EUCTR2020-002826-97-DE进行中（未招募）1 期
A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 and Severe Renal Impairment, With or Without Dialysis - PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1 and ESRD
Dicerna Pharmaceuticals Inc. (a Novo Nordisk Company)0 个研究点目标入组 28 人开始时间: 2020年12月8日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 28
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 order to be eligible to participate in this study, an individual must
- •meet all of the following criteria:
- •1. Four age groups of participants will be enrolled:
- •a. adults and adolescents (aged = 12 years)
- •b. children 6 to 11 years of age
- •c. children 2 to 5 years of age and
- •d. infants and newborns from birth to < 2 years of age.
- •Type of Participant and Disease Characteristics
- •2. Documented diagnosis of PH1 confirmed by genotyping (historically
- •available genotype information is acceptable for study eligibility)
- •3. Estimated GFR at Screening < 30 mL/min normalized to 1.73 m2 BSA.
- •See Section 8.2.6.2 of the protocol for equations. For infants aged less
- •than 12 months, serum creatinine above the 97.5th percentile of a
- •healthy population
- •4. Mean of 2 plasma oxalate values > 20 µmol/L during Screening. See
- •Section 8.1.1 of the protocol for collection details.
- •5. For participants receiving hemodialysis or peritoneal dialysis, total
- •duration of hemodialysis or peritoneal dialysis must be less than or
- •equal to 24 months and hemodialysis or peritoneal dialysis regimen must
- •have been stable for at least 2 weeks prior to Screening.
- •7. Male or female
- •Male participants:
- •A male participant with a female partner of childbearing potential must
- •agree to use contraception, as detailed in Section 10.5.2, during the
- •treatment period and for at least 12 weeks after the last dose of study
- •intervention and refrain from donating sperm during this period.
- •Female participants:
- •A female participant is eligible to participate if she is not pregnant (see
- •Section 10.5.3), not breastfeeding, and at least one of the following
- •conditions applies:
- •Not a woman of childbearing potential (WOCBP) as defined in Section
- •10.5.1.1.
- •A WOCBP who agrees to follow the contraceptive guidance in Section
- •10.5.2.2 for the 4 weeks prior to randomization, during the treatment
- •period, and for at least 12 weeks after the last dose of study intervention
- •and agrees to refrain from harvesting/freezing eggs during this period.
- •Contraceptive use by men or women should be consistent with local
- •regulations regarding the methods of contraception for those
- •participating in clinical studies.
- •Informed Consent/Assent
- •8. Participant (and/or participant's parent or legal guardian if
- •participant is a minor [defined as patient < 18 years of age, or younger
- •than the age of majority according to local regulations]) is capable of
- •giving signed informed consent, which includes compliance with the
- •requirements and restrictions listed in the informed consent form (ICF)
- •and in this protocol.
- •a. Adolescents (12 to < 18 years of age, or older than 12 years but
- •younger than the age of majority according to local regulations) must be
- •able to provide written assent for participation.
- •b. For children younger than 12 years of age, assent will be based on
- 另有 9 项未显示
排除标准
- •An individual who meets any of the following criteria will be excluded from participation in this study:
- •Medical Conditions
- •1. Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Renal transplantation planned in the 6 months from Day 1. Prior renal transplantation is allowed.
- •2. Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
- •3. Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:
- •a. Severe intercurrent illness
- •b. Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
- •c. Non-PH related conditions contributing to renal insufficiency
- •d. Physician concerns about intake of drugs of abuse or excessive alcohol intake, or history of excessive alcohol intake in the 2 years prior to enrollment (defined as = 21 units of alcohol per week in men and = 14 units of alcohol per week in women; where a unit” of alcohol is equivalent to a 12-ounce beer, 4-ounce glass of wine, or 1 ounce shot of hard liquor)
- •Prior/Concomitant Therapy
- •4. Use of an RNAi drug, other than DCR-PHXC, within the last 6 months
- •5. History of one or more of the following reactions to an oligonucleotide-based therapy:
- •a. Severe thrombocytopenia (platelet count = 100,000/µL)
- •b. Hepatotoxicity, defined as alanine transaminase (ALT) or aspartate transaminase (AST) > 3 times the upper limit of normal (ULN) and total bilirubin > 2 × ULN or international normalized ratio (INR) >1.5
- •c. Severe flu-like symptoms leading to discontinuation of therapy
- •d. Localized skin reaction from the injection (graded severe) leading to discontinuation of therapy
- •e. Coagulopathy/clinically significant prolongation of clotting time
- •Prior/Concurrent Clinical Study Experience
- •6. Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months or 5 times the half-life of the drug (whichever is longer) before Screening.
- •Diagnostic Assessments
- •7. Liver function test abnormalities: ALT and/or AST >1.5 × ULN for age and gender
- •8. Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening
- •Other Exclusions
- •9. Known hypersensitivity to DCR-PHXC or any of its ingredients
- •10. Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations detailed in Section 5.3.
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