7 completed, 5 recruiting, 2 not yet recruiting, 2 terminated. 5 Phase 2, 4 Phase 3, 2 Phase 1, 1 Not applicable, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 3, Recruiting 1, Terminated 1 |
| Phase 3 | 4 | Completed 2, Recruiting 1, Terminated 1 |
| Phase 1 | 2 | Completed 1, Recruiting 1 |
| Not applicable | 1 | Completed 1 |
| Phase 4 | 1 | Completed 1 |
Follow Lipodystrophy, Congenital Generalized: new trials by email Literature Review of Lipodystrophy, Congenital Generalized trials Research Report on Lipodystrophy, Congenital Generalized
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy Mibavademab, Placebo - Generalized Lipodystrophy | Phase 3 | Recruiting | Regeneron Pharmaceuticals | 28 | 2026-03-13 | 2026-09-11 |
| Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy Metreleptin - Generalized Lipodystrophy | Phase 3 | Terminated | Amryt Pharma | 1 | 2025-05-23 | 2026-07-22 |
| Granzyme B-targeted PET Imaging Monitoring Tumor Responses to Immunotherapy PET/CT Imaging with GZMB-targeted tracer - PET/CT, Solid Tumors, Adult | Not yet recruiting | Zhongnan Hospital | 200 | 2025-04-01 | 2025-04-08 | |
| The Value of PET Imaging Targeting Granzyme B in Predicting Efficacy and Prognosis in T-NK Cell Lymphoma T-NK Cell Lymphoma | Not yet recruiting | Ruijin Hospital | 50 | 2025-03-01 | 2025-03-07 | |
| A Study of the Safety of Mibavademab in Pediatric and Adult Participants Switching From Metreleptin to Mibavademab for the Treatment of Generalized Lipodystrophy (GLD) mibavademab - Generalized Lipodystrophy | Phase 3 | Completed | Regeneron Pharmaceuticals | 9 | 2024-12-16 | 2026-07-29 |
| GzmB-PET/CT to Evaluate Neoadjuvant Therapy Efficacy in Breast Cancer 68Ga-GZMB PET/CT - Breast Cancer Invasive | Recruiting | Shanghai Jiao Tong University School of Medicine | 50 | 2024-11-01 | 2024-12-10 | |
| PET Imaging Targeting Granzyme B Predicts Immunotherapy Efficacy in Diffuse Large B-cell Lymphoma Diffuse Large B-Cell Lymphoma | Recruiting | Ruijin Hospital | 150 | 2024-07-23 | 2025-01-01 | |
| [68Ga]Ga-GZP PET for Early Response Prediction in Colorectal Cancer During Neoadjuvant Therapy Received 68Ga-NOTA-GZP PET scanning - Colorectal Cancer Control and Prevention, Colorectal Cancer Recurrent | Not applicable | Completed | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology | 21 | 2024-05-06 | 2025-05-21 |
| 64Cu-GRIP B in Patients With Advanced Malignancies Copper-64 labeled Granzyme B (64Cu-GRIP B), Positron Emission Tomography (PET) - Prostate Cancer, Renal Cancer | Phase 1 / Phase 2 | Recruiting | Rahul Aggarwal | 91 | 2023-05-25 | 2026-03-17 |
| 68Ga-grazytracer PET/CT of Tumor Responses to Immunotherapy 68Ga-grazytracer - Cancer | Phase 1 / Phase 2 | Completed | Peking University Cancer Hospital & Institute | 24 | 2021-09-22 | 2024-12-04 |
| Study of REGN4461, a Leptin Receptor Agonist Antibody, in Patients With Generalized Lipodystrophy Placebo, Low-Dose REGN4461, High-dose REGN4461 - Generalized Lipodystrophy | Phase 2 | Completed | Regeneron Pharmaceuticals | 16 | 2020-01-07 | 2026-06-15 |
| Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy Metreleptin - Generalized Lipodystrophy | Phase 4 | Completed | Aegerion Pharmaceuticals, Inc. | 11 | 2018-11-14 | 2025-11-26 |
| Analyzing Childhood Recall Antigens in Patients With Pancreatic Cancer Gemcitabine, TT vaccine booster - Pancreatic Cancer | Phase 2 | Terminated | Albert Einstein College of Medicine | 10 | 2017-07-21 | 2024-04-18 |
| MEASuRE: Metreleptin Effectiveness And Safety Registry Metreleptin - Generalised Lipodystrophy, Partial Lipodystrophy | Recruiting | Chiesi Farmaceutici S.p.A. | 100 | 2016-10-11 | 2026-07-16 | |
| Lipodystrophy Connect Patient Registry Lipodystrophy | Completed | PatientCrossroads | 257 | 2014-01-01 | 2019-04-22 | |
| Ocular Surface Changes in Patients With Cystic Fibrosis impression cytology, obtain the tear fluid - Eye Manifestations | Unknown | University of Bialystok | 26 | 2006-08-01 | 2008-06-17 | |
| Trial of Leptin Replacement Therapy in Patients With Lipodystrophy Leptin, Placebo - Hypoleptinemia, Generalized Lipodystrophy | Phase 2 / Phase 3 | Completed | University of Texas Southwestern Medical Center | 25 | 2006-04-01 | 2024-03-27 |
| Expanded Access to REGN4461 for Patients With Diseases Associated With Deficient Leptin Signaling REGN4461 - Lipodystrophy, Generalized Lipodystrophy (GLD) | Available | Regeneron Pharmaceuticals | 2026-03-12 |
Lipodystrophy (5) · Lipodystrophy, Familial Partial (3) · Lipodystrophy, Partial, Acquired (3) · Insulin Resistance (2) · Breast Neoplasms (1) · Colorectal Neoplasms (1) · Cystic Fibrosis (1) · Diabetes Mellitus (1) · Eye Manifestations (1) · Hypertriglyceridemia (1) · Inflammation (1) · Kidney Neoplasms (1) · Lymphoma, Extranodal NK-T-Cell (1) · Lymphoma, Large B-Cell, Diffuse (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Genetic Diseases, Inborn (18) · Lipid Metabolism Disorders (18) · Lipid Metabolism, Inborn Errors (18) · Metabolic Diseases (18) · Metabolism, Inborn Errors (18) · Nutritional and Metabolic Diseases (18) · Skin Diseases (18) · Skin Diseases, Metabolic (18) · Skin and Connective Tissue Diseases (18) · Lipodystrophy (13) · Neoplasms (6) · Neoplasms by Site (4) · Digestive System Diseases (3) · Laminopathies (3)
metreleptin (3) · Gemcitabine (1) · Leptin (1) · Magnetic Resonance Spectroscopy (1) · Tetanus Toxoid (1) · recombinant methionyl human leptin (1)
Regeneron Pharmaceuticals (4) · Ruijin Hospital (2) · Aegerion Pharmaceuticals, Inc. (1) · Albert Einstein College of Medicine (1) · Amryt Pharma (1) · Chiesi Farmaceutici S.p.A. (1) · PatientCrossroads (1) · Peking University Cancer Hospital & Institute (1) · Rahul Aggarwal (1) · Shanghai Jiao Tong University School of Medicine (1) · Union Hospital, Tongji Medical College, Huazhong University of Science and Technology (1) · University of Bialystok (1) · University of Texas Southwestern Medical Center (1) · Zhongnan Hospital (1)
United States (9) · China (6) · France (3) · Germany (2) · Italy (2) · Belgium (1) · Brazil (1) · Peru (1) · Poland (1) · Russia (1) · Turkey (Türkiye) (1) · United Kingdom (1)
Abhimanyu Garg, MD (1) · Claudia Gravekamp, PhD (1) · Janet Boylan (1) · Lori Hartnett (1) · Malgorzata Mrugacz, MD, PhD (1) · Nan Li, M.D. (1) · Rahul Aggarwal, MD (1) · Vanessa Rangel Miller, M.S., M.B.A (1) · Xiaosong Chen (1) · Yong He, MD, PhD (1) · Zhaofei Liu, Ph.D. (1) · Zhi Yang, Ph.D. (1)
PMID 35788116, PMID 37237416, PMID 39389969
2019 (1), R01CA229354 (1), R01CA258297 (1)