Clinical Trials

Numerous clinical trials evaluate mycophenolic acid across Early Phase 1 through Phase 4 studies for organ transplantation, including kidney and pediatric liver transplants, as well as conditions such as systemic lupus erythematosus, Crohn's disease, and acute myeloid leukemia. Sponsored by major pharmaceutical companies like Novartis and Chiesi alongside academic medical centers such as Rigshospitalet and Children's Hospital Medical Center in Cincinnati, these trials encompass completed, actively recruiting, withdrawn, terminated, and not yet recruiting protocols.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07611214 RECRUITING
Lupus Nephritis
Aurinia Pharmaceuticals Inc.
 2026-04-22 PHASE4
NCT07508787 RECRUITING
Renal Transplant Failure and Rejection
Nefro Avillion Clinical Development, LLC
 2026-06-29 PHASE2
NCT07203664 NOT_YET_RECRUITING
Evaluate the Effectiveness of CMV Prevention Strategies in Kidney Transplant Recipients
Hospital Universitario La Paz
 2025-10-30 PHASE4
NCT02416388 RECRUITING
Acute Myeloid Leukemia (AML)
University Hospital, Angers
 2015-01 PHASE2; PHASE3
NCT05702931 Not yet recruiting
Hyperglycemia|Renal Transplant Complication Primary Non-Function|Diabetes
Rigshospitalet Denmark|Aarhus University Hospital|Odense University Hospital
 2024-04-01 Phase 4
NCT06268769 Recruiting
Immunosuppression
Edward Geissler|Chiesi Pharmaceuticals GmbH|University of Regensburg
 2024-03-09 Phase 4
NCT04773392 TERMINATED
Kidney Transplantation; Kidney Transplant Rejection
University of Southern California
 2021-11-23 PHASE4
NCT04066114 COMPLETED
Living-Donor Kidney Transplant; Kidney Transplant Recipients
National Institute of Allergy and Infectious Diseases (NIAID)
 2019-12-11 PHASE1; PHASE2
NCT06001320 Recruiting
Kidney Transplant; Complications|CMV
Virginia Commonwealth University|Merck Sharp & Dohme LLC
 2023-09-25 Early Phase 1
NCT02711826 COMPLETED
Kidney Transplant; Adult Living Donor Kidney Transplant Recipients; Renal Transplant; Living Kidney Donor
National Institute of Allergy and Infectious Diseases (NIAID)
 2016-09-20 PHASE1; PHASE2
NCT05859191 Recruiting
Systemic Lupus Erythematosus|Physiopathology
University Hospital Tours|Research Center for Respiratory Diseases Inserm U1100
 2023-07-21 --
NCT06361732 Recruiting
Pediatric Liver Transplant
Institute of Liver and Biliary Sciences India
 2022-12-17 --
NCT03644485 COMPLETED
Kidney Transplant
Astellas Pharma China, Inc.
 2018-10-21 PHASE4
NCT04734197 COMPLETED
Dry Eye Disease
Surface Ophthalmics, Inc.
 2021-01-11 PHASE2
NCT02498977 TERMINATED
Transplantation, Liver
King's College London
 2015-10 PHASE4
NCT02224872 COMPLETED
Severe Aplastic Anemia; Bone Marrow Failure Syndromes
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2014-08 PHASE2
NCT03828682 UNKNOWN
Kidney Transplantation; Immunosuppression
Simon Tremblay, PharmD, PhD
 2019-06-21 PHASE4
NCT03468478 COMPLETED
Kidney Transplant Infection
Hospital do Rim e Hipertensão
 2017-06-18 PHASE4
NCT02974686 TERMINATED
Kidney Transplant Rejection; Gastrointestinal Disorder, Functional
Washington University School of Medicine
 2016-11 PHASE4
NCT01936519 COMPLETED
Immunosuppression; Renal Failure
Milton S. Hershey Medical Center
 2013-12-16
NCT02991768 TERMINATED
Diarrhea
University of Kansas Medical Center
 2017-01-27 PHASE2
NCT01570348 TERMINATED
Crohn Disease
Fred Hutchinson Cancer Center
 2012-07-17 PHASE2
NCT02550652 COMPLETED
Lupus Nephritis
Hoffmann-La Roche
 2015-11-13 PHASE2
NCT02993133 COMPLETED
Autoimmune Bullous Dermatose
University Hospital, Limoges
 2016-12 PHASE3
NCT02328963 COMPLETED
Transplantation Infection; Cytomegalovirus Infection
University Hospital, Bordeaux
 2014-05-02 PHASE4
NCT01878786 TERMINATED
Delayed Graft Function
Matthew Cooper
 2013-06 PHASE2; PHASE3
NCT02036554 UNKNOWN
Kidney; Complications, Allograft
Seoul St. Mary's Hospital
 2013-03 PHASE4
NCT01560572 COMPLETED
Renal Insufficiency; Kidney Transplantation
University Medical Center Groningen
 2011-04 PHASE4
NCT01822483 COMPLETED
Renal Transplantation
Irmandade Santa Casa de Misericórdia de Porto Alegre
 2013-04 PHASE4
NCT01625377 COMPLETED
Liver Transplantation
Novartis Pharmaceuticals
 2012-12 PHASE3
NCT01624948 COMPLETED
BK Virus Infection
University of California, San Francisco
 2012-09 PHASE4
NCT02123108 COMPLETED
Liver Transplantation
University of California, Los Angeles
 2011-01 PHASE4
NCT01197040 COMPLETED
Nephrotic Syndrome
Assistance Publique - Hôpitaux de Paris
 2009-10 PHASE3
NCT01336296 COMPLETED
Kidney Transplant Recipients
University of Cincinnati
 2010-09 PHASE4
NCT01169701 COMPLETED
Renal Transplant
Novartis Pharmaceuticals
 2010-08 PHASE4
NCT01410162 COMPLETED
Renal Transplant; Kidney Pancreas Transplant
Norman Muirhead
 2010-12 PHASE4
NCT00956293 TERMINATED
Renal Transplantation
Novartis Pharmaceuticals
 2009-07 PHASE4
NCT01473732 TERMINATED
Chronic Allograft Injury; Calcineurin Inhibitor Toxicity
Montefiore Medical Center
 2012-03
NCT01230502 TERMINATED
Liver Transplant
University of Wisconsin, Madison
 2011-11
NCT00997412 UNKNOWN
Myasthenia Gravis
Qualitix Clinical Research Co., Ltd.
 2009-05
NCT00433953 TERMINATED
Kidney Transplant
Children's Hospital Medical Center, Cincinnati
 2007-02 PHASE1
NCT00251004 COMPLETED
Kidney Transplantation; Graft Rejection
Novartis
 2005-10 PHASE3
NCT00167492 WITHDRAWN
Liver Transplantation
The University of Texas Health Science Center, Houston
 2005-09 PHASE4
NCT00284947 COMPLETED
Kidney Transplantation; Adverse Effects
Novartis
 2006-01 PHASE4
NCT00403416 COMPLETED
Kidney Transplantation
Novartis Pharmaceuticals
 2006-10 PHASE1; PHASE2
NCT00374803 COMPLETED
End Stage Renal Disease (ESRD)
University of Cincinnati
 2006-04 PHASE4
NCT00128947 COMPLETED
Healthy
National Institutes of Health Clinical Center (CC)
 2005-07 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-08-03)

Check the Mycophenolic Acid (Mycophenolate, MPA) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Mycophenolic acid selectively inhibits inosine monophosphate dehydrogenase, blocking the rate-limiting step in the de novo pathway of guanine nucleotide synthesis and thereby halting the proliferation of T and B lymphocytes. This targeted suppression of adaptive immune cell expansion prevents cellular host-versus-graft reactions, establishing its therapeutic efficacy in kidney transplantation and autoimmune disorders such as systemic lupus erythematosus.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.