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WithdrawnNCT06138327Updated Jun 11, 2024

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

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 Withdrawn at 7 sites in United States.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

Why this study was withdrawn
The study was withdrawn because BioMarin decided to end the overall development program. The study withdrawal was not due to any patient safety concerns.
Phase
Phase 1
Study type
Interventional
Enrollment
0
Allocation
Randomized
Ages
18 Years to 70 Years
Sex
All
01

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.

02

Conditions studied

  • Hyperoxaluria
  • Nonalcoholic Fatty Liver Disease
  • Kidney Stone

Keywords

  • Kidney Stones
  • Nonalcoholic Fatty Liver Disease
  • Hyperoxaluria
  • NAFLD
  • MASLD
  • Metabolic Dysfunction-associated Steatotic Liver Disease
03

In context

Liver Diseases

2,081 studies on the registry are indexed under Liver Diseases; 390 are open to participants now.

Browse Liver Diseases studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 110 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

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 fat fraction (MRI-PDFF) during screening.
  2. 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
  3. 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.
  4. Contraceptive use by men and women use throughout the study period
  5. Participants must be capable of giving signed informed consent

Exclusion criteria

Exclusion Criteria:

  1. Clinical history (including family history) or genetic analyses consistent with primary hyperoxaluria (Type 1, 2, or 3).
  2. 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).
  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 the investigator. Participants with Type II diabetes will be permitted to enroll but must meet the concomitant therapy requirements listed below.
  4. 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).

  5. 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.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Crossover assignment
Masking
Double (Participant, Investigator)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    BMN 255 Investigational drug arm

    Oral administration of BMN 255 at a dose of 100mg per day for 7 days in Treatment Period 1 or 2

    Drug: BMN 255

  • Placebo comparator
    Placebo Comparative drug arm

    Oral administration of Placebo at a dose of 100mg per day for 7 days in Treatment Period 1 or 2

    Drug: Placebo

Interventions

  • DrugBMN 255

    Oral Capsule

  • DrugPlacebo

    Oral Capsule

06

What researchers measure

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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

  2. 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

07

Study locations

7 sites
  • University of Alabama - Department of Urology
    Birmingham, Alabama 35249, United States
  • ProSciento, Inc.
    Chula Vista, California 91911, United States
  • ProSciento, Inc.
    Chula Vista, Florida 91911, United States
  • Georgia Clinical Research, LLC
    Lawrenceville, Georgia 30044, United States
  • Medpace Clinical Pharmacology Unit
    Cincinnati, Ohio 45227, United States
  • Centricity Research
    Columbus, Ohio 43213, United States
  • Prolato Clinical Research Center
    Houston, Texas 77054, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 11, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06138327
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Nov 18, 2023
Start date
Sep 26, 2023
Primary completion
Mar 25, 2024
Completion
Mar 25, 2024
Last update
Jun 11, 2024

Study contacts

Medical Director, MD
study director · BioMarin Pharmaceutical

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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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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