A Phase 1 interventional study of BMN 255 and Placebo in Hyperoxaluria, Nonalcoholic Fatty Liver Disease and Kidney Stone, sponsored by BioMarin Pharmaceutical. Withdrawn at 7 sites in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2024-06-11.
Sponsored by BioMarin Pharmaceutical · Phase 1, Interventional, and Treatment
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.
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Exclusion Criteria:
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).
Oral administration of BMN 255 at a dose of 100mg per day for 7 days in Treatment Period 1 or 2
Drug: BMN 255
Oral administration of Placebo at a dose of 100mg per day for 7 days in Treatment Period 1 or 2
Drug: Placebo
Oral Capsule
Oral Capsule
The primary safety endpoint is the incidence of adverse events in adult participants with NAFLD and hyperoxaluria
- Including but not limited to acute liver or kidney injury
Time frame: 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.
To characterize the daily oral dose plasma pharmacokinetics of BMN 255 in adult participants with NAFLD and hyperoxaluria.
Area under the plasma concentration-time curve (AUC)
Time frame: 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
To characterize the daily oral dose plasma pharmacokinetics of BMN 255 in adult participants with NAFLD and hyperoxaluria.
Maximum observed concentration (Cmax)
Time frame: 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 study is withdrawn, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.
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