EUCTR2017-001082-24-SE进行中（未招募）1 期
A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
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
入选标准
- •1. Willing to provide written informed consent and to comply with the study requirements
- •2. Age 18 years or older
- •3. Clinical presentation consistent with a diagnosis of primary aHUS
- •4. Clinical thrombotic microangiopathy (TMA) activity as defined by:
- •a. Evidence of low platelet count and
- •b. Evidence of hemolysis and
- •c. Evidence of impaired renal function
- •5. ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) >10% (at pre-Screening, Screening, or historically documented in their chart) in a plasma sample that was obtained prior to start of plasma therapy
- •a. Patients who have already started plasma therapy and who do not have a documented pre-plasma ADAMTS13 result will not be eligible for the study unless they are proven to have a documented aHUS-associated mutation.
- •6. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
- •7. Vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations as well as prophylactic antibiotic treatment for at least 2 weeks after completing the recommended vaccination series or longer, if required by local standard of care.
- •8. Decision by an independent specialist physician not to treat this patient with eculizumab. The specialist must be independent from this clinical study, have relevant expertise, and document this decision in the patient’s medical records.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 22
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Abnormal liver function tests
- •2. Positive Shiga toxin producing Escherichia coli test
- •3. Suspected secondary aHUS, in the opinion of the investigator, unless the patient has a documented aHUS-associated genetic mutation.
- •4. Positive direct Coombs test
- •5. Prior eculizumab exposure within 3 months
- •6. Patients considered non-eculizumab responders. except for those with known C5 polymorphisms
- •7. Patients who have received hemodialysis for >3 months
- •8. Patients who require hemodialysis or plasma exchange more frequently than every 24 hours
- •9. Clinical laboratory test results or concomitant conditions considered clinically relevant and unacceptable in the opinion of the Investigator
- •10. Malignancy (except for non-melanoma skin cancers, cervical in-situ carcinoma, breast
- •ductal carcinoma in situ, or stage 1 prostate carcinoma) within the last 5 years
- •11. Patients with active systemic bacterial or fungal infections, as demonstrated by a positive culture result, that require systemic treatment with antibiotics or antifungals
- •a. Patients receiving empiric or prophylactic antibiotics are not excluded
- •12. Patients with sepsis or a life-threatening infection
- •13. Received an investigational agent within the last 90 days or are in follow-up of another clinical study before the first ALN-CC5 administration
- •14. Active psychiatric disorder, including, but not limited to, schizophrenia, bipolar disorder, or severe depression requiring current pharmacological intervention
- •15. Patients with aphasia (verbal Glasgow Coma Scale [GCS] score <5 and National
- •Institutes of Health Stroke Scale [NIHSS] score >0 in item 9)
- •16. Patients with a reduced level of consciousness (GCS score <14, Restless Leg Syndrome score >1, NIHSS total score =1 in item 1)
- •17. Bone marrow transplant recipients
- •18. Organ transplant recipients will be excluded, except for kidney transplant recipients with primary aHUS
- •a. For kidney transplant recipients, primary aHUS will be defined by known genetic mutation and no confounding diagnosis (eg, antibody-mediated rejection or viral infection) by routine kidney biopsy, as judged by the Investigator
研究者
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