19 completed, 6 terminated, 4 active not recruiting, 3 recruiting. 12 Phase 2, 7 Phase 1, 4 Phase 3, 1 Not applicable, 1 Phase 4.
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 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Registry of Patients Diagnosed With Lysosomal Storage Diseases There is no intervention - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Recruiting | University of California, San Francisco | 250 | 2022-05-31 | 2026-04-08 | |
| 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 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| 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 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) Lysosomal Storage Disease, Lung Diseases | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| A Study in MPS VI to Assess Safety and Efficacy of Odiparcil Odiparcil, Placebo - Mucopolysaccharidosis VI | Phase 2 | Completed | Inventiva Pharma | 20 | 2017-12-30 | 2019-11-01 |
| MPS (RaDiCo Cohort) (RaDiCo-MPS) Mucopolysaccharidosis I, Mucopolysaccharidosis II | Recruiting | Institut National de la Santé Et de la Recherche Médicale, France | 1000 | 2017-12-20 | 2026-02-11 | |
| 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 |
| Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome Naglazyme® - Maroteaux-Lamy Syndrome | Not applicable | Terminated | Masonic Cancer Center, University of Minnesota | 1 | 2016-01-26 | 2020-12-23 |
| 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 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project) Mucopolysaccharidosis (MPS) | Terminated | Shire | 159 | 2014-05-21 | 2021-03-17 | |
| Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome Morquio Syndrome A, Maroteaux Lamy Syndrome | Completed | Greenwood Genetic Center | 17 | 2013-10-01 | 2015-08-13 | |
| 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 |
| Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease Enzyme testing - Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI | Completed | Children's Hospitals and Clinics of Minnesota | 22 | 2012-10-01 | 2015-08-04 | |
| Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network Mucopolysaccharidoses | Completed | University of Minnesota | 1147 | 2012-03-01 | 2021-02-24 |
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Mucopolysaccharidosis I (18) · Mucopolysaccharidosis II (15) · Mucopolysaccharidosis IV (12) · Mucopolysaccharidoses (11) · Mucopolysaccharidosis VII (10) · Leukodystrophy, Metachromatic (9) · Leukodystrophy, Globoid Cell (8) · Wolman Disease (8) · Sudden Infant Death (7) · Fucosidosis (6) · Gaucher Disease (6) · Mucopolysaccharidosis III (6) · Adrenoleukodystrophy (5) · Metabolism, Inborn Errors (5)
Carbohydrate Metabolism, Inborn Errors (36) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (36) · Connective Tissue Diseases (36) · Metabolic Diseases (36) · Mucinoses (36) · Nutritional and Metabolic Diseases (36) · Skin and Connective Tissue Diseases (36) · Genetic Diseases, Inborn (35) · Lysosomal Storage Diseases (33) · Metabolism, Inborn Errors (31) · Mucopolysaccharidoses (25) · Nervous System Diseases (21) · Genetic Diseases, X-Linked (17) · Heredodegenerative Disorders, Nervous System (17) · Intellectual Disability (17)
N-Acetylgalactosamine-4-Sulfatase (4) · Stem Cell Transplantation (4) · Busulfan (3) · Cyclophosphamide (3) · galsulfase (3) · Adalimumab (2) · Alemtuzumab (2) · Bone Marrow Transplantation (2) · Clinical Enzyme Tests (1) · Cyclosporine (1) · GAA protein, human (1) · GALNS protein, human (1) · Growth Hormone (1) · Human Growth Hormone (1) · Iduronidase (1)
BioMarin Pharmaceutical (7) · Masonic Cancer Center, University of Minnesota (5) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (3) · University of California, San Francisco (3) · University of Minnesota (2) · Albert Einstein College of Medicine (1) · Aldagen (1) · CENTOGENE GmbH Rostock (1) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospitals and Clinics of Minnesota (1) · Fairview University Medical Center (1) · Fondazione Telethon (1) · Greenwood Genetic Center (1) · Hospital de Clinicas de Porto Alegre (1) · Institut National de la Santé Et de la Recherche Médicale, France (1)
United States (30) · France (5) · Germany (4) · Portugal (4) · United Kingdom (4) · Italy (3) · Australia (2) · Belgium (2) · Brazil (2) · Canada (2) · Turkey (Türkiye) (2) · Austria (1) · Egypt (1) · India (1) · Ireland (1)
Paul Orchard, MD (3) · Aaron S Kelly, Ph.D. (1) · Ashley Schneider (1) · Celeste Decker, MD (1) · Chester B. Whitley, M.D., Ph.D. (1) · Chester B. Whitley, Ph.D., M.D. (1) · Curt Scharfe, MD, PhD (1) · Elizabeth Braulin, M.D. (1) · Ellen B Fung, PhD (1) · Guilherme Baldo, PhD (1) · Heather Lau, M.D. (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1)
PMID 33874971, PMID 15324318, PMID 19948615, PMID 21586746, PMID 21637107, PMID 22867884, PMID 22974573, PMID 23510062, PMID 24108527, PMID 24368159, PMID 24764221, PMID 25541100, PMID 26095521, PMID 26303610, PMID 26873528, PMID 27114913, PMID 27114914, PMID 27339555, PMID 28374935, PMID 28657663, PMID 28983455, PMID 29751845, PMID 30337566, PMID 31315600, PMID 32102934, PMID 33775523, PMID 35012890, PMID 35142618, PMID 37955580, PMID 38319253, PMID 39547230, PMID 39891377, PMID 9918480
U54NS065768 (3), 2019 (1), R01HD073292 (1)