10 completed, 2 terminated. 5 Phase 2, 2 Phase 1, 1 Not applicable, 1 Phase 3, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 3, Terminated 2 |
| Phase 1 | 2 | Completed 2 |
| Not applicable | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
| Phase 4 | 1 | Completed 1 |
Follow Enzyme Replacement Therapy: new trials by email Literature Review of Enzyme Replacement Therapy trials Research Report on Enzyme Replacement Therapy
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 |
|---|---|---|---|---|---|---|
| A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy. Enzyme replacement therapy for Gaucher disease in combination with altus care application - Gaucher Disease Type 1 | Phase 2 | Completed | Hadassah Medical Organization | 5 | 2022-04-01 | 2023-09-22 |
| Intestinal Microbiota Composition in Patients With Chronic Pancreatitis and Pancreatic Exocrine Insufficiency enzyme replacement therapy - Chronic Pancreatitis, Pancreatic Exocrine Insufficiency | Completed | Federal Research and Clinical Center of Physical-Chemical Medicine | 96 | 2020-11-01 | 2021-11-24 | |
| A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. Enzyme Replacement Therapy (ERT) - Fabry Disease | Completed | Amicus Therapeutics | 82 | 2020-03-01 | 2026-02-05 | |
| Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease Enzyme replacement therapy (Agalsidase alpha (Replagal®)), Magnetic Resonance Imaging - Anderson-Fabry Disease | Not applicable | Completed | University Hospital, Rouen | 26 | 2017-03-25 | 2026-04-17 |
| Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance Enzyme replacement therapy - Fabry Disease | Completed | Ulla Feldt-Rasmussen | 52 | 2015-04-01 | 2018-10-16 | |
| A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease Lucerastat, Enzyme replacement therapy (ERT) - Fabry Disease | Phase 1 | Completed | Idorsia Pharmaceuticals Ltd. | 14 | 2015-02-01 | 2018-07-10 |
| Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease velaglucerase alfa - Gaucher Disease, Type 3 | Phase 1 / Phase 2 | Completed | Shire | 7 | 2012-09-14 | 2021-06-11 |
| Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Phase 2 | Terminated | BioMarin Pharmaceutical | 16 | 2012-08-01 | 2016-01-12 |
| Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Phase 2 | Terminated | BioMarin Pharmaceutical | 25 | 2012-04-01 | 2016-02-01 |
| Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - Mucopolysaccharidosis IVA, Morquio A Syndrome | Phase 2 | Completed | BioMarin Pharmaceutical | 15 | 2011-10-01 | 2017-08-10 |
| Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - Weekly, BMN 110 - Every Other Week - Mucopolysaccharidosis IV A, Morquio A Syndrome | Phase 3 | Completed | BioMarin Pharmaceutical | 173 | 2011-07-01 | 2021-07-21 |
| Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. Enzyme replacement - Fabry Disease | Phase 4 | Completed | Copenhagen University Hospital, Rigshospitalet | 39 | 2001-09-01 | 2014-10-28 |
Fabry Disease (5) · Lysosomal Storage Diseases (4) · Mucopolysaccharidosis IV (4) · Gaucher Disease (2) · Exocrine Pancreatic Insufficiency (1) · Kidney Diseases (1) · Pancreatitis, Chronic (1)
BioMarin Pharmaceutical (4) · Amicus Therapeutics (1) · Copenhagen University Hospital, Rigshospitalet (1) · Federal Research and Clinical Center of Physical-Chemical Medicine (1) · Hadassah Medical Organization (1) · Idorsia Pharmaceuticals Ltd. (1) · Shire (1) · Ulla Feldt-Rasmussen (1) · University Hospital, Rouen (1)
United States (5) · Germany (4) · United Kingdom (4) · Denmark (3) · Taiwan (3) · Brazil (2) · Canada (2) · France (2) · Italy (2) · Japan (2) · Turkey (Türkiye) (2) · Argentina (1) · Colombia (1) · Egypt (1) · India (1)
Debra Lounsbury (2) · Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · Elena Ilina, Prof (1) · Igor Maev, Prof (1) · Noa Hurvitz, MD (1) · Ulla Feldt-Rasmussen, MD, DMSc (1) · Ulla V Feldt-Rasmussen, MD, DMSc (1)
PMID 26331768, PMID 27955919, PMID 28088251, PMID 28535791, PMID 29159458, PMID 29248359, PMID 39988522, PMID 40790487