NCT05538208

Brief Summary

The study is a 1-year 2-part double-blinded placebo controlled 2-arm clinical trial. Treatment arms are (1) MMF dosed as per body-surface area (MMFBSA; 600mg/m2 body surface area per dose about every 12 hours) and (2) pharmacokinetically-guided precision-dosing of MMF (MMFPK; MMF dosed twice daily to achieve an area under the concentration-time curve (AUC0-12h) of MPA \>60-70 mg\*h/L. The study goal is to determine the safety and efficacy of MMFPK compared to MMFBSA for the treatment of proliferative LN in subjects 8 to \<21 years.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
105

participants targeted

Target at P50-P75 for phase_2

Timeline
7mo left

Started Jun 2024

Geographic Reach
1 country

19 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress76%
Jun 2024Jan 2027

First Submitted

Initial submission to the registry

September 9, 2022

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 13, 2022

Completed
1.7 years until next milestone

Study Start

First participant enrolled

June 7, 2024

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2026

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2027

Last Updated

March 18, 2026

Status Verified

March 1, 2026

Enrollment Period

2.1 years

First QC Date

September 9, 2022

Last Update Submit

March 16, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • To compare the efficacy of MMFPK therapy to the efficacy of MMFBSA therapy

    the percentage of subjects achieving at least partial remission of LN (PRR) as per the adapted ACR/EULAR Criteria at Week 26

    26 week

Study Arms (2)

MMFBSA

ACTIVE COMPARATOR

MMF dosed as per body-surface area

Drug: Mycophenolate Mofetil

MMFPK

EXPERIMENTAL

MMF dosed as per pharmacokinetically-guided precision-dosing

Drug: Mycophenolate Mofetil

Interventions

MMF dosed 600mg/m2 body surface area per dose about every 12 hours

Also known as: MMF dosed per body surface area
MMFBSA

Eligibility Criteria

Age8 Years - 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Male or female aged 8 to \< 21 years;
  • Must meet Classification Criteria for SLE as per the criteria of the American College of Rheumatology (ACR)/ European League Against Rheumatism
  • Diagnosed with proliferative LN as per the International Society of Nephrology/Renal Pathology Society4 based on kidney biopsy done within 90 days prior to enrollment into the study;
  • Treatment of LN with twice daily MMF as per the decision of the treating physician.
  • The subject will have taken MMF as prescribed by their treating physician for a minimum of 4 days (or 8 doses).
  • Subject tolerates MMF as per the treating physician's opinion;
  • Able to swallow MMF tablets and capsules;
  • If subject is treated with belimumab, must be IV or SQ;
  • Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures;
  • Evidence of a personally signed and dated Informed Consent document and Assent document (as appropriate) indicating that the subject and a legally acceptable representative/ parent(s)/legal guardian has been informed of all pertinent aspects of the study.
  • Parent or legal guardian must have a smart phone available and able to support the PLUMM smart phone application.
  • Must be able to complete study questionnaires in English or Spanish.

You may not qualify if:

  • Perceived or stated inability to adhere to the study protocol;
  • Hypersensitivity to MMF or any component of the drug product;
  • Presence of features (from SLE or other chronic disease) that a-priori suggest that the subject benefits from other therapies than that suggested or allowable by the study protocol; These disease features include but are not limited to severe, progressive, or uncontrolled hepatic, hematologic, gastrointestinal, metabolic, endocrine, pulmonary, cardiac or neurologic disease.
  • History of other kidney disease besides LN or prior to the diagnosis of SLE;
  • Need for renal replacement therapy within 2 weeks from Baseline Subjects can have required short-term renal replacement therapy prior to Baseline, for example due to preceding acute kidney injury.
  • Infections:
  • Untreated latent or active tuberculosis (TB);
  • Chronic infections requiring treatment;
  • A subject known to be infected with Human Immunodeficiency Virus (HIV), Hepatitis B;
  • Diagnosis of any infection requiring hospitalization, parenteral antimicrobial therapy or judged to be opportunistic by the investigator within 4 weeks prior to Baseline visit;
  • Any treated infections within 2 weeks of Baseline visit;
  • History of infected joint prosthesis with prosthesis still in situ;
  • Blood dyscrasias, including:
  • Hemoglobin \<8.5 g/dL or Hematocrit \<22%;
  • White Blood Cell count \<2.6 x 109/L;
  • +15 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

University of California, San Francisco

San Francisco, California, 94518, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Emory Children's Center

Atlanta, Georgia, 30322, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60614, United States

RECRUITING

University of Chicago Medicine- Comer Children's

Chicago, Illinois, 60637, United States

RECRUITING

Washington University in St. Louis School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

RECRUITING

Hospital for Special Surgery

New York, New York, 10021, United States

RECRUITING

Children's Hospital at Montefiore

New York, New York, 10467, United States

RECRUITING

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Akron Children's Hospital

Akron, Ohio, 44307, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45223, United States

RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Baylor College of Medicine Pediatric Immunology Allergy Rheumatology

Houston, Texas, 77030, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84132, United States

RECRUITING

Seattle Children's Hospital/University of Washington

Seattle, Washington, 98105, United States

RECRUITING

Children's Wisconsin/Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

MeSH Terms

Conditions

Lupus Nephritis

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

GlomerulonephritisNephritisKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesLupus Erythematosus, SystemicConnective Tissue DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

CaproatesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsFatty AcidsLipids

Study Officials

  • Hermine I Brunner, MD

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Angela Sr CRC

CONTACT

Cat Clinical Research Coordinator

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
During this double-blinded study, the Sponsor, subject, and investigator site staff will all be blinded to the subject's treatment assignment.
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Eligible patients enrolled in the study will be randomized (1:1) at baseline to the 53-week double-blind, active comparator 2-part study to receive either MMFPK or MMFBSA. Subjects who are partial renal responders (PRR) to MMFBSA at week 26, will cross over to the MMFPK arm. Complete renal responders (CRR) at week 26 in MMFBSA arm will continue to be treated with MMFBSA. Subjects with at least a PRR (or even CRR) in the MMFPK arm at week 26 will remain in the MMFPK arm and continue to receive MMF dosage targeting MPA-AUC0-12 \> 60-70 mg\*h/l. Subjects whose LN fails to respond to therapy by week 26 will be discontinued from the study interventions to receive LN treatment as per their local physician's decision. Subjects who experience a single episode of a LN flare during Part 1 of the study or fulfill other criteria for discontinuation from the study intervention, will also receive LN treatment as per their local physician's decision.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Division Director Rheumatology

Study Record Dates

First Submitted

September 9, 2022

First Posted

September 13, 2022

Study Start

June 7, 2024

Primary Completion (Estimated)

July 1, 2026

Study Completion (Estimated)

January 1, 2027

Last Updated

March 18, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will share

The Sponsor fulfills its commitment to publicly disclose clinical trial results through posting the results of studies on ClinicalTrials.gov, or EudraCT, and/or www.cincinnatichildrens.com, and other public registries in accordance with applicable local laws/regulations. In all cases, study results are reported by the Sponsor regardless of the outcome of the study. Every effort will be made to report the basic results within 1 year of the end of the trial. Results will be posted at www.clinicaltrials.gov/. For all publications relating to the study, the institution and investigators will comply with recognized ethical standards concerning publications and authorship, including Section II - "Ethical Considerations in the Conduct and Reporting of Research" of the Uniform Requirements for Manuscripts Submitted to Biomedical Journals, http://www.icmje.org/index.html#authorship. A Publication Committee will be established to oversee publication of the results.

Time Frame
within one year for
Access Criteria
results are available to the public
More information

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