EUCTR2018-003098-91-PL进行中（未招募）1 期
A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria - PHYOX-2
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 36
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. At least 6 years of age, at the time of signing the informed
- •consent/assent.
- •Type of Participant and Disease Characteristics
- •2. Documented diagnosis of PH1 or PH2, confirmed by genotyping
- •(historically available genotype information is acceptable for study
- •eligibility)
- •3. 24-hour Uox excretion = 0.7 mmol (adjusted per 1.73 m2 BSA in
- •participants < 18 years of age) in both collections performed in the
- •screening period. Of the first 24 participants enrolled, at least 12 (50%)
- •must have at least one 24 hour Uox excretion = 1.6 mmol (adjusted per 1.73 m2 BSA in participants aged < 18 years).
- •4. Less than 20% variation between the two 24-hour urinary creatinine
- •measurements in the screening period. Individuals who do not achieve <
- •20% variation between the 2 screening values may undergo a second
- •round of urine collection. An extra 7 calendar days may be added to the
- •screening window for participants to complete a second round of urine
- •collection. Should potential participants again fail to achieve the within-
- •20% variation, they will be excluded from participation.
- •5. Estimated GFR at screening = 30 mL/min normalized to 1.73 m2 BSA,calculated using the CKD-EPI equation in participants aged = 18 years(Levey & Stevens, 2010) or the2012 multivariate equation by Schwartz in participants aged 6 to 17 years (Schwartz et al., 2012).
- •In Japan, the equation by Matsuo et al. will be used for participants aged = 18 years (Uemura et al., 2014 Matsuo et al., 2009).
- •6. Male or female
- •Male participants:
- •A male participant with a female partner of childbearing potential must
- •agree to use contraception, as detailed in Section 10.4.2, 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 10.4.1), not breastfeeding, and at least 1 of the following
- •conditions applies:
- •Not a woman of childbearing potential (WOCBP) as defined in Section
- •A WOCBP who agrees to follow the contraceptive guidance in Section
- •10.4.2 during the treatment period and for at least 12 weeks after the
- •last dose of study intervention.
- •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
- •7. 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
- •local regulations.
- 另有 6 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 renal or hepatic transplantation; or planned transplantation
- •within the study period
- •2. Currently receiving dialysis or anticipating requirement for dialysis
- •during the study period
- •3. Plasma oxalate > 30 µmol/L
- •4. Documented evidence of clinical manifestations of systemic oxalosis
- •(including pre-existing retinal, heart, or skin calcifications, or history of
- •severe bone pain, pathological fractures, or bone deformations)
- •5. 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 or transaminase elevation
- •(e.g., alcoholic liver disease, nonalcoholic fatty liver
- •disease/steatohepatitis)
- •c. 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)
- •d. history of serious mental illness that includes, but is not limited to,
- •schizophrenia, bipolar disorder, or severe depression requiring
- •hospitalization or pharmacological intervention
- •e. clinically relevant history or presence of cardiovascular, respiratory,
- •gastrointestinal, hematological, lymphatic, neurological,
- •musculoskeletal, genitourinary, immunological diseases, including
- •dermatological including rash, severe eczema or dermatitis, or
- •connective tissue diseases or disorders
- •Prior/Concomitant Therapy
- •6. Routine or chronic use of more than 3 grams of
- •acetaminophen/paracetamol daily
- •7. Use of an RNA interference (RNAi) drug within the last 6 months
- •8. 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 aminotransferase (ALT) or
- •aspartate aminotransferase (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
- •9. Participants receiving pyridoxine must have been at a stable dose for
- •at least 4 weeks prior to Day 1 and must be willing to remain on the
- •same stable dose throughout the study.
- 另有 11 项未显示
研究者
相似试验
已完成
2 期
A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary HyperoxaluriaNL-OMON49278Dicerna Pharmaceuticals Inc6
进行中（未招募）
1 期
Study for patients with Primary Hyperoxaluria to evaluate the efficacy, safety and tolerability of DCR-PHXCEUCTR2018-003098-91-GBDicerna Pharmaceuticals Inc36
进行中（未招募）
1 期
Study for patients with Primary Hyperoxaluria to evaluate the efficacy, safety and tolerability of DCR-PHXCPrimary HyperoxaluriaMedDRA version: 20.1Level: PTClassification code 10020703Term: HyperoxaluriaSystem Organ Class: 10038359 - Renal and urinary disordersEUCTR2018-003098-91-FRDicerna Pharmaceuticals Inc36
已完成
2 期
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)JPRN-jRCT2080225181Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)5
进行中（未招募）
1 期
Study for patients with Primary Hyperoxaluria to evaluate the efficacy, safety and tolerability of DCR-PHXCEUCTR2018-003098-91-ESDicerna Pharmaceuticals Inc36