Patients with proven TTP as per historical data (as defined by ADAMST13 activity test)
- •and if already available results of ADAMST13 test done at screening.
- •11. Patients previously treated with eculizumab for TAM.
- •12. Patents with known or suspected hereditary complement pathway deficiency
研究者
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