EUCTR2019-001346-17-IT进行中（未招募）1 期
ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) - ILLUMINATE-C
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
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
- •Cohorts A and B
- •1. Have reached at least 37 weeks estimated gestational age (full-term infant) at consent (or assent).
- •2. Documentation or confirmation of PH1 as determined by genetic analysis prior to initial dosing.
- •3. Patients with eGFR <=45 mL/min/1.73 m2 as calculated by the MDRD formula if >=18 years or Schwartz Bedside Formula if >=12 months to <18 years, or patients <12 months of age with serum creatinine that is considered elevated for age at consent.
- •4. The mean of the three most recent screening plasma oxalate samples collected prior to Day 1 is >=20 µmol/L. In Cohort B, these 3 collections may include the first pre-dialysis sample from each plasma oxalate profile.
- •5. If taking therapeutic vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days before consent, and is able and willing to remain on this stable regimen until at least the Month 6 visit. Dose adjustments for interval weight gain are acceptable.
- •6. Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
- •Cohort B Only
- •1. On a stable hemodialysis regimen for at least 4 weeks prior to Screening plasma oxalate assessment; able and willing to maintain this regimen through Month 6 visit, with changes to dialysis regimen permitted only when medically indicated.
- •Are the trial subjects under 18? yes
- •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 14
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Has any of the following laboratory parameter assessments at Screening:
- •a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× ULN for age
- •b. Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is <2× ULN
- •c. International normalized ratio (INR) >1.5 for patients not on anticoagulants. Patients on oral anticoagulants (eg, warfarin) with an INR <3.5 will be allowed.
- •d. Hemoglobin <8.0 dL
- •2. Has known active human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B (HBV) infection.
- •3. Received an investigational agent within the last 30 days or 5 halflives, whichever is longer, prior to the first dose of study drug, or are in follow-up of another clinical study during Screening. Consultation with the Medical Monitor is required if treated with unapproved products prior to consent regardless of the time interval prior to the first dose of study drug.
- •4. Known history of allergic reaction to an oligonucleotide or GalNAc.
- •5. Diagnosis of conditions other than PH1 contributing to renal insufficiency such as glomerulonephritis, nephrotic syndrome, or lupus nephritis.
- •6. Any condition or comorbidity, which in the opinion of the Investigator, would make the patient unsuitable for dosing or would interfere with study compliance, data interpretation, patient safety, and/or patient participation in the study. This includes significant active and poorly controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders unrelated to PH1 identified by key laboratory abnormalities or medical history.
- •7. Unwilling or unable to limit alcohol consumption throughout the course of the study. Alcohol intake of >2 units/day is excluded during the study (unit: 1 glass of wine [approximately 125 mL] = 1 measure of spirits [approximately 1 fluid ounce] = ½ pint of beer [approximately 284 mL].
- •8. History of alcohol abuse, within the last 12 months before screening, in the opinion of the investigator.
- •9. Patients with a previous liver transplant or for whom a liver transplant is anticipated in the next 6 months.
- •10. Patients with a previous kidney transplant who are currently receiving immunosuppression to prevent transplant rejection.
- •11. Patients maintained on a peritoneal dialysis regimen.
- •12. Patients who in the opinion of the Investigator plan to start dialysis replacement therapy in the next 6 months.
- •13. Any comorbidity or condition that, in the opinion of the Investigator, is anticipated to prevent participation in at least 12 months of the study
- •14. Is not willing to comply with the contraceptive requirements during the study period, as described in study protocol.
- •15. Female patient is pregnant, planning a pregnancy, or breast feeding.
研究者
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