11 terminated. 11 Phase 1, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 11 | Terminated 11 |
| Phase 2 | 8 | Terminated 8 |
Follow Mucinoses: new trials by email Literature Review of Mucinoses trials Research Report on Mucinoses
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 |
|---|---|---|---|---|---|---|
| Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease ABO-102 - MPS IIIA, Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 5 | 2019-09-18 | 2023-07-25 |
| Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB rAAV9.CMV.hNAGLU - Mucopolysaccharidosis Type 3 B | Phase 1 / Phase 2 | Terminated | Abeona Therapeutics, Inc | 11 | 2017-10-16 | 2022-05-05 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I SB-318 - MPS I | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 3 | 2017-05-24 | 2023-01-26 |
| Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II SB-913 - Mucopolysaccharidosis II, MPS II | Phase 1 / Phase 2 | Terminated | Sangamo Therapeutics | 9 | 2017-05-11 | 2022-10-25 |
| A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously SBC-103 - Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B | Phase 1 / Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 3 | 2015-10-15 | 2018-04-17 |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) rhHNS-10 mg, rhHNS-45 mg, rhHNS-90 mg - Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Shire | 12 | 2011-03-01 | 2021-06-14 |
| A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) BMN 110 - MPS IV A, Mucopolysaccharidosis IVA | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 20 | 2010-11-01 | 2015-09-30 |
| Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I laronidase - Spinal Cord Compression, Mucopolysaccharidosis I | Phase 1 | Terminated | Patricia I. Dickson, M.D. | 3 | 2008-01-01 | 2013-02-21 |
| Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I laronidase - Mucopolysaccharidosis I, Lysosomal Storage Diseases | Phase 1 | Terminated | Patricia I. Dickson, M.D. | 4 | 2005-11-01 | 2013-02-21 |
Mucopolysaccharidosis III (5) · Lysosomal Storage Diseases (4) · Mucopolysaccharidosis I (4) · Mucopolysaccharidoses (3) · Mucopolysaccharidosis II (2) · Spinal Cord Compression (2) · Sudden Infant Death (2) · Adrenoleukodystrophy (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Congenital Bone Marrow Failure Syndromes (1) · Dyskeratosis Congenita (1) · Fanconi Anemia (1) · Granulomatous Disease, Chronic (1) · Hemoglobinopathies (1)
Carbohydrate Metabolism, Inborn Errors (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Connective Tissue Diseases (11) · Genetic Diseases, Inborn (11) · Metabolism, Inborn Errors (11) · Nutritional and Metabolic Diseases (11) · Skin and Connective Tissue Diseases (11) · Metabolic Diseases (10) · Mucopolysaccharidoses (8) · Lysosomal Storage Diseases (7) · Nervous System Diseases (5) · Central Nervous System Diseases (4) · Genetic Diseases, X-Linked (3) · Pathologic Processes (3)
Iduronidase (2) · GALNS protein, human (1) · Stem Cell Transplantation (1)
Patricia I. Dickson, M.D. (2) · Sangamo Therapeutics (2) · Abeona Therapeutics, Inc (1) · Alexion Pharmaceuticals, Inc. (1) · Bellicum Pharmaceuticals (1) · BioMarin Pharmaceutical (1) · Shire (1) · Talaris Therapeutics Inc. (1) · Ultragenyx Pharmaceutical Inc (1)
United States (8) · United Kingdom (3) · Finland (2) · Spain (2) · Australia (1) · France (1) · Germany (1) · Netherlands (1)
Celeste Decker, MD (1) · Patricia I Dickson, M.D. (1) · Patricia I Dickson, MD (1) · Suzanne T Ildstad, MD (1)
PMID 36299240, PMID 22547151, PMID 25421091, PMID 29526614, PMID 32278382, PMID 32884151, PMID 34600820
2015 (1), 2018 (1)