2 completed, 2 terminated, 1 not yet recruiting. 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Terminated 2, Not yet recruiting 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 |
| 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 |
| 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 (3) · Mucopolysaccharidosis IV (3) · Glycogen Storage Disease (1) · Glycogen Storage Disease Type II (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Rare Diseases (1)
BioMarin Pharmaceutical (3) · Masonic Cancer Center, University of Minnesota (1) · Shionogi (1)
United States (4) · United Kingdom (3) · Germany (2) · Canada (1) · Italy (1) · Taiwan (1)
Adam Shaywitz, MD PhD (1) · Celeste Decker, M.D. (1) · Debra Lounsbury (1) · Paul Orchard, MD (1)