4 completed, 2 terminated, 1 not yet recruiting, 1 recruiting. 5 Phase 2, 2 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Terminated 2, Not yet recruiting 1 |
| Not applicable | 2 | Completed 1, Unknown 1 |
| Phase 3 | 1 | Completed 1 |
Follow ERT: new trials by email Literature Review of ERT trials Research Report on ERT
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 Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD) S-606001, ERT - Pompe Disease | Phase 2 | Not yet recruiting | Shionogi | 60 | 2026-11-02 | 2026-10-13 |
| A Study of Patients With Fabry Disease (US Specific) migalastat HCl, ERT - Fabry Disease | Recruiting | Amicus Therapeutics | 450 | 2026-02-13 | 2026-09-09 | |
| MINDD 3: Prediabetes and Delay Discounting Episodic Future Thinking, Episodic Recent Thinking, Scarcity Narrative - PreDiabetes | Not applicable | Completed | Leonard Epstein | 78 | 2018-02-12 | 2023-02-21 |
| Miglustat on Gaucher Disease Type IIIB Miglustat, ERT - Gaucher Disease | Not applicable | Unknown | National Taiwan University Hospital | 19 | 2015-07-01 | 2019-03-29 |
| 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 |
| Stem Cell Transplant w/Laronidase for Hurler Stem Cell Transplant, Laronidase ERT - Mucopolysaccharidosis I, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 25 | 2004-03-01 | 2019-07-11 |
Lysosomal Storage Diseases (4) · Mucopolysaccharidosis IV (4) · Fabry Disease (1) · Gaucher Disease (1) · Glycogen Storage Disease (1) · Glycogen Storage Disease Type II (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Prediabetic State (1) · Rare Diseases (1)
BioMarin Pharmaceutical (4) · Amicus Therapeutics (1) · Leonard Epstein (1) · Masonic Cancer Center, University of Minnesota (1) · National Taiwan University Hospital (1) · Shionogi (1)
United States (7) · United Kingdom (4) · Germany (3) · Taiwan (3) · Canada (2) · Italy (2) · Argentina (1) · Brazil (1) · Colombia (1) · Denmark (1) · France (1) · Japan (1) · Netherlands (1) · Norway (1) · Portugal (1)
Debra Lounsbury (2) · Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · Leonard H Epstein (1) · Paul Orchard, MD (1) · Warren K Bickel (1) · Yin-Hsiu Chien, M.D., Ph.D. (1)
PMID 26331768, PMID 27955919, PMID 28535791, PMID 29159458, PMID 29248359, PMID 32868537, PMID 32989616