4 completed, 2 active not recruiting, 1 recruiting. 7 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 7 | Completed 4, Active not recruiting 2, Recruiting 1 |
| Phase 2 | 4 | Active not recruiting 2, Completed 2 |
Follow Mucopolysaccharidosis VI: new trials by email Literature Review of Mucopolysaccharidosis VI trials Research Report on Mucopolysaccharidosis VI
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI Vosoritide Injection [Voxzogo] - MPS IVA, MPS VI | Phase 1 / Phase 2 | Active not recruiting | University of California, San Francisco | 6 | 2023-09-25 | 2026-03-10 |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| Gene Therapy in Patients With Mucopolysaccharidosis Disease AAV2/8.TBG.hARSB - Mucopolysaccharidosis Type VI | Phase 1 / Phase 2 | Completed | Fondazione Telethon | 9 | 2017-07-17 | 2024-11-22 |
| Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Adalimumab Injection [Humira], Saline Solution for Injection - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Phase 1 / Phase 2 | Active not recruiting | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 14 | 2017-06-05 | 2026-02-02 |
| Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI Adalimumab, Placebo - Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II | Phase 1 / Phase 2 | Completed | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 2 | 2015-05-01 | 2017-09-15 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI N-acetylgalactosamine 4-sulfatase - Mucopolysaccharidosis VI | Phase 1 | Completed | BioMarin Pharmaceutical | 2000-09-01 | 2006-11-03 |
Mucopolysaccharidosis I (3) · Gaucher Disease (2) · Mucopolysaccharidosis II (2) · Mucopolysaccharidosis IV (2) · Wolman Disease (2) · Adrenoleukodystrophy (1) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Fucosidosis (1) · Inflammation (1) · Leukemia, Myeloid, Acute (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Metabolism, Inborn Errors (1)
Carbohydrate Metabolism, Inborn Errors (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Connective Tissue Diseases (7) · Genetic Diseases, Inborn (7) · Lysosomal Storage Diseases (7) · Metabolic Diseases (7) · Mucinoses (7) · Nutritional and Metabolic Diseases (7) · Skin and Connective Tissue Diseases (7) · Metabolism, Inborn Errors (6) · Mucopolysaccharidoses (6) · Nervous System Diseases (4) · Genetic Diseases, X-Linked (3) · Heredodegenerative Disorders, Nervous System (3) · Intellectual Disability (3)
Adalimumab (2) · GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Injections (1) · N-Acetylgalactosamine-4-Sulfatase (1) · Saline Solution (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1) · vosoritide (1)
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (2) · University of California, San Francisco (2) · BioMarin Pharmaceutical (1) · Fondazione Telethon (1) · New York Medical College (1)
United States (6) · Italy (1) · Turkey (Türkiye) (1)
Ellen B Fung, PhD (1) · Lynda E Polgreen, MD, MS (1) · Lynda Polgreen, MD (1) · Mitchell S Cairo, MD (1) · Nicola Brunetti-Pierri (1) · Stuart J Swiedler, MD, Ph.D. (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 37955580, PMID 38319253, PMID 39547230, PMID 39891377, PMID 9918480