18 unknown. 18 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 18 | Unknown 18 |
| Phase 2 | 4 | Unknown 4 |
Follow Metabolism, Inborn Errors: new trials by email Literature Review of Metabolism, Inborn Errors trials Research Report on Metabolism, Inborn Errors
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 |
|---|---|---|---|---|---|---|
| The MAD Study of SSGJ-613 in Healthy Subjects SSGJ-613, Placebo - Arthritis, Gouty | Phase 1 | Unknown | Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. | 36 | 2023-08-10 | 2023-08-01 |
| Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide Allogeneic T cell progenitors, cultured ex-vivo - Hematological Malignancies | Phase 1 / Phase 2 | Unknown | Smart Immune SAS | 40 | 2023-06-06 | 2023-09-25 |
| Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease Low-dose CAN103, High-dose CAN103 - Gaucher Disease, Type 1, Gaucher Disease, Type 3 | Phase 1 / Phase 2 | Unknown | CANbridge (Suzhou) Bio-pharma Co., Ltd. | 40 | 2022-07-11 | 2022-07-27 |
| To Evaluate Drug Interactions Between XNW3009, Febuxostat, and Colchicine in Patients With Gout Colchicine - Gout | Phase 1 | Unknown | The Affiliated Hospital of Qingdao University | 20 | 2021-10-18 | 2022-05-09 |
| Study to Evaluate the Safety and PK of UI018 administration of UI018, co-administration of UIC201806 and UIC201602 - Hyperlipemia, Mixed | Phase 1 | Unknown | Korea United Pharm. Inc. | 44 | 2021-09-06 | 2021-12-21 |
| A Study to Determine the Safety, Tolerability, PK and Preliminary PD of SSS11 in Chinese Healthy Adult Volunteers PEGylated recombinant candida urate oxidase - Gout | Phase 1 | Unknown | Shenyang Sunshine Pharmaceutical Co., LTD. | 33 | 2019-10-22 | 2021-07-08 |
| Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C Hydroxypropyl-β-cyclodextrin - Niemann-Pick Disease, Type C1 | Phase 1 | Unknown | Cyclo Therapeutics, Inc. | 12 | 2019-05-23 | 2021-06-18 |
| Japanese OMI Treatment With Kit-positive Cells for Enhanced Regeneration Stem cells administered to participants - Chronic Heart Failure, Old Myocardial Infarction | Phase 1 | Unknown | Sakakibara Heart Institute | 6 | 2017-11-15 | 2021-03-18 |
| Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14 ORL-1G - D-galactose - Glycogen Storage Disease, Type 14 | Phase 1 / Phase 2 | Unknown | Orpha Labs | 5 | 2017-10-31 | 2019-01-25 |
| A Study to Assess the Safety and Tolerability of SSS11 in Healthy Subjects. SSS11, placebo - Gout | Phase 1 | Unknown | Shenyang Sunshine Pharmaceutical Co., LTD. | 40 | 2017-10-09 | 2018-09-14 |
| Study of Cytolytic Viral Activation Therapy (CVAT) for Recurrent/Metastatic Nasopharyngeal Carcinoma Gemcitabine, Valproic acid, Valganciclovir - Nasopharyngeal Carcinoma | Phase 1 | Unknown | Chang Gung Memorial Hospital | 18 | 2016-05-01 | 2016-05-16 |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy transduced CD34+ hematopoietic stem cell - Metachromatic Leukodystrophy, Adrenoleukodystrophy | Phase 1 / Phase 2 | Unknown | Shenzhen Second People's Hospital | 50 | 2015-01-01 | 2022-05-31 |
| Safety and Efficacy Study of Intramuscular Uricase-PEG 20 Uricase-PEG 20 - Gout, Hyperuricemia | Phase 1 | Unknown | EnzymeRx | 28 | 2009-12-01 | 2009-12-24 |
| Safety and Efficacy Study of Intravenous Uricase-PEG 20 Uricase-PEG 20 - Gout, Hyperuricemia | Phase 1 | Unknown | EnzymeRx | 20 | 2009-10-01 | 2010-02-23 |
| The Efficacy of Using Far Infrared Radiation to Manage Muscular Dystrophies Far Infrared Radiation - Muscular Dystrophies | Phase 1 | Unknown | GAAD Medical Research Institute Inc. | 4 | 2008-05-01 | 2009-08-17 |
| GTA-Glyceryltriacetate for Canavan Disease GTA: Glyceryltriacetate - Infantile Canavan Disease, Deficiency Disease, Aspartoacylase | Phase 1 | Unknown | Sheba Medical Center | 5 | 2006-01-01 | 2006-08-15 |
| Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets somatropin - Hypophosphatemic Rickets, Growth Disorders | Phase 1 | Unknown | University of Rostock | 2004-08-01 | 2007-05-14 | |
| Study of Treatment and Metabolism in Patients With Urea Cycle Disorders Protein and calorie controlled diet, Ornithine transcarbamylase vector - Amino Acid Metabolism, Inborn Errors | Phase 1 | Unknown | National Center for Research Resources (NCRR) | 66 | 1999-12-01 | 2005-06-24 |
Gout (5) · Hyperuricemia (2) · Adrenoleukodystrophy (1) · Amino Acid Metabolism, Inborn Errors (1) · Arthritis, Gouty (1) · Canavan Disease (1) · Distal Myopathies (1) · Epstein-Barr Virus Infections (1) · Familial Hypophosphatemic Rickets (1) · Gaucher Disease (1) · Glycogen Storage Disease (1) · Glycogen Storage Disease Type V (1) · Glycogen Storage Disease XIV (1) · Growth Disorders (1) · Hematologic Neoplasms (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Genetic Diseases, Inborn (18) · Metabolic Diseases (18) · Nutritional and Metabolic Diseases (18) · Musculoskeletal Diseases (8) · Nervous System Diseases (7) · Arthritis (6) · Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Central Nervous System Diseases (6) · Crystal Arthropathies (6) · Joint Diseases (6) · Purine-Pyrimidine Metabolism, Inborn Errors (6)
Caloric Restriction (1) · Colchicine (1) · Gemcitabine (1) · Human Growth Hormone (1) · Proteins (1) · Valganciclovir (1) · Valproic Acid (1)
EnzymeRx (2) · Shenyang Sunshine Pharmaceutical Co., LTD. (2) · CANbridge (Suzhou) Bio-pharma Co., Ltd. (1) · Chang Gung Memorial Hospital (1) · Cyclo Therapeutics, Inc. (1) · GAAD Medical Research Institute Inc. (1) · Korea United Pharm. Inc. (1) · National Center for Research Resources (NCRR) (1) · Orpha Labs (1) · Sakakibara Heart Institute (1) · Sheba Medical Center (1) · Shenzhen Second People's Hospital (1) · Smart Immune SAS (1) · Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. (1) · The Affiliated Hospital of Qingdao University (1)
China (6) · United States (4) · Canada (1) · France (1) · Germany (1) · Israel (1) · Japan (1) · South Korea (1) · Taiwan (1) · Turkey (Türkiye) (1)
Anthony S Fiorino, MD, PhD (2) · Alan Kivitz, MD (1) · Bing Han, MD (1) · Brendan Lee (1) · Caroline Hastings, MD (1) · Cheng-Lung Hsu, Physican (1) · Dieter Haffner, MD (1) · Dirk Schnabel, MD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hagen Staude (1) · Hua Jiang, M.D,Ph.D (1) · Injin Jang, Dr. (1) · Jiacai Zhuo (1) · Jianzhong Shentu, Doctor (1)
PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770, PMID 26416517, PMID 36279795