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BMN 255 for Non-alcoholic Fatty Liver Disease
Verified Trial
Phase 1
Recruiting
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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
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
Must not have
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)
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 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
Summary
This trial tests the safety of BMN 255, an oral medication, in adults with NAFLD and hyperoxaluria. The goal is to see if it can reduce urine oxalate levels, potentially preventing kidney stones and other issues.
Who is the study for?
This trial is for adults with Non-Alcoholic Fatty Liver Disease (NAFLD) and Hyperoxaluria, evidenced by high urine oxalate levels and a history of kidney stones. Participants must not have NASH or cirrhosis, inflammatory bowel disease, significant other medical conditions, or be on certain medications. They should have a liver fat content ≥ 8% confirmed by Fibroscan or MRI-PDFF.
What is being tested?
The study tests the safety and effectiveness of BMN 255 in reducing urine oxalate levels compared to a placebo. It involves daily oral doses of BMN 255 for participants with NAFLD and hyperoxaluria to see if it can improve their condition.
What are the potential side effects?
While specific side effects are not listed here, the trial aims to assess the safety and tolerability of BMN 255. Potential side effects could range from mild digestive issues to more serious organ-related symptoms.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have had at least one kidney stone but no family history of specific stone types.
Select...
I have a fatty liver with at least 8% fat content confirmed by a scan.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have or had inflammatory bowel disease or chronic issues absorbing fat.
Select...
I only use approved medications for hypertension, cholesterol, or diabetes and do not use insulin or specific diabetes drugs.
Select...
I have been diagnosed with NASH or have signs of severe liver scarring.
Select...
I have a diagnosis or family history of primary hyperoxaluria (Type 1, 2, or 3).
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 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
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~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
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
The primary safety endpoint is the incidence of adverse events in adult participants with NAFLD and hyperoxaluria
Secondary study objectives
To characterize the daily oral dose plasma pharmacokinetics of BMN 255 in adult participants with NAFLD and hyperoxaluria.
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: BMN 255 Investigational drug armExperimental Treatment1 Intervention
Oral administration of BMN 255 at a dose of 100mg per day for 7 days in Treatment Period 1 or 2
Group II: Placebo Comparative drug armPlacebo Group1 Intervention
Oral administration of Placebo at a dose of 100mg per day for 7 days in Treatment Period 1 or 2
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The primary treatment goal for hyperoxaluria is to reduce urine oxalate levels to prevent kidney stone formation and potential kidney damage. Common treatments include dietary modifications to reduce oxalate intake, calcium supplements to bind oxalate in the gut, and medications like BMN 255, which are designed to reduce oxalate production or enhance its breakdown.
BMN 255, for example, is being studied for its potential to lower 24-hour urine oxalate levels in patients with hyperoxaluria associated with NAFLD. These treatments are crucial as they help manage oxalate levels, thereby reducing the risk of kidney stones and preserving kidney function.
Oxidative stress and endoplasmic stress in calcium oxalate stone disease: the chicken or the egg?Oxalate-induced changes in renal epithelial cell function: role in stone disease.
Oxidative stress and endoplasmic stress in calcium oxalate stone disease: the chicken or the egg?Oxalate-induced changes in renal epithelial cell function: role in stone disease.
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Who is running the clinical trial?
BioMarin PharmaceuticalLead Sponsor
158 Previous Clinical Trials
114,978 Total Patients Enrolled
Medical Director, MDStudy DirectorBioMarin Pharmaceutical
80 Previous Clinical Trials
16,492 Total Patients Enrolled
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