EUCTR2017-003534-89-FR进行中（未招募）1 期
A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi Center Study in Patients with Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR PHXC Solution for Injection (subcutaneous use) - 2-part study of DCR-PHXC in NHV and PH patients
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 41
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
- •PH patients must meet all of the following criteria to be eligible for participation in this study.
- •1. Patient and/or patient's parent or guardian if the patient is a minor (defined as patient < 18 years of age, or younger than the age of majority, according to local regulations),
- •a. Understands the full nature and purpose of the study, including possible risks and side effects.
- •b. Is willing and able to comply with all study procedures including collection of 24-hr urine samples.
- •c. Provides informed consent. 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. For children younger than 12 years of age, assent will be based on local regulations.
- •2. Male or female, at least 6 years of age at the time of obtaining informed consent.
- •3. Patient must have a minimum body weight of 25 Kg.
- •4. Documented diagnosis of PH1 or PH2, confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
- •5. 24-hr urine oxalate excretion =0.7 mmol for patients 18 years and older, or =0.7 mmol per 1.73 m2 body surface area (BSA) for patients less than 18 years of age, on at least one of the two assessments conducted in the screening period, with less than 30% variation between both oxalate measurements.
- •6. eGFR =30 mL/min normalized to 1.73 m2 BSA calculated using the Modification of Diet in Renal Disease (MDRD) formula in adults (age =18 years), or the formula by Schwartz in patients 6 to < 18 years old (Levey et al., 1999; Schwartz et al., 2009; National Kidney Foundation, 2002).
- •7. Males, female patients of childbearing potential and female partners of male patients of childbearing potential must be willing to use a highly effective and approved contraceptive method(s) from the date of informed consent until 12 weeks after the last dose of IMP. A highly effective method of contraception is defined as fulfilling at least one of the following:
- •a. Strict abstinence: When this is in line with the preferred and usual lifestyle of the patient. [Periodic abstinence (e.g., calendar, ovulation, symptom-thermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.]
- •b. Surgically sterile (having undergone one of the following surgical procedures: hysterectomy, bilateral tubal ligation, bilateral oophorectomy, or bilateral salpingectomy) and at least 6 weeks post-sterilization.
- •c. Combined hormonal oral contraceptive (estrogen and progesterone), implanted, or injectable contraceptive on a stable dose for at least 1 month prior to the screening visit plus a barrier method. Combined hormonal contraception is considered a highly effective method of contraception only if it is associated with inhibition of ovulation. If associated with inhibition of ovulation, progesterone-only hormonal contraception is also considered a highly effective method of contraception.
- •d. Intrauterine devices plus condoms. Hormonal IUD inserted at least 1 month prior to the screening visit.
- •e. Double-barrier methods [e.g., Condom and Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository].
排除标准
- •PH patients meeting any of the following criteria will be excluded from this study:
- •1. Prior renal and/or hepatic transplantation.
- •2. Currently receiving dialysis.
- •3. Documented evidence of clinical manifestations of systemic oxalosis.
- •4. Participation in any clinical study where they received an investigational medical product within 4 months before enrollment. For IMPs with the potential to reduce Uox and/or plasma oxalate, these concentrations must have returned to historical baseline levels. a. If patient participated in an earlier cohort in this study (DCR-PHXC-101), a minimum of 8 weeks must have elapsed prior to re-enrollment and urinary oxalate excretion must have returned to =80% of baseline.
- •5. Presence of any medical condition or co-morbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to: a. severe intercurrent illness
- •b. routine vaccination within 30 days prior to dosing and through EOS visit
- •c. known causes of active liver disease/ injury or transaminase elevation (e.g., alcoholic liver disease, Nonalcoholic fatty liver disease/ steatohepatitis (NAFLD/NASH)
- •d. physician concerns about excess alcohol consumption
- •e. routine or chronic use of more than 3 grams of acetaminophen daily.
- •6. History of alcohol consumption exceeding more than 21 units in males, 14 units in females, per week as determined by the Investigator. See Section 5 for details.
- •7. Women who are pregnant, lactating, or planning to attempt to become pregnant during this study or within 90 days after last dosing of IMP.
- •8. Liver function test (LFT) abnormalities: ALT and/or AST >1.5 times ULN for age and gender.
- •9. History of one or more of the following reactions to an oligonucleotide-based therapy a. Severe thrombocytopenia
- •b. Hepatotoxicity
- •c. Severe flu-like symptoms leading to discontinuation of therapy
- •d. Localized skin reaction from the injection (Grade 3 or higher) leading to discontinuation of therapy.
- •e. Coagulopathy/ clinically important prolongation of clotting time
研究者
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