Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 4 and unassigned studies evaluate Cyclosporin A across diverse indications, including renal transplantation complications, hypertrophic cardiomyopathy, acute myeloid leukemia, vernal keratoconjunctivitis, and post-transplant glycemic control. Supported by academic institutions, university hospitals, and government entities, these investigations reflect varied recruitment statuses ranging from not yet recruiting and actively recruiting to completed and unknown.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06903884 RECRUITING
Vernal Keratoconjunctivitis
The University of Hong Kong
2026-02-09 PHASE3
NCT04239859 NOT_YET_RECRUITING
Plaque Psoriasis
Singapore General Hospital
2024-01 PHASE4
NCT04547049 ACTIVE_NOT_RECRUITING
Leukemia; MDS
First Affiliated Hospital of Zhejiang University
2020-09-01 PHASE3
NCT06802055 RECRUITING
Aplastic Anaemia
Peking Union Medical College Hospital
2025-01-25 PHASE2
NCT04945096 UNKNOWN
Engraft Failure; Relapse; GVHD
The First Affiliated Hospital of Soochow University
2021-07-01 PHASE3
NCT06525948 UNKNOWN
Aplastic Anemia; Transfusion-dependent Anemia
Peking Union Medical College Hospital
2024-07-30 PHASE4
NCT06246786 WITHDRAWN
Triple Negative Breast Cancer; DNA Damage Repair Deficiency
Virginia G. Kaklamani
2024-07-01 EARLY_PHASE1
NCT06944132 COMPLETED
Pterygium; Pterygium of the Conjunctiva and Cornea
Benha University
2024-04-01
NCT06366685 Not yet recruiting
Esophageal Cancer|Exercise Training|Psychotherapy|Rehabilitation
Mengmeng Yuan|Anhui Medical University
2024-05-01 Not Applicable
NCT06320561 Not yet recruiting
Stroke
Vrije Universiteit Brussel|University Ghent|Research Foundation Flanders
2024-05 --
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
NCT06052761 Recruiting
Child in Hospital
Adiyaman University
2024-02-10 Not Applicable
NCT05692843 UNKNOWN
Atopic Dermatitis
Instituto de Investigación Hospital Universitario La Paz
2022-10-10 PHASE4
NCT05771012 COMPLETED
Management of Punctal Stenosis
Farwaniya Hospital
2021-01-01
NCT03026946 UNKNOWN
Hand Eczema
University Medical Center Groningen
2017-05-29 PHASE3
NCT04376528 UNKNOWN
Hepatitis, Autoimmune; Primary Biliary Cholangitis; Immunosuppression
West China Hospital
2021-06-16 PHASE4
NCT02370550 UNKNOWN
Sjogren's Syndrome
Peking University People's Hospital
2015-03 PHASE4
NCT03896971 COMPLETED
Aplastic Anemia
Safaa AA Khaled
2019-11-15 PHASE4
NCT04540926 UNKNOWN
COVID 19 Pneumonia
Instituto de Seguridad y Servicios Sociales de los Trabajadores del Estado
2020-09 PHASE1; PHASE2
NCT04451239 UNKNOWN
Keratonjunctivitis
Ameera Gamal Abdelhameed
2020-06-30
NCT02487069 COMPLETED
Acute Leukemia; Chronic Myeloid Leukemia; Myelodysplastic Syndrome
Nanfang Hospital, Southern Medical University
2015-06
NCT02472054 COMPLETED
Hemophagocytic Lymphohistiocytosis (HLH)
Assistance Publique - Hôpitaux de Paris
2015-06-29 PHASE1; PHASE2
NCT02876250 WITHDRAWN
Lung Transplantation
Hospices Civils de Lyon
2017-01 PHASE3
NCT02834468 UNKNOWN
Immune Thrombocytopenia
Shandong University
2016-06 PHASE3
NCT02706470 UNKNOWN
Miscarriage, Recurrent
Fudan University
2016-05 PHASE2; PHASE3
NCT01760096 UNKNOWN
Paroxysmal Nocturnal Hemoglobinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal; Proteinuria; Urination Disorders; Thrombosis; Bone Marrow Failure; Aplastic Anemia,; Anemia, Hemolytic
Institute of Hematology & Blood Diseases Hospital, China
2013-01 PHASE2
NCT01642979 UNKNOWN
Paroxysmal Nocturnal Hemoglobinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal; Proteinuria; Urination Disorders; Thrombosis; Bone Marrow Failure; Aplastic Anemia,; Anemia, Hemolytic
Institute of Hematology & Blood Diseases Hospital, China
2012-07 PHASE2
NCT01843348 COMPLETED
Kidney Transplantation; Renal Transplantation
Novartis Pharmaceuticals
2012-12-27 PHASE3
NCT01766375 UNKNOWN
Acute Myeloid Leukemia
Guangxi Medical University
2012-08 PHASE3
NCT02199964 TERMINATED
Dry Eye
Baylor College of Medicine
2014-03
NCT02203422 WITHDRAWN
Immune Thrombocytopenia
Shandong University
2014-07 PHASE3
NCT02314312 UNKNOWN
Transplantation of Kidney; Expanded Criteria Donor; ECD
Mahidol University
2012-01 PHASE3
NCT00167583 COMPLETED
Behcet's Disease; Panuveitis; Posterior Uveitis; Retinal Vasculitis
University Hospital Tuebingen
2004-11 PHASE3
NCT01693393 COMPLETED
Sjögren´s Syndrome
Charite University, Berlin, Germany
2010-03 PHASE2
NCT01182662 UNKNOWN
Aplastic Anemia
Shandong University
2010-08 PHASE2
NCT00332839 TERMINATED
Renal Transplantation
Novartis Pharmaceuticals
2005-11 PHASE4
NCT00956293 TERMINATED
Renal Transplantation
Novartis Pharmaceuticals
2009-07 PHASE4
NCT00513474 COMPLETED
Chronic Myeloproliferative Disorders; Graft Versus Host Disease; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms
Massachusetts General Hospital
2008-01 PHASE1
NCT00176865 COMPLETED
Hemophagocytic Lymphohistiocytosis; X-Linked Lymphoproliferative Disorders; Chediak-Higashi Syndrome; Griscelli Syndrome; Immunologic Deficiency Syndromes; Langerhans-Cell Histiocytosis
Masonic Cancer Center, University of Minnesota
2002-08 PHASE2
NCT00303667 COMPLETED
Acute Myelogenous Leukemia
Masonic Cancer Center, University of Minnesota
2005-01 PHASE1; PHASE2
NCT01527240 COMPLETED
Stroke
Hospices Civils de Lyon
2009-10 PHASE2
NCT00520468 COMPLETED
Myelodysplastic Syndrome
M.D. Anderson Cancer Center
2004-06 PHASE2
NCT00565669 COMPLETED
Dry Eye Syndrome
Medical University of South Carolina
2007-10
NCT00375895 TERMINATED
Chronic Hepatitis C; Evidence of Liver Transplantation
Rennes University Hospital
2006-06 PHASE3
NCT00359814 COMPLETED
Heart Transplantation
Hannover Medical School
2004-11
NCT00359658 COMPLETED
Heart Transplantation
Hannover Medical School
2004-11
NCT01163942 TERMINATED
Aplastic Anaemia
European Society for Blood and Marrow Transplantation
2001-03 PHASE3
NCT00114777 COMPLETED
Renal Transplantation
Bristol-Myers Squibb
2005-02 PHASE3
NCT00521924 TERMINATED
Rheumatoid Arthritis
Merck Sharp & Dohme LLC
2007-06 PHASE3
NCT00001965 COMPLETED
Cardiomyopathy, Hypertrophic; Heart Hypertrophy
National Heart, Lung, and Blood Institute (NHLBI)
1999-12 PHASE2
NCT00003920 UNKNOWN
Breast Cancer
University of California, Davis
1996-04 PHASE1
NCT00001212 COMPLETED
Nephrotic Syndrome; Systemic Lupus Erythematosus
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
1986-11 PHASE2
NCT00008021 UNKNOWN
Lymphoma
University of California, Davis
2001-02 PHASE1
NCT00002688 UNKNOWN
Leukemia
University Medical Center Groningen
1995-02 PHASE2
NCT00009776 UNKNOWN
Lymphoma
University of California, Davis
2001-03 PHASE1
NCT00001731 COMPLETED
Keratoconjunctivitis Sicca; Sjogren's Syndrome
National Eye Institute (NEI)
1997-11 PHASE2
NCT00458250 COMPLETED
Leukemia, Myeloid, Acute; Leukemia, Lymphoblastic, Acute
Tehran University of Medical Sciences
2006-09 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-04-30)

Check the Cyclosporin A product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Cyclosporin A binds to the cytosolic immunophilin cyclophilin, forming a complex that inhibits calcineurin and prevents the dephosphorylation and nuclear translocation of nuclear factor of activated T-cells, thereby suppressing interleukin-2 transcription and T-lymphocyte activation. This targeted immunosuppressive activity suppresses allogeneic immune responses, providing therapeutic utility in organ transplantation such as kidney transplant management and associated complications evaluated in clinical trials.

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>).

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