What side effects are associated with this type of intervention?

The study of BMN 255 aims to assess its safety and efficacy in reducing urine oxalate levels in patients with hyperoxaluria associated with NAFLD. Common side effects of treatments targeting hyperoxaluria and similar conditions may include gastrointestinal disturbances, such as nausea and diarrhea, as well as potential increases in liver enzymes. In clinical trials, other adverse effects can include headache, chest pain, and changes in blood pressure. It is important for patients to discuss these potential side effects with their healthcare provider to ensure appropriate monitoring and management.

What prior FDA approvals exist?

Currently, there are limited FDA-approved treatments specifically targeting hyperoxaluria with a focus on reducing urine oxalate levels. Treatments often involve dietary modifications, hydration, and the use of medications like potassium citrate to prevent stone formation. BMN 255 is being studied for its potential to reduce urine oxalate levels in patients with NAFLD and hyperoxaluria, which could represent a novel therapeutic approach if proven effective.

What other types of research exist?

Promising novel alternatives to BMN 255 for hyperoxaluria patients include: 1) Oxalobacter formigenes, a probiotic therapy aimed at degrading oxalate in the gut, thereby reducing urinary oxalate levels. 2) Stiripentol, an antiepileptic drug that has shown potential in reducing oxalate production. 3) RNA interference (RNAi) therapies targeting genes involved in oxalate synthesis, such as HAO1 and LDHA, which are currently under investigation in clinical trials.

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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 below

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I have had at least one kidney stone but no family history of specific stone types.

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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.

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I only use approved medications for hypertension, cholesterol, or diabetes and do not use insulin or specific diabetes drugs.

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I have been diagnosed with NASH or have signs of severe liver scarring.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 for reporting.

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

0 / 6Eligibility criteria met

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.