39 unknown. 39 Phase 2, 10 Phase 3, 4 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 39 | Unknown 39 |
| Phase 3 | 10 | Unknown 10 |
| Phase 1 | 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 |
|---|---|---|---|---|---|---|
| An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day - Sanfilippo Syndrome, MPS3 | Phase 2 / Phase 3 | Unknown | Ozlem Goker-Alpan | 10 | 2024-12-01 | 2025-08-20 |
| Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 Eliglustat Tartrate Capsules - Gaucher Disease | Phase 2 | Unknown | Peking Union Medical College Hospital | 5 | 2024-08-01 | 2024-07-26 |
| Phase II/III Study of AR882 Capsules Compared to Febuxostat Tablets in Patients with Primary Gout and Hyperuricemia Febuxostat 20MG Tablets, Febuxostat 20MG Placebo, AR882 25MG - Primary Gout, Hyperuricemia | Phase 2 / Phase 3 | Unknown | Guangzhou Ruianbo Pharmaceutical Technology Co., Ltd | 636 | 2024-04-22 | 2024-09-19 |
| Nutritional Therapy in Late-onset Pompe Disease Multi-ingredient supplement (PDT-MIS), Placebo (PLA) - Pompe Disease, Muscle Loss | Phase 2 | Unknown | McMaster University | 28 | 2024-04-01 | 2023-11-14 |
| A Study of SSGJ-613 in Gout Subjects Initiating Urate-Lowering Treatment. Recombinant Anti-IL-1β Humanized Monoclonal Antibody Injection 100 mg, Recombinant Anti-IL-1β Humanized Monoclonal Antibody Injection 200 mg, Colchicine 0.5 mg - Gout Arthritis | Phase 2 | Unknown | Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. | 157 | 2024-03-30 | 2024-08-05 |
| Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease AL01211 - Fabry Disease | Phase 2 | Unknown | AceLink Therapeutics, Inc. | 16 | 2023-10-25 | 2024-07-30 |
| Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients Dapagliflozin 10mg Tab, Placebo - Fabry Disease | Phase 2 | Unknown | Albina Nowak, MD | 46 | 2023-08-01 | 2023-02-02 |
| 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 |
| Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia Empagliflozin - Glucose 6 Phosphatase Deficiency | Phase 2 | Unknown | Children's Memorial Health Institute, Poland | 20 | 2021-07-01 | 2021-06-23 |
| A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hypercholesterolemia SHR-1209 - Familial Hypercholesterolemia | Phase 2 | Unknown | Jiangsu HengRui Medicine Co., Ltd. | 8 | 2020-09-01 | 2020-07-02 |
| Oral Huzhang Granules for Acute Gouty Arthritis Huzhang granule, Etoricoxib, Huzhang granule Placebo - Gouty Arthritis | Phase 2 | Unknown | Shanghai Yueyang Integrated Medicine Hospital | 267 | 2020-07-01 | 2020-07-08 |
| Efficacy and Safety of Pirfenidone Treatment in HPS-ILD Pirfenidone - Hermansky Pudlak Syndrome, Interstitial Lung Disease | Phase 2 | Unknown | Jesse Roman | 50 | 2019-12-01 | 2019-12-10 |
| Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia IBI306 - Familial Hypercholesterolemia - Homozygous, Lipid Metabolism Disorders | Phase 2 / Phase 3 | Unknown | Shenzhen People's Hospital | 8 | 2019-11-05 | 2021-07-01 |
| Use of Propranolol Hydrochloride in the Treatment of Metastatic STS Propranolol Hydrochloride, Doxorubicin - Malignant Soft Tissue Sarcoma | Phase 2 | Unknown | Ain Shams University | 50 | 2019-08-30 | 2018-02-23 |
| Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency Empagliflozin - Glycogen Storage Disease Type I, Glucose 6 Phosphatase Deficiency | Phase 2 | Unknown | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | 5 | 2019-06-20 | 2019-10-28 |
| Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) LYS-SAF302 - Mucopolysaccharidosis Type IIIA | Phase 2 / Phase 3 | Unknown | LYSOGENE | 20 | 2018-12-17 | 2021-08-31 |
| Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI Losartan, Placebo - Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI | Phase 2 | Unknown | Hospital de Clinicas de Porto Alegre | 10 | 2018-11-07 | 2022-12-19 |
| A Treatment Extension Study of Mucopolysaccharidosis Type IIIB AX 250 - Mucopolysaccharidosis Type IIIB, MPS III B | Phase 2 | Unknown | Allievex Corporation | 20 | 2018-02-19 | 2023-01-18 |
| 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 |
Showing 20 of 39. Sign in to see all of them.
Gaucher Disease (4) · Mucopolysaccharidosis III (3) · Rare Diseases (3) · Adrenal Hyperplasia, Congenital (2) · Arthritis, Gouty (2) · Fabry Disease (2) · Glycogen Storage Disease Type I (2) · Gout (2) · Hyperuricemia (2) · Lysosomal Storage Diseases (2) · Mucopolysaccharidoses (2) · Smith-Lemli-Opitz Syndrome (2) · Acidosis, Lactic (1) · Adrenoleukodystrophy (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (39) · Metabolic Diseases (39) · Nutritional and Metabolic Diseases (39) · Genetic Diseases, Inborn (38) · Lipid Metabolism, Inborn Errors (14) · Lysosomal Storage Diseases (14) · Lipid Metabolism Disorders (13) · Nervous System Diseases (13) · Carbohydrate Metabolism, Inborn Errors (12) · Brain Diseases (10) · Brain Diseases, Metabolic (10) · Brain Diseases, Metabolic, Inborn (10) · Central Nervous System Diseases (10) · Lysosomal Storage Diseases, Nervous System (9)
empagliflozin (2) · Alipogene tiparvovec (1) · Ambroxol (1) · Amlodipine (1) · Antilymphocyte Serum (1) · Antioxidants (1) · Chenodeoxycholic Acid (1) · Cholesterol (1) · Colchicine (1) · Contraceptives, Oral (1) · Cortisone (1) · Cyclophosphamide (1) · Cyclosporine (1) · Dexamethasone (1) · Dichloroacetic Acid (1)
National Center for Research Resources (NCRR) (3) · Ain Shams University (2) · AceLink Therapeutics, Inc. (1) · Albina Nowak, MD (1) · Allievex Corporation (1) · Amsterdam Molecular Therapeutics (1) · Boston Children's Hospital (1) · CANbridge (Suzhou) Bio-pharma Co., Ltd. (1) · Children's Memorial Health Institute, Poland (1) · Cliniques universitaires Saint-Luc- Université Catholique de Louvain (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Green Cross Corporation (1) · Guangzhou Ruianbo Pharmaceutical Technology Co., Ltd (1) · Haukeland University Hospital (1) · Hospital de Clinicas de Porto Alegre (1)
United States (12) · China (8) · France (4) · Germany (2) · Turkey (Türkiye) (2) · United Kingdom (2) · Belgium (1) · Brazil (1) · Canada (1) · Colombia (1) · Denmark (1) · Egypt (1) · Iran (1) · Netherlands (1) · Norway (1)
William Connor (2) · Allan M. Lund, MD (1) · Bing Han (1) · Bing Han, MD (1) · Darryl C De Vivo, MD (1) · Ellen R Elias, MD (1) · GuangFu Chen, M.D.,Ph.D (1) · Guilherme Baldo, PhD (1) · Hejian Zou, MD (1) · Hua Jiang, M.D,Ph.D (1) · Jens Fogh (1) · Jesse Roman, MD (1) · Jia Zhou (1) · Jiacai Zhuo (1)
PMID 11231629, PMID 11399225, PMID 19892975, PMID 22717743, PMID 23845948, PMID 23966770, PMID 24795390, PMID 27400896, PMID 35365187, PMID 36507973, PMID 36919608, PMID 40587053, PMID 42861767
R01HD058567 (1)