leflunomide / Generic mfg. - LARVOL DELTA

"Effect of Vitamin E (α-Tocopherol) on Clinical Activity and Inflammation in Rheumatoid Arthritis" (clinicaltrials.gov) - P2/3 | N=46 | Not yet recruiting | Sponsor: University of Guadalajara | Trial completion date: Dec 2027 ➔ Jan 2027 | Trial primary completion date: Jun 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date • Immunology • Inflammation • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

Efficacy and Safety of Therapies for Pediatric Steroid-Resistant Idiopathic Nephrotic Syndrome: A Systematic Review of the Last Decade. (PubMed, Cureus) - "Interventions evaluated included tacrolimus (TAC), cyclosporine A (CsA), mycophenolate mofetil (MMF), cyclophosphamide (oral and intravenous), leflunomide (LEF), rituximab (RTX), and ofatumumab (OFA), mostly in combination with steroids. MMF appears favorable after RTX compared to CsA, while cyclophosphamide shows no advantage between oral and intravenous administration. Despite progress, evidence remains limited by small sample sizes and heterogeneity, underscoring the need for large-scale, multicenter trials to optimize therapeutic strategies."

Journal • Review • Chronic Kidney Disease • Glomerulonephritis • Hematological Disorders • Infectious Disease • Nephrology • Pediatrics • Renal Disease

Romiplostim N01 for the treatment of cancer treatment-induced thrombocytopenia (ChiCTR) - P=N/A | N=950 | Not yet recruiting | Sponsor: Yidu Central Hospital of Weifang City; Yidu Central Hospital of Weifang City

New trial • Oncology • Thrombocytopenia

REDUCED-TOXICITY MYELOABLATIVE TBI-BASED CONDITIONING FOR ≥2-MISMATCH PARTIALLY MATCHED RELATED DONOR HSCT WITH PTCY IN HIGH-RISK HEMATOLOGICAL MALIGNANCIES: SINGLE-CENTRE REAL-WORLD OUTCOMES FROM INDIA (EBMT 2026) - "Background: Partially matched related donor (PMRD) HSCT with post-transplant cyclophosphamide (PTCy) broadens access to allogeneic transplantation...Conditioning comprised fludarabine–TBI 8 Gy (predominantly ALL) and fludarabine–melphalan 75 mg/m²–TBI 4 Gy...Program specific modifications adapted from earlier clinical experience of program leads included (i) early UGIE for upper-GI symptoms enabling prompt diagnosis of predominantly upper-GI aGVHD, (ii) planned early immunosuppression taper/withdrawal (typically by ~day 60) to augment graft-versus-leukemia effect, (iii) universal prophylactic/pre-emptive granulocyte transfusions for all patients, and (iv) a resource-adapted CMV strategy with off-label leflunomide-based pre-emptive management for low-level CMV DNAemia before letermovir availability...CMV reactivation occurred in 5/10 and BK virus in 2/10; TA-TMA occurred in 1/10 (resolved with Rituximab , Narsoplimab) and VOD/SOS in 0/10... An RT-MAC TBI-based..."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology

Pharmacogenetics of Leflunomide in the Management of Rheumatoid Arthritis (clinicaltrials.gov) - P=N/A | N=110 | Recruiting | Sponsor: Foundation University Islamabad

Biomarker • New trial • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

"Beyond Corticosteroids: A Systematic Review and Meta-analysis of Novel Therapies for Hypersensitivity Pneumonitis". (PubMed, Respir Med) - "Due to heterogeneity and limited high-quality evidence, definitive conclusions about novel therapies in HP remain elusive. However, rituximab and MMF showed promising results. Long-term randomized trials with standardized endpoints are needed to guide therapeutic decisions."

Journal • Retrospective data • Fibrosis • Immunology • Inflammation • Interstitial Lung Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases

Trastuzumab Combined With Chemotherapy and Leflunomide for HER2-Expressing Unresectable Advanced Biliary Tract Malignancies: An Open-Label, Single-Arm, Phase II Study (ChiCTR) - P2 | N=53 | Not yet recruiting | Sponsor: The Third Affiliated Hospital of Naval Medical University (Shanghai Eastern Hepatobiliary Surgery Hospital); The Third Affiliated Hospital of Naval Me

New P2 trial • Biliary Cancer • Biliary Tract Cancer • Cholangiocarcinoma • Gallbladder Cancer • Oncology • Solid Tumor • HER-2

Formulation and Optimization of Leflunomide-Loaded NLC-Gel for Improved Skin Permeation and Anti-Inflammatory Efficacy. (PubMed, J Drug Target) - "The in-vivo pharmacodynamic study using a carrageenan-induced paw edema model exhibited an 89.40% reduction in inflammation, exceeding the efficacy of the marketed leflunomide formulation. These findings suggest that the leflunomide-loaded NLC-based gel offers a promising platform for dermal drug distribution and enhanced anti-inflammatory activity."

Journal • Immunology • Inflammation • Rheumatology

Integrative miRNA-mRNA Network and Molecular Dynamics-Based Identification of Therapeutic Candidates for Paroxysmal Nocturnal Hemoglobinuria. (PubMed, Pharmaceuticals (Basel)) - "Molecular docking and molecular dynamics simulations showed stable binding of these potential drugs to the critical molecules, indicating a viable molecular interaction foundation. Leflunomide, Dipyridamole, and Pentoxifylline may serve as promising therapeutic agents for PNH, and the hsa-miR-23a-3p/CXCL8 regulatory axis could play a pivotal role in the pathogenesis and progression of PNH."

Journal • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • CXCL12 • CXCL8 • HES1 • MIR23A

Treatment of Reactive Histiocytosis With Oclacitinib: A Retrospective Case Series of 10 Dogs. (PubMed, Vet Dermatol) - "Although reactive histiocytosis can have a naturally waxing and waning course, oclacitinib appears to be rapidly effective for management, even in cases refractory to other immunomodulatory agents or with involvement of the oral or nasal cavities."

Journal • Retrospective data

DRESSed for Relapse: A Rare Case of Leflunomide-Induced DRESS With Hepatic Complications. (PubMed, Int J Rheum Dis) - No abstract available

Journal

Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (clinicaltrials.gov) - P=N/A | N=3 | Terminated | Sponsor: University Hospital, Basel, Switzerland | N=15 ➔ 3 | Active, not recruiting ➔ Terminated; The study was prematurely terminated due to significant difficulties in recruiting participants, arising from the extremely rare nature of the targeted gene mutation.

Enrollment change • Trial termination

Effects of Telitacicept vs Cyclophosphamide on Lupus Related Interstitial Lung Disease (clinicaltrials.gov) - P4 | N=100 | Recruiting | Sponsor: Tongji Hospital | Enrolling by invitation ➔ Recruiting | Trial completion date: Dec 2025 ➔ Mar 2027 | Trial primary completion date: Dec 2025 ➔ Oct 2026

Enrollment status • Trial completion date • Trial primary completion date • Immunology • Inflammatory Arthritis • Interstitial Lung Disease • Lupus • Pulmonary Disease • Respiratory Diseases • Systemic Lupus Erythematosus

MFN2 interacts with phosphorylated AMPK to mediate mitophagy in MCF-7 cells. (PubMed, Int J Biochem Cell Biol) - "MFN2 overexpression triggers mitochondrial degradation, as verified by multiple mitophagy markers, whereas MFN2 knockdown abolishes the mitophagic response induced by Leflunomide (Lef), a compound that promotes mitophagy by upregulating MFN2...Notably, AMPK activation through Acadesine (AICAR) treatment is insufficient to induce mitophagy, but it markedly enhances mitophagy markers when combined with MFN2 overexpression. In conclusion, MFN2 mediates efficient mitophagy by recruiting Thr172-phosphorylated AMPK to mitochondria through a phosphorylation-dependent MFN2-AMPK complex."

Journal • MFN2

Leflunomide-induced pancytopenia and agranulocytosis resulting in septic shock: A case report. (PubMed, Medicine (Baltimore)) - "Clinicians should maintain a high index of suspicion for drug-induced bone-marrow suppression in patients receiving leflunomide who develop unexplained cytopenias. Early drug discontinuation, aggressive supportive therapy, and timely initiation of cholestyramine washout may improve outcomes in this rare but life-threatening complication."

Journal • Agranulocytosis • Critical care • Granulocytopenia • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology • Septic Shock

BCOR mutations define a therapeutic vulnerability to DHODH Inhibition in acute myeloid leukemia. (PubMed, Ann Hematol) - "We demonstrate that BCOR-deficient cells have a heightened sensitivity to DHODH inhibitors such as brequinar and leflunomide, that are already in clinical use. Rather, DHODH's role in the electron transport chain, essential for mitigating reactive oxygen species, may be the physiological vulnerability that pushes BCOR-mutant cells toward cell death when DHODH is inhibited. DHODH inhibitors could be repurposed as targeted therapies for BCOR-mutant tumors, offering a promising strategy for precision medicine in AML and other cancers."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BCL6 • BCOR

Modified Proposomes as a Novel Intra-Articular Delivery Approach for Rheumatoid Arthritis. (PubMed, J Drug Target) - "A novel intra-articular (IA) nanocarrier system for leflunomide (LEF) was developed using modified proposomes to enhance therapeutic efficacy in rheumatoid arthritis (RA)...Histopathological evaluation confirmed significant preservation of joint architecture and attenuation of synovial inflammation in the LEF-HA-proposomes group. Collectively, these findings highlight LEF-HA-proposomes as a promising IA nanocarrier platform for targeted RA therapy, offering prolonged drug retention, enhanced local anti-inflammatory efficacy, and minimized systemic toxicity compared with conventional oral treatments."

Journal • Immunology • Inflammation • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology • IL1B • NFE2L2 • TNFA

Efficacy of Low-Dose Intravenous Immunoglobulin Combined With Immunosuppression Modification in the Treatment of BK Polyomavirus Nephropathy: A Case Series. (PubMed, Cureus) - "Apart from the reduction and manipulation of immunosuppressive medications, various treatment options such as intravenous immunoglobulin (IVIg), cidofovir, leflunomide, and fluoroquinolones have been tried to treat BK virus nephropathy. We report our experience with low-dose IVIg in six renal transplant recipients with BK polyomavirus nephropathy. We suggest that low-dose IVIg can be a promising treatment option to treat and prevent a decline in renal function and subsequent graft loss."

Journal • Infectious Disease • Nephrology • Renal Disease • Transplant Rejection • Transplantation

Praziquantel ameliorates leflunomide-induced nephrotoxicity in mice: a novel therapeutic approach targeting TGF-β/Smad/Wnt/β-catenin/NF-κB/PPAR-γ signaling and Nrf-2. (PubMed, Naunyn Schmiedebergs Arch Pharmacol) - "PZQ also significantly reduced oxidative stress markers (MDA 56.98%, p < 0.05) and pro-inflammatory cytokines (IL-6 - 73.69%, p < 0.05), while upregulating antioxidant (Nrf-2 + 6.7-fold) and anti-inflammatory (PPAR-γ + 3.1-fold) pathways. These statistically significant improvements confirm the nephroprotective efficacy of PZQ against LF-induced nephrotoxicity through modulation of TGF-β/Smad, Wnt/β-catenin, NF-κB, Nrf-2, and PPAR-γ signaling pathways."

Journal • Preclinical • Anesthesia • Immunology • CTNNB1 • IL6 • TGFB1

Generalized pustulosis after leflunomide (PubMed, Dermatologie (Heidelb)) - "It is a pyrimidine synthesis inhibitor that has an inhibiting effect of tumor necrosis factor alpha (TNF α) signaling. We present a case of with generalized pustular skin lesions after intake of leflunomide, which resemble a pustular exacerbation of a pre-existing psoriasis as well as a generalized pustular psoriasis."

Journal • Dermatology • Immunology • Inflammatory Arthritis • Oncology • Psoriasis • Psoriatic Arthritis • Pustular Psoriasis • Rheumatoid Arthritis • Rheumatology • Seronegative Spondyloarthropathies • TNFA

Evaluation of the ocular surface in rheumatoid arthritis patients on conventional synthetic disease-modifying antirheumatic drugs. (PubMed, J Fr Ophtalmol) - "Although a grade 3 CIC score was observed at a higher rate in the leflunomide group, this difference was not statistically significant. In addition, posterior blepharitis was observed more frequently in patients on methotrexate."

Journal • Dry Eye Disease • Immunology • Inflammatory Arthritis • Ocular Inflammation • Ophthalmology • Rheumatoid Arthritis • Rheumatology

Leflunomide for the Treatment of High-Risk Smoldering Multiple Myeloma (clinicaltrials.gov) - P1 | N=1 | Active, not recruiting | Sponsor: City of Hope Medical Center | Trial completion date: Dec 2025 ➔ Nov 2026 | Trial primary completion date: Dec 2025 ➔ Nov 2026

Trial completion date • Trial primary completion date • Hematological Malignancies • Multiple Myeloma • Oncology • Smoldering Multiple Myeloma

Rhupus Syndrome With Multiple Drug Intolerances Managed by Reduced-dose Rituximab and Adjunctive Molecular Hydrogen Therapy: A Case Report. (PubMed, In Vivo) - "This case suggests molecular hydrogen therapy as a potential adjuvant for refractory Rhupus with multiple drug intolerances, showing immune modulation, reduced inflammation, steroid withdrawal, and sustained control with low-dose rituximab. Further studies are needed to confirm its efficacy and standardize its use."

Journal • Glomerulonephritis • Immune Modulation • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Orthopedics • Rheumatoid Arthritis • Rheumatology • Systemic Lupus Erythematosus

The Effect of Cilostazol on Rheumatoid Arthritis Patients (clinicaltrials.gov) - P2/3 | N=70 | Completed | Sponsor: Ain Shams University | Active, not recruiting ➔ Completed

Trial completion • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

ELF: The Efficacy of Everolimus With Reduced-dose Tacrolimus Versus Reduced-dose Tacrolimus and Leflunomide in Treatment of BK Virus Infection in Kidney Transplantation Recipient (clinicaltrials.gov) - P=N/A | N=50 | Recruiting | Sponsor: King Chulalongkorn Memorial Hospital | Trial completion date: Jan 2026 ➔ Jul 2026 | Trial primary completion date: Sep 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date • Infectious Disease • Transplantation