A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria

Last updated: June 10, 2024
Sponsor: BioMarin Pharmaceutical
Overall Status: Trial Not Available

Phase

1

Condition

Liver Disease

Liver Disorders

Hypertriglyceridemia

Treatment

Placebo

BMN 255

Clinical Study ID

NCT06138327
255-102
  • Ages 18-70
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. History of NAFLD with a liver fat content ≥ 8.0%, as determined by Fibroscan (transient elastography) or magnetic resonance imaging-derived proton density fatfraction (MRI-PDFF) during screening.

  2. Hyperoxaluria, as defined by 24-hour urine oxalate excretion ≥ 45 mg/24 hours/1.73m2at 2 independent assessments during screening. The pre-dose 24-hour urine oxalatemeasure at Day 1 will be used for baseline but will not be required for inclusion inthe study

  3. History of kidney stones (at least 1 stone prior to screening based on medicalhistory); participants with a known personal or family history of cystinuria orcystine kidney stones, calcium phosphate stones, struvite stones, or urate stonesshould not be included.

  4. Contraceptive use by men and women use throughout the study period

  5. Participants must be capable of giving signed informed consent

Exclusion

Exclusion Criteria:

  1. Clinical history (including family history) or genetic analyses consistent withprimary hyperoxaluria (Type 1, 2, or 3).

  2. History or current evidence of inflammatory bowel disease (including, but notlimited to: Crohn's disease, ulcerative colitis, celiac disease / gluten-sensitiveenteropathy) or evidence of chronic fat malabsorption (steatorrhea) due to any cause (eg, pancreatic insufficiency).

  3. 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 theinvestigator. Participants with Type II diabetes will be permitted to enroll butmust meet the concomitant therapy requirements listed below.

  4. Use or intend to use any prescription medications/products within 14 days prior toPeriod 1 check-in, other than permitted oral medications to treat controlledhypertension, dyslipidemia and/or to lower triglycerides, and oralanti-hyperglycemic agents (AHAs), including, but not limited to, metformin,sulfonylureas, and dipeptidyl peptidase IV (DPP-IV) inhibitors, if approved by theinvestigator. Participants who require insulin injections, glucagon-like peptide-1agonists, pioglitazone, or vitamin E ≥ 800 mg should not be included in the study. Note: Participants receiving lipid-modifying therapies and participants withcontrolled hypertension and/or diabetes must have been on a stable treatment regimen (medication, dose strength, dose interval) for 12 weeks prior to screening and nochange in that regimen should be anticipated for the entire duration of this study (ie, from screening to final follow-up visit).

  5. Confirmed diagnosis or NASH or evidence of hepatic cirrhosis, based on clinicalassessment (eg, physical examination), historical liver biopsy or other priorimaging study, or a liver stiffness value ≥ 14 kPa during the FibroScan® examinationat screening.

Study Design

Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1
Study Start date:
September 26, 2023
Estimated Completion Date:
March 25, 2024

Connect with a study center

  • University of Alabama - Department of Urology

    Birmingham, Alabama 35249
    United States

    Site Not Available

  • ProSciento, Inc.

    Chula Vista, California 91911
    United States

    Site Not Available

  • Georgia Clinical Research, LLC

    Lawrenceville, Georgia 30044
    United States

    Site Not Available

  • Medpace Clinical Pharmacology Unit

    Cincinnati, Ohio 45227
    United States

    Site Not Available

  • Centricity Research

    Columbus, Ohio 43213
    United States

    Site Not Available

  • Prolato Clinical Research Center

    Houston, Texas 77054
    United States

    Site Not Available

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