| recombinant human alkaline phosphatase intravenous (recAP) / AM Pharma |
2019-004625-24: Study to Investigate the Efficacy and Safety of Alkaline Phosphatase in Patients With Sepsis-Associated Acute Kidney Injury |
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| Not yet recruiting | 3 | 1600 | Europe, RoW | Recombinant Human Alkaline Phosphatase, recAP, Solution for infusion | AM-Pharma B.V., AM-Pharma B.V. | Sepsis-associated acute kidney injury, Sepsis-associated acute kidney injury, Body processes [G] - Immune system processes [G12] | | | | |
NCT05890794: Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia |
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| Completed | 1/2 | 12 | Europe | Ilofotase Alfa, 0.8 mg/kg, recAP, Ilofotase Alfa, 3.2 mg/kg | AM-Pharma | Hypophosphatasia | 07/23 | 07/23 | | |
| Fabagal (fabrazyme biosimilar) / ISU Abxis |
NCT06081062: Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease |
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| Recruiting | 3 | 24 | RoW | Fabagal® (Agalsidase beta), Active comparator (Agalsidase beta) | ISU Abxis Co., Ltd. | Fabry Disease | 06/25 | 12/25 | | |
| Aldurazyme (laronidase) / BioMarin, Sanofi |
2006-005216-27: Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. |
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| Ongoing | 4 | 1 | Europe | Aldurazyme | HUS, Hospital for Children and Adolescents | Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respiratory dysfunction, and in severe fore, mental retardation | | | | |
2007-001453-26: Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. |
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| Ongoing | 4 | 50 | Europe | idursulfase, Naglazyme, Aldurazyme, Elaprase, Naglazyme, Aldurazyme, Elaprase, Naglazyme, Aldurazyme | | Mucopolysaccharidosis type I, II and VI. | | | | |
NCT05134571: China Post-marketing Surveillance (PMS) Study of Aldurazyme® |
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| Completed | 4 | 12 | RoW | Laronidase | Genzyme, a Sanofi Company | Mucopolysaccharidosis I | 07/23 | 07/23 | | |
2023-001027-16: China post-marketing surveillance (PMS) study of Aldurazyme® |
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| Not yet recruiting | 4 | 16 | RoW | Aldurazyme, Concentrate for solution for infusion, Aldurazyme® | Genzyme Europe B. V, Genzyme Europe B. V | Mucopolysaccharidosis I, Mucopolysaccharidosis I, Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
NCT00418821 / 2007-007003-33: A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants |
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| Terminated | 4 | 2 | Europe | Laronidase, Aldurazyme, Recombinant human alpha L iduronidase | Genzyme, a Sanofi Company, BioMarin/Genzyme LLC | Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie | 12/22 | 12/22 | | |
2015-003031-35: Immune Tolerance Induction with Methotrexate in Hurler Syndrome |
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| Ongoing | 3 | 4 | Europe | Methotrexate, Oral solution | Central Manchester University Hospitals NHS Foundation Trust | Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH), MPS IH is an inherited disorder caused by an enzyme deficiency. Complex molecules accumulate in the body, affecting multiple organs and causing skeletal problems and developmental delay., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
NCT06406153: Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients |
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| Completed | 3 | 12 | RoW | Laronidase, Antihistamine, Antipyretic | Cinnagen | Mucopolysaccharidosis Type 1 | 08/23 | 11/23 | | |
| Recruiting | 1 | 10 | US | Aldurazyme (laronidase), Elaprase (idursulfase), Vimizim (elosulfase alfa), Naglazyme (galsulfase), Mepsevii (vestronidase alfa-vjbk), Lumizyme (alglucosidase alfa), Kanuma (sebelipase alfa) | University of California, San Francisco, Duke University | MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease | 07/31 | 07/32 | | |
HomERT, NCT05073783: A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting |
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| Completed | N/A | 57 | Europe | | Sanofi | Pompe Disease, Mucopolysaccharidosis Type I (MPS I) | 01/24 | 01/24 | | |
NCT05634512: Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH. |
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| Recruiting | N/A | 24 | US | Laronidase therapy and a stem cell transplant | Masonic Cancer Center, University of Minnesota | Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I | 10/24 | 10/25 | | |
| Naglazyme (galsulfase) / AnGes MG, BioMarin |
NCT05824663: A Study Evaluating the Safety, Tolerance and Anti-tumor Activity of HBM1020 in Subjects With Advanced Solid Tumors |
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| Not yet recruiting | 1 | 50 | NA | HBM1020 | Harbour BioMed US, Inc. | Advanced Solid Tumor | 12/24 | 06/25 | | |
| Kanuma (sebelipase alfa) / AstraZeneca |
NCT02376751: An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency |
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| No Longer Available | N/A | | US | sebelipase alfa, SBC-102, recombinant human lysosomal acid lipase, rhLAL | Alexion Pharmaceuticals | Lysosomal Acid Lipase Deficiency | | | | |
| Elfabrio (pegunigalsidase alfa-iwxj) / Protalix, Chiesi |
| Active, not recruiting | 3 | 29 | Europe, US, RoW | pegunigalsidase alfa, PRX-102 | Chiesi Farmaceutici S.p.A. | Fabry Disease | 09/24 | 12/24 | | |
| Active, not recruiting | 3 | 97 | Europe, Canada, US, RoW | pegunigalsidase alfa, PRX-102 | Chiesi Farmaceutici S.p.A. | Fabry Disease | 01/25 | 04/25 | | |
FLY, NCT06328608: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease |
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| Not yet recruiting | 2/3 | 22 | NA | PRX-102 1 mg/kg every two weeks, pegunigalsidase alfa, Recombinant human alpha galactosidase-A | Chiesi Farmaceutici S.p.A., ICON plc | Fabry Disease | 12/27 | 03/28 | | |
RISE, NCT05710692: Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease |
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| Recruiting | 2/3 | 18 | Japan | PRX-102 1 mg/kg every 2 weeks, pegunigalsidase alfa, Recombinant human alpha galactosidase-A, PRX-102 2 mg/kg every 4 weeks | Chiesi Farmaceutici S.p.A., ICON plc | Fabry Disease | 03/26 | 03/28 | | |
NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients |
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| Approved for marketing | N/A | | US | Pegunigalsidase Alfa | Chiesi Farmaceutici S.p.A., Chiesi USA, Inc. | Fabry Disease | | | | |
GoPEG, NCT06095713: German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa |
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| Recruiting | N/A | 60 | Europe | Pegunigalsidase-alfa | Westfälische Wilhelms-Universität Münster, Chiesi GmbH | Fabry Disease | 03/26 | 03/26 | | |
| recombinant-iduronate-2-sulfatase (JR-032) / GSK, JCR Pharma |
| Recruiting | 3 | 80 | Europe, US, RoW | JR-141, Idursulfase, JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 01/26 | 01/26 | | |
| Cerezyme (imiglucerase) / Sanofi |
SEED, NCT04656600: Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ |
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| Completed | 4 | 12 | RoW | Cerezyme® / Imiglucerase | Sanofi | Gaucher's Disease | 10/23 | 10/23 | | |
2024-000041-27: Phase IV study to evaluate efficacy and safety of imiglucerase treatment in Chinese patients with Gaucher disease type Ⅲ |
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| Not yet recruiting | 4 | 12 | RoW | cerezyme, Powder for concentrate for solution for infusion, Cerezyme® | SANOFI (CHINA) INVESTMENT CO., LTD, SANOFI (CHINA) INVESTMENT CO., LTD | Gaucher's disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
2021-005402-10: Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 |
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| Ongoing | 3 | 50 | Europe | venglustat, SAR402671, GZ402671 or GZ / SAR402671, Tablet, Powder for concentrate for solution for infusion, Cerezyme 400 Units Powder for concentrate for solution for infusion | Sanofi-aventis recherche & développement, Sanofi-aventis recherche & développement | Gaucher's disease type III, Gaucher's disease type III, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
| Recruiting | 3 | 40 | Europe, Canada, Japan, US, RoW | Venglustat, imiglucerase, Cerezyme® | Sanofi, Sanofi-aventis recherche & développement | Gaucher's Disease Type III | 09/25 | 10/26 | | |
| Active, not recruiting | 3 | 57 | Europe, Canada, Japan, RoW | Eliglustat GZ385660, Cerdelga, Imiglucerase GZ437843, Cerezyme | Sanofi | Gaucher's Disease Type I, Gaucher's Disease Type III | 11/25 | 11/25 | | |
2020-003120-17: Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3 |
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| Ongoing | 2/3 | 12 | Europe | Venglustat, GZ402671, Tablet | Genzyme Corporation, Genzyme Corporation | Gaucher disease type 3, Gaucher disease type 3, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
| Active, not recruiting | 2 | 12 | Europe, Japan, US | venglustat (GZ402671), imiglucerase, Cerezyme | Genzyme, a Sanofi Company | Gaucher Disease Type 1, Gaucher Disease Type 3 | 09/25 | 09/25 | | |
2010-019622-13: Etude de la Cinétique IntraMonocytaire de l’Imiglucérase (CIMI) et de sa corrélation avec l’évolution clinique et biologique de la maladie de Gaucher |
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| Ongoing | 1 | 60 | Europe | imiglucérase, Cerezyme®, Cerezyme® | CHU de Clermont-Ferrand | Maladie de Gaucher | | | | |
NCT04370665: Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease |
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| Active, not recruiting | N/A | 4 | Canada | Exablate BBBD with Cerezyme | InSightec | Parkinson Disease | 12/21 | 12/22 | | |
| Fabrazyme (agalsidase beta) / Sanofi |
NCT05054387: China Post-marketing Surveillance (PMS) Study of Fabrazyme® |
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| Completed | 4 | 22 | RoW | Agalsidase beta, GZ419828 Fabrazyme | Genzyme, a Sanofi Company | Fabry Disease | 03/23 | 03/23 | | |
2023-000624-11: China post-marketing surveillance (PMS) study of Fabrazyme® |
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| Not yet recruiting | 4 | 18 | RoW | Powder for solution for infusion, Fabrazyme | Genzyme Europe B.V., Genzyme Europe B.V. | Fabry’s disease, Fabry’s disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
| Terminated | 4 | 7 | Europe, US | agalsidase beta, r-haGAL, Fabrazyme | Genzyme, a Sanofi Company | Fabry Disease, Alpha Galactosidase A Deficiency | 02/24 | 02/24 | | |
SHORTEN, NCT06019728: A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme |
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| Recruiting | 4 | 18 | US | AGALSIDASE BETA (GZ419828), Fabrazyme, Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast | Sanofi | Fabry's Disease | 10/25 | 10/25 | | |
2021-002320-20: A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease Un estudio para evaluar el efecto de comprimidos de venglustat sobre el índice de masa ventricular izquierda en pacientes hombres y mujeres adultos con enfermedad de Fabry. |
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| Ongoing | 3 | 110 | Europe | Venglustat, SAR402671, GZ402671 or GZ/SAR402671, Tablet, Concentrate for solution for infusion, Powder for concentrate for solution for infusion, Capsule, hard, Replagal, Fabrazyme, Galafold | sanofi-aventis recherche et développement, sanofi-aventis recherche et développement | Fabry’s disease Enfermedad de Fabry, Fabry’s disease Enfermedad de Fabry, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
| Recruiting | 3 | 90 | Europe, Canada, Japan, US, RoW | Venglustat (GZ402671), Agalsidase alfa, Replagal®, Agalsidase beta (GZ419828), Fabrazyme®, Migalastat, Galafold® | Sanofi, sanofi-aventis recherche et développement | Fabry Disease | 12/25 | 07/27 | | |
NCT05343715: PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion |
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| Completed | 1 | 24 | RoW | Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg | Bio Sidus SA | Fabry Disease | 03/22 | 04/22 | | |
NCT06052800: Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan |
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| Recruiting | N/A | 100 | RoW | | Sanofi | Fabry Disease | 09/26 | 09/26 | | |
NCT05698901: Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease |
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| Recruiting | N/A | 150 | RoW | Agalsidase beta | Mackay Memorial Hospital | Fabry Disease | 09/26 | 09/27 | | |
| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda |
NCT05067868: A Study of Replagal in Children and Adults With Fabry Disease in India |
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| Active, not recruiting | 4 | 5 | RoW | Replagal | Shire | Fabry Disease | 10/26 | 11/26 | | |
| Terminated | 3 | 17 | Europe, Canada | REPLAGAL, SHP675 | Shire | Fabry Disease | 12/22 | 12/22 | | |
NCT04974749: A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease |
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| Completed | 3 | 20 | RoW | REPLAGAL, Agalsidase Alfa, TAK-675 | Takeda | Fabry Disease | 01/24 | 01/24 | | |
2022-004246-35: A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease |
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| Not yet recruiting | 3 | 20 | RoW | Replagal, TAK-675, Concentrate for solution for injection/infusion, Replagal | Takeda Development Center Americas, Inc, Takeda Development Center Americas, Inc | Fabry Disease, Fabry Disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
NCT01031173: Treatment Protocol of Replagal for Patients With Fabry Disease |
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| No Longer Available | N/A | | US | agalsidase alfa, Replagal | Shire | Fabry Disease | | | | |
NCT03230591: Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy |
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| Recruiting | N/A | 25 | RoW | Echocardiography | Yonsei University | Fabry Disease | 01/25 | 01/25 | | |
| recombinant human arylsulfatase A (TAK-611) / Takeda |
2007-007165-20: A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients with high residual level of voluntary function |
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| Ongoing | 2 | 6 | Europe | Metazym, rhASA, | Shire Pharmaceuticals Ireland Limited | Metachromatic Leukodystrophy (MLD) in late infantile patients | | | | |
| Active, not recruiting | 2 | 36 | Europe, Canada, Japan, US, RoW | SHP611, HGT-1110, TAK-611, recombinant human arylsulfatase A [rhASA] | Shire, Takeda Development Center Americas, Inc. | Metachromatic Leukodystrophy (MLD) | 03/23 | 03/25 | | |
| Active, not recruiting | 1/2 | 24 | Europe, Japan, RoW | HGT-1110, Recombinant human arylsulfatase A | Shire, Takeda Development Center Americas, Inc. | Metachromatic Leukodystrophy (MLD) | 12/24 | 12/24 | | |
| Creon (pancrelipase delayed-release) / AbbVie, Eisai |
ACTRN12613000269730: Examining the effect of Creon(trademark) on patients who have had an oesphagectomy or gastrectomy. |
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| Recruiting | 4 | 24 | | | Box Hill Hospital Department of Surgery, Box Hill Hospital Surgical department | Pancreatic exocrine insufficiency (PEI) | | | | |
2013-002748-10: Clinical trial to assess the efficacy of pancreatic enzyme replacement therapy in patients with pancreatic exocrine insufficiency secondary to type 1 diabetes mellitus. Ensayo clínico para evaluar la eficacia del tratamiento sustitutivo con enzimas pancreáticas en pacientes con insuficiencia pancreática exocrina secundaria a diabetes mellitus tipo 1 |
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| Ongoing | 4 | 50 | Europe | Creon 25.000, PL 00512/0150, Capsule, Kreon 25.000 | Fundación para la Investigación en Enfermedades del Aparato Digestivo, Fundación para la Investigación en Enfermedades del Aparato Digestivo | Patients with pancreatic exocrine insufficiency (PEI) secondary to type 1 diabetes mellitus (DM) Pacientes con insuficiencia pancreática exocrina (IPE) secundaria a diabetes mellitus (DM) tipo 1, Patients with type 1 diabetes mellitus and maldigestion Pacientes con diabetes mellitus tipo 1 y maldigestión, Diseases [C] - Digestive System Diseases [C06] | | | | |
2013-005310-35: Pancreatic enzyme replacement therapy (PERT) in subjects with pancreatic exocrine insufficiency (PEI) after acute necrotizing pancreatitis tratamiento sustitutivo con enzimas pancreáticas (TSEP) en sujetos tras un episodio de pancreatitis aguda necrotizante que padecen insuficiencia exocrina pancreática (IPE) |
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| Ongoing | 4 | 68 | Europe | Creon 25000, PL 00512/0150, Kreon 25.000, Kreon 25.000 | Fundación para la Investigación en Enfermedades del Aparato Digestivo, Fundación para la Investigación en Enfermedades del Aparato Digestivo | Patients 2 weeks after discharge from the hospital after an attack of acute necrotizing pancreatitis Pacientes dos semanas tras el alta hospitalaria despues de un episodio de pancreatitis aguda necrotizante | | | | |
2015-002570-20: Optimizing treatment of maldigestion in patients with chronic pancreatitis |
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| Ongoing | 4 | 30 | Europe | Creon 25000, PL 00512/0150, Capsule, Kreon 25000 | Department of gastroenterology. University Hospital of Santiago, Dpt. of Gastroenterology. University Hospital of Santiago | Patients diagnosed of chronic pancreatitis with pancreatic exocrine insufficiency under pancreatic enzyme replacement therapy Pacientes diagnosticados de pancreatitis crónica con insuficiencia pancreática exocrina bajo tratamiento enzimático sustitutivo, Pancreatic exocrine insufficiency (maldigestion) in patients with chronic pancreatitis Insuficiencia pancreática exocrina en pacientes con pancreatitis crónica, Diseases [C] - Digestive System Diseases [C06] | | | | |
2017-001227-45: Pancreatic replacement therapy and glycaemic control in diabetes |
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| Ongoing | 4 | 18 | Europe | Creon, Capsule, Creon | Portsmouth Hospitals NHS Trust, Mylan | Type 1 diabetes and type 2 diabetes mellitusPancreatic exocrine insufficiency, DiabetesPancreatic insufficiency, Diseases [C] - Hormonal diseases [C19] | | | | |
2018-000242-19: Preoperative supplementation of pancreatic enzymes for Whipple’s procedure for malignancies (PREPARE). |
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| Ongoing | 4 | 128 | Europe | Creon 25000, Capsule, hard, Creon 25000 | University Hospitals Southampton NHS Trust, Mylan Products, Abbott Laboratories Hannover, Catalent Schorndorf | Patients undergoing Whipple’s procedure for pancreatic head tumours, Whipple procedure is a major surgical operation involving resection of the pancreas, duodenum, and other organs. This operation is performed to treat cancerous tumours on the head of the pancreas., Diseases [C] - Cancer [C04] | | | | |
2021-005874-24: Clinical trial to evaluate the efficacy of the oral treatment with pancreatic enzymes in patients with pancreatic cancer, who are not suitable for surgical therapy. Ensayo clínico para evaluar el impacto del tratamiento oral con enzimas pancreáticas en pacientes con cáncer de páncreas que no son candidatos a intervención quirúrgica. |
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| Not yet recruiting | 4 | 100 | Europe | Creon / Kreon 35000, Capsule, Creon 35,000 | Juan Enrique Domínguez Muñoz, Viatris | pancreatic exocrine insufficiency (PEI) in patients with unresectable pancreatic cancer., controlled trial of pancreatic enzyme replacement therapy (PERT) for pancreatic exocrine insufficiency (PEI) in patients with unresectable pancreatic cancer., Diseases [C] - Digestive System Diseases [C06] | | | | |
NCT05069597: Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon |
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| Completed | 4 | 30 | US | CREON, Pancrelipase | AbbVie | Cystic Fibrosis, Chronic Pancreatitis | 07/23 | 08/23 | | |
PERT-AP, NCT06477159: Pancreatic Enzyme Replacement Therapy for Acute Pancreatitis-Associated Exocrine Pancreatic Insufficiency |
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| Recruiting | 4 | 60 | US | Pancrelipase Capsules, CREON | Ohio State University, University of Pittsburgh, University of Illinois Chicago, University of Southern California, New York University, AbbVie | Exocrine Pancreatic Insufficiency | 12/26 | 07/27 | | |
| Not yet recruiting | 4 | 100 | NA | creon 35.000 Ph.U (R), Best Standarard of Care | Hospital Clinico Universitario de Santiago, Complejo Hospitalario de Navarra, Karolinska Institutet, San Raffaele University Hospital, Italy, Beaujon Hospital | Unresectable Pancreatic Cancer | 12/24 | 06/25 | | |
2004-005012-80: Double-blind, multicenter, randomized, placebo-controlled, parallel-group study to investigate the effect of Creon 25 000 Minimicrospheres on body mass index (BMI) in patients after gastrectomy. |
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| Ongoing | 3 | 6 | Europe | Creon enterocapsule | Solvay Pharmaceuticals GmbH | Patients atleast 18 years old with total or subtotal gastrektomy. | | | | |
| Recruiting | 3 | 60 | RoW | Creon® 25000, Creon® 10000 | Abbott | Pancreatic Insufficiency | 04/22 | 04/22 | | |
PB-SAM, NCT04542473: Pancreatic Enzymes and Bile Acids in Acutely Ill Severely Malnourished Children |
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| Active, not recruiting | 2/3 | 400 | RoW | Pancreatic Enzyme, CREON, Ursodeoxycholic acid, UDCAMENT, Pancreatic Enzyme placebo, Placebo-PE, Ursodeoxycholic acid placebo, Placebo-UA | University of Oxford, University of Amsterdam, University of Toronto, University of Washington, Oregon Health and Science University, Kenya Medical Research Institute, Queen Elizabeth Central Hospital, Blantyre, Malawi, Makerere University, KEMRI-Wellcome Trust Collaborative Research Program, International Centre for Diarrhoeal Disease Research, Bangladesh | SEPSIS, MALNUTRITION, CHILD | 10/23 | 06/24 | | |
| Enrolling by invitation | 1 | 10 | US | CREON, Placebo | Vanderbilt University Medical Center | Type 1 Diabetes | 08/24 | 02/25 | | |
ChiCTR2400086621: Study on the efficacy and safety of pancreatic enzyme replacement therapy for total enteral nutrition-related diarrhea in moderately to severely active Crohn's disease |
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| Recruiting | 1 | 100 | | The dosage of Creon (trypsin enteric-coated capsule) (according to the drug instruction manual): 1 tablet every time, once every 2 hours, 6 times a day, and then stop taking it for 4 weeks; Treat according to routine procedure. The control group did not receive trypsin replacement therapy | Department of Gastroenterology, Renmin Hospital of WuhanUniversity; Renmin Hospital of WuhanUniversity, self-finance | Crohn’s disease | | | | |
| Completed | N/A | 63 | US | CREON, pancrelipase, ABT-SLV245 | AbbVie | Chronic Pancreatitis | 02/24 | 02/24 | | |
| Pertzye (pancrelipase) / Chiesi |
NCT05642962: Pancrelipase in People With Pancreatic Ductal Adenocarcinoma (PDAC) |
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| Recruiting | 1/2 | 40 | US | Pertzye, Pancrelipase | Memorial Sloan Kettering Cancer Center | Metastatic Pancreatic Ductal Adenocarcinoma, Metastatic Pancreatic Cancer, Metastatic Pancreatic Adenocarcinoma, Metastatic Pancreatic Carcinoma, Pancreatic Carcinoma, Pancreatic Carcinoma Metastatic, Pancreatic Carcinoma Non-resectable, Pancreatic Cancer, Pancreatic Cancer Non-resectable, Pancreatic Cancer Stage IV, Pancreatic Cancer Metastatic, PDAC, PDAC - Pancreatic Ductal Adenocarcinoma | 11/24 | 11/24 | | |
| Izcargo (pabinafusp alfa) / JCR Pharma |
NCT05594992: An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects |
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| Enrolling by invitation | 3 | 80 | US | JR-141 | JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 02/28 | 02/28 | | |
NCT04348136: An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II |
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| Active, not recruiting | 2/3 | 27 | Japan | JR-141 | JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 03/30 | 03/30 | | |
NCT03708965: An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II |
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| Active, not recruiting | 2 | 19 | RoW | JR-141 | JCR Pharmaceuticals Co., Ltd. | Mucopolysaccharidosis II | 03/24 | 03/24 | | |
| SB-913 / Sangamo Therap |
2018-000192-33: A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in patients with Mucopolysaccharidosis II (MPS II) |
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| Not yet recruiting | 1/2 | 32 | Europe | rAAV2/6 Left ZFN Vector, rAAV2/6 Right ZFN Vector, rAAV 2/6 hIDS DONOR, SB-47171, SB-47898, hIDS DONOR, Solution for infusion | Sangamo Therapeutics, Inc., Sangamo Therapeutics, Inc. | Mucopolysaccharidosis type II (MPS II), Hunter Syndrome, is a rare, inherited disease caused by a deficiency in an enzyme called iduronate-2-sulfatase. This causes glycosaminoglycans (GAGs) to build up in the body and cause damage., Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
LTFU, NCT04628871: Long Term Follow-up () of Subjects Who Received SB-318, SB-913, or SB-FIX |
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| Active, not recruiting | N/A | 13 | US | SB-318, SB-913, SB-FIX | Sangamo Therapeutics | Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II | 01/30 | 01/30 | | |
| Strensiq (asfotase alfa) / AstraZeneca |
RESTORE, NCT06015750: Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia |
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| Not yet recruiting | 4 | 8 | Europe, US | methotrexate, rituximab, bortezomib, IVIg, Folic Acid | Alexion Pharmaceuticals, Inc. | Hypophosphatasia | 09/28 | 09/28 | | |
CHESTNUT, NCT06079372: Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa |
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| Recruiting | 3 | 40 | Europe, Canada, Japan, US, RoW | ALXN1850, asfotase alfa | Alexion Pharmaceuticals, Inc. | Hypophosphatasia | 06/25 | 01/28 | | |
| Recruiting | 3 | 30 | Europe, Canada, US, RoW | ALXN1850, Placebo | Alexion Pharmaceuticals, Inc. | Hypophosphatasia | 09/25 | 04/28 | | |
HICKORY, NCT06079281: Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa |
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| Recruiting | 3 | 114 | Europe, Canada, Japan, US, RoW | ALXN1850, Placebo | Alexion Pharmaceuticals, Inc. | Hypophosphatasia | 12/25 | 07/28 | | |
| Not yet recruiting | 3 | 114 | | ALXN1850; placebo | Peking Union Medical College Hospital; Peking Union Medical College Hospital, AZ | adolescent (≥ 12 to < 18 years of age at Day 1) and adult (≥ 18 years of age at Day 1) participants with HPP who have not previously been treated with asfotase alfa. | | | | |
NCT04195763: Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa) |
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| Completed | N/A | 50 | US | asfotase alfa, Strensiq® | Alexion Pharmaceuticals, Inc., Xcenda, LLC | Hypophosphatasia | 04/24 | 04/24 | | |
| Enrolling by invitation | N/A | 900 | Europe, Canada, US, RoW | | Alexion Pharmaceuticals, Inc. | Hypophosphatasia (HPP) | 12/31 | 12/31 | | |
NCT05234567: A Prospective Sub-Study of the Global Hypophosphatasia Registry |
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| Recruiting | N/A | 30 | US | Asfotase Alfa, Strensiq | Alexion Pharmaceuticals, Inc. | Hypophosphatasia | 08/29 | 08/29 | | |
| Elelyso (taliglucerase alfa) / Pfizer |
NCT04002830: A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease |
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| Completed | 4 | 14 | RoW | Elelyso, Taliglucerase Alfa | Ari Zimran, Pfizer | Gaucher Disease, Type 3 | 07/23 | 07/23 | | |
NCT05815004: An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 |
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| Withdrawn | 2/3 | 40 | NA | Gene therapy, AVR-RD-02, Enzyme Replacement Agent, Imiglucerase, velaglucerase, taliglucerase alfa | AVROBIO | Gaucher Disease, Type 3 | 12/27 | 12/27 | | |
NCT00962260: Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease |
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| No Longer Available | N/A | | US, RoW | Plant cell expressed recombinant glucocerebrosidase (prGCD), taliglucerase alfa | Pfizer | Gaucher Disease | | | | |
| adrulipase alfa (FW-EPI) / First Wave Bio |
SPAN, NCT05719311: Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis |
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| Completed | 2 | 13 | US | adrulipase, MS1819 | Entero Therapeutics | Exocrine Pancreatic Insufficiency, Cystic Fibrosis | 06/23 | 07/23 | | |
2010-018900-10: Safety and preliminary clinical activity of Yarrowia lipolytica lipase (MS1819) in patients with malabsorption syndrome due to exocrine pancreatic insufficiency resulting from chronic pancreatitis |
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| Ongoing | 1/2 | 12 | Europe | MS1819, MS1819, | Laboratoires Mayoly Spindler | Exocrine pancreatic insufficiency resulting from chronic pancreatitis | | | | |
ACTRN12616000962437: MS1819-SD phase IIa clinical trial for patients with exocrine pancreatic insufficiency (EPI) caused by chronic pancreatitis (CP) and/or distal pancreatectomy. |
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| Recruiting | N/A | 13 | | | INC Research Australia Pty Ltd, AzurRx | Exocrine pancreatic insufficiency (EPI) caused by chronic pancreatitis (CP) , Exocrine pancreatic insufficiency (EPI) caused by distal pancreatectomy. | | | | |
| Duoc-01 / Duke University Medical Center |
| Recruiting | 1 | 20 | US | DUOC-01 | Joanne Kurtzberg, MD | Primary Progressive Multiple Sclerosis | 08/25 | 08/25 | | |
DUOC-01, NCT02254863: UCB Transplant of Inherited Metabolic Diseases with Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells |
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| Recruiting | 1 | 40 | US | DUOC-01 | Joanne Kurtzberg, MD, The Marcus Foundation | Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses | 10/25 | 10/25 | | |
| recombinant lipase (CDX-7108) / Codexis, Nestle |
NCT05082051: Oral CDX-7108 in Healthy Adults and EPI Subjects |
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| Completed | 1 | 54 | RoW | Part A, Single Ascending Dose Study, Part B, Multiple Ascending Dose Study, Part C, Proof-of-Concept Study | Société des Produits Nestlé (SPN) | Exocrine Pancreatic Insufficiency | 03/23 | 03/23 | | |
| tividenofusp alfa (DNL310) / Denali Therap |
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) |
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| Recruiting | 2/3 | 54 | Europe, Canada, US, RoW | tividenofusp alfa, idursulfase | Denali Therapeutics Inc. | Mucopolysaccharidosis II | 12/25 | 12/25 | | |
NCT06075537: An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 |
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| Enrolling by invitation | 2/3 | 99 | Europe, Canada, US, RoW | tividenofusp alfa | Denali Therapeutics Inc. | Mucopolysaccharidosis II | 06/27 | 06/27 | | |
| Active, not recruiting | 1/2 | 47 | Europe, Canada, US | tividenofusp alfa | Denali Therapeutics Inc. | Mucopolysaccharidosis II | 07/27 | 07/27 | | |
| N-sulphoglucosamine sulphohydrolase (DNL126) / Denali Therap |
NCT06181136: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) |
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| Recruiting | 1/2 | 20 | US | DNL126 | Denali Therapeutics Inc. | Mucopolysaccharidosis Type IIIA | 08/28 | 08/28 | | |
| pegtarviliase (AGLE-177) / Aeglea |
| Terminated | 1/2 | 13 | Europe, US, RoW | Pegtarviliase IV, ACN00177, Pegtarviliase SC | Aeglea Biotherapeutics | Homocystinuria Due to Cystathionine Beta-Synthase Deficiency | 04/23 | 04/23 | | |
2019-004791-19: A Phase 1/2 Study of ACN00177 in Subjects With Homocystinuria Due to Cystathionine β-Synthase (CBS) Deficiency |
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| Not yet recruiting | 1 | 25 | Europe | ACN00177, Solution for injection | Aeglea Biotherapeutics, Inc., Aeglea Biotherapeutics | Homocystinuria Due to Cystathionine β-Synthase (CBS) Deficiency, Homocystinuria, Body processes [G] - Metabolic Phenomena [G03] | | | | |
| ANG003 / Anagram Therap |
NCT06052293: Phase 1 Study to Assess Safety and Efficacy of ANG003 |
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| Completed | 1 | 51 | US | ANG003, Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4. | Anagram Therapeutics, Inc. | Exocrine Pancreatic Insufficiency | 07/24 | 07/24 | | |
| INZ701 / Inozyme Pharma |
NCT06046820: The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency |
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| Recruiting | 3 | 33 | Europe, Canada, US, RoW | INZ-701, (rhENPP1-Fc)., Control Arm (Conventional Therapy) | Inozyme Pharma | Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy | 05/25 | 06/25 | | |
NCT06462547: ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency |
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| Recruiting | 2 | 200 | Europe, US | INZ-701, rhENPP1-Fc | Inozyme Pharma | Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets Type 2 | 11/30 | 12/30 | | |
2020-004000-33: A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of INZ-701 in Adults with ABCC6 Deficiency causing Pseudoxanthoma elasticum (PXE) |
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| Not yet recruiting | 1/2 | 9 | Europe | INZ-701, INZ-701, Lyophilisate for solution for injection | Inozyme Pharma, Inc., Inozyme Pharma, Inc. | Treatment of patients with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE), ABCC6 Deficiency causing a condition called Pseudoxanthoma elasticum, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
NCT04686175: Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency |
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| Active, not recruiting | 1/2 | 9 | Europe, Canada, US | INZ-701, rhENPP1-Fc | Inozyme Pharma | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy | 11/24 | 12/24 | | |
NCT05030831: Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE |
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| Active, not recruiting | 1/2 | 10 | Europe, US | INZ-701, rhENPP1-Fc | Inozyme Pharma, IQVIA Biotech | ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy | 11/24 | 12/24 | | |
