EUCTR2020-002437-15-HU进行中（未招募）1 期
A PHASE III, MULTICENTER, SINGLE-ARM STUDY EVALUATING THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CROVALIMAB IN PEDIATRIC PATIENTS WITH ATYPICAL HEMOLYTIC UREMIC SYNDROME (aHUS)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 35
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
- •In addition to Naive and Switch patients (described above), Pretreated patients (patients who previously received treatment and then discontinued either eculizumab or ravulizumab and/or with a known C5 single nucleotide polymorphism [SNP]) will be enrolled as a separate third Cohort.
- •For ALL (Naive, Switch, and Pretreated) Cohorts:
- •Age >=28 days and <18 years (at the time of signing the Informed Consent Form or Assent Form)
- •Body weight >=5 kg at screening
- •Vaccination against Neisseria meningitidis < 3 years prior to initiation of study treatment, in accordance with current local guidelines or standard-of-care, as applicable in patients with complement deficiency
- •For female patients of childbearing potential, an agreement to remain abstinent or use contraception
- •Patients with a prior kidney transplant are eligible, if they have a known history of complement-mediated aHUS prior to the kidney transplant
- •For Naive Cohort ONLY:
- •Onset of thrombotic microangiopathy (TMA) <=28 days prior to first crovalimab administration
- •For Switch Cohort ONLY:
- •Documented treatment with either eculizumab or ravulizumab, according to the local product label for aHUS. Patients switching from IV eculizumab must have received treatment for at least 90 days. Patients switching from IV ravulizumab must have received at least two maintenance doses.
- •Clinical evidence of response to either eculizumab or ravulizumab
- •For Pretreated Cohort only:
- •If TMA is present at time of screening: First crovalimab administration <=28 days from onset of TMA recurrence
- •Received and subsequently discontinued treatment with either eculizumab or ravulizumab, for a minimum of 5.5 drug half-lives
- •For C5 SNP patients: Known C5 polymorphism AND documented treatment with either eculizumab or ravulizumab for aHUS, with poorly controlled TMA
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 35
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •For ALL (Naive, Switch, and Pretreated) Cohorts:
- •At TMA onset, diagnosis of thrombotic thrombocytopenic purpura (TTP), diagnosis of Shiga toxin–producing Escherichia coli hemolytic uremic syndrome (STEC-HUS), clinical suspicion of pneumococcal HUS, TMA secondary to cobalamin C defect or TMA related to diacylglycerol kinase-E (DGKE) nephropathy
- •TMA associated with non-aHUS related renal disease
- •Positive direct Coombs test
- •Identified drug exposure-related TMA
- •History of organ transplant, other than kidney transplant
- •Chronic dialysis, and/or end stage renal disease (ESRD)
- •History of a kidney disease other than aHUS, affecting renal function
- •History of Neisseria meningitidis infection within 6 months prior to screening and up to the first drug administration
- •Known or suspected immune deficiency (e.g., history of frequent recurrent infections)
- •Positive human immunodeficiency virus (HIV) test
- •Life expectancy of < 4 weeks
- •Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration
- •Presence of fever (>=38 Degrees Celsius) within 7 days before the first drug administration
- •Patient with active or evolving multisystem organ dysfunction or failure
- •Immunized with a live attenuated vaccine within 1 month before first drug administration
- •Known systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or anti-phospholipid antibody positivity or syndrome
- •Patients receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks prior to start of screening, unless for unrelated medical condition
- •Female patients who are pregnant, breastfeeding, or have the intention of becoming pregnant during the study or within 6 months after the final dose of the study treatment
- •Splenectomy < 6 months prior to screening
- •Use of tranexamic acid within 7 days prior to screening
- •Concurrent disease, treatment, procedure or surgery, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose any additional risk for the patient, or would, in the opinion of the investigator, preclude the patient's safe participation in and completion of the study
- •For Naive Cohort ONLY:
- •Current or previous treatment with a complement inhibitor
- •For Switch Cohort ONLY:
- •Positive for hepatitis B surface antigen (HBsAg) at screening
- •Positive for hepatitis C virus (HCV) antibody at screening
- •History of or ongoing cryoglobulinemia at screening
研究者
相似试验
招募中
3 期
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)Atypical Hemolytic Uremic SyndromeJPRN-jRCT2041210086Mona D. Shah45
进行中（未招募）
1 期
A Study Evaluating the Efficacy and Safety of Crovalimab in Adult and Adolescent Patients with Atypical Hemolytic Uremic Syndrome (aHUS)EUCTR2020-002475-35-HUF. Hoffmann-La Roche Ltd90
进行中（未招募）
1 期
A Study Evaluating the Efficacy and Safety of Crovalimab in Adult and Adolescent Patients with Atypical Hemolytic Uremic Syndrome (aHUS)Atypical Hemolytic Uremic Syndrome (aHUS)MedDRA version: 20.1Level: LLTClassification code 10079841Term: Atypical hemolytic uremic syndromeSystem Organ Class: 100000004851EUCTR2020-002475-35-ITF. HOFFMANN - LA ROCHE LTD.90
进行中（未招募）
3 期
Clinical trial study for atypical hemolytic uremic syndrome in childrens aged more than 28 days to less than 18 years.CTRI/2023/12/060590F HoffmannLa Roche Ltd
进行中（未招募）
1 期
A Study Evaluating the Efficacy and Safety of Crovalimab in Adult and Adolescent Patients with Atypical Hemolytic Uremic Syndrome (aHUS)Atypical Hemolytic Uremic Syndrome (aHUS)MedDRA version: 20.1Level: LLTClassification code 10079841Term: Atypical hemolytic uremic syndromeSystem Organ Class: 100000004851EUCTR2020-002475-35-FRF. Hoffmann-La Roche Ltd90