NCT06138327撤回1 期
A Phase 1b, Randomized, Double-Blind, Sponsor-Open, Placebo-Controlled, 2-Period Crossover Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of an Oral Administration of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease (NAFLD) And Hyperoxaluria
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 6
- 主要终点
- The primary safety endpoint is the incidence of adverse events in adult participants with NAFLD and hyperoxaluria
研究概览
简要总结
The purpose of this study is to test the safety of BMN 255 and to learn about the effect BMN 255 has on you and your hyperoxaluria associated with NAFLD, and compare these effects with a placebo.
The primary safety objective of the study is to assess the safety and tolerability of daily oral doses of BMN 255 in adult participants with NAFLD and hyperoxaluria.
The primary efficacy objective of the study is to assess 24-hour urine oxalate levels (24-hour urine collection corrected for BSA) following daily oral doses of BMN 255 in adult participants with NAFLD and hyperoxaluria.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •History of NAFLD with a liver fat content ≥ 8.0%, as determined by Fibroscan (transient elastography) or magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF) during screening.
- •Hyperoxaluria, as defined by 24-hour urine oxalate excretion ≥ 45 mg/24 hours/1.73m2 at 2 independent assessments during screening. The pre-dose 24-hour urine oxalate measure at Day 1 will be used for baseline but will not be required for inclusion in the study
- •History of kidney stones (at least 1 stone prior to screening based on medical history); participants with a known personal or family history of cystinuria or cystine kidney stones, calcium phosphate stones, struvite stones, or urate stones should not be included.
- •Contraceptive use by men and women use throughout the study period
- •Participants must be capable of giving signed informed consent
排除标准
- •Clinical history (including family history) or genetic analyses consistent with primary hyperoxaluria (Type 1, 2, or 3).
- •History or current evidence of inflammatory bowel disease (including, but not limited to: Crohn's disease, ulcerative colitis, celiac disease / gluten-sensitive enteropathy) or evidence of chronic fat malabsorption (steatorrhea) due to any cause (eg, pancreatic insufficiency).
- •Significant history or clinical manifestation of any other allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, neurological, respiratory, endocrine, or psychiatric disorder, as determined by the investigator. Participants with Type II diabetes will be permitted to enroll but must meet the concomitant therapy requirements listed below.
- •Use or intend to use any prescription medications/products within 14 days prior to Period 1 check-in, other than permitted oral medications to treat controlled hypertension, dyslipidemia and/or to lower triglycerides, and oral anti-hyperglycemic agents (AHAs), including, but not limited to, metformin, sulfonylureas, and dipeptidyl peptidase IV (DPP-IV) inhibitors, if approved by the investigator. Participants who require insulin injections, glucagon-like peptide-1 agonists, pioglitazone, or vitamin E ≥ 800 mg should not be included in the study.
- •Note: Participants receiving lipid-modifying therapies and participants with controlled hypertension and/or diabetes must have been on a stable treatment regimen (medication, dose strength, dose interval) for 12 weeks prior to screening and no change in that regimen should be anticipated for the entire duration of this study (ie, from screening to final follow-up visit).
- •Confirmed diagnosis or NASH or evidence of hepatic cirrhosis, based on clinical assessment (eg, physical examination), historical liver biopsy or other prior imaging study, or a liver stiffness value ≥ 14 kPa during the FibroScan® examination at screening.
研究组 & 干预措施
BMN 255 Investigational drug arm
Experimental
Oral administration of BMN 255 at a dose of 100mg per day for 7 days in Treatment Period 1 or 2
干预措施: BMN 255 (Drug)
Placebo Comparative drug arm
Placebo Comparator
Oral administration of Placebo at a dose of 100mg per day for 7 days in Treatment Period 1 or 2
干预措施: Placebo (Drug)
结局指标
主要结局
The primary safety endpoint is the incidence of adverse events in adult participants with NAFLD and hyperoxaluria
时间窗: Treatment Periods 1 & 2 through a 15 day follow-up after period 2. Each treatment period is 7 days separated by a 7-9 day washout period.
- Including but not limited to acute liver or kidney injury
次要结局
- To characterize the daily oral dose plasma pharmacokinetics of BMN 255 in adult participants with NAFLD and hyperoxaluria.(Day 1, Day 7 of treatment periods 1 & 2, each treatment period is 7 days separated by a 7-9 day washout period and Day 15)
研究者
研究点 (6)
Loading locations...
相似试验
终止
1 期
A Study of BMS-986224 in Healthy Subjects and Heart Failure Patients With Reduced Ejection FractionHeart FailureNCT03281122Bristol-Myers Squibb199
进行中（未招募）
1 期
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular DystrophyDuchenne Muscular DystrophyNCT06280209BioMarin Pharmaceutical18
已完成
1 期
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) DiseaseBatten DiseaseJansky-Bielschowsky DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2CLN2 DiseaseNCT01907087BioMarin Pharmaceutical24
已完成
1 期
A Study to Assess the Safety and Drug Levels of BMS-986256 in Participants With Active Cutaneous Lupus ErythematosusLupus Erythematosus, CutaneousNCT04493541Bristol-Myers Squibb13
进行中（未招募）
2 期
Study of Zatolmilast (BPN14770) in Participants With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome [JS])Jordan's SyndromePPP2R5D Neurodevelopmental DisorderNCT06717438Shionogi30