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My Organizations' Clinical Trials

Showing 100 of 107 entries
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A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

Phase: N/A Priority: Normal
NCT ID: NCT06668805 Org: User:
Start: 11/22/24 End: 09/30/41 Due: 09/30/42

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Phase: N/A Priority: Normal
NCT ID: NCT00048620 Org: User:
Start: 09/30/00 End: 11/30/05 Due: 11/30/06

A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

Phase: N/A Priority: Normal
NCT ID: NCT05580692 Org: User:
Start: 03/14/23 End: 07/31/24 Due: 07/31/25

Lifetime Impact Study for Achondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT03872531 Org: User:
Start: 03/31/19 End: 02/06/21 Due: 02/06/22

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

Phase: N/A Priority: Normal
NCT ID: NCT00884949 Org: User:
Start: 04/30/09 End: 03/31/11 Due: 03/31/12

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

Phase: N/A Priority: Normal
NCT ID: NCT01957059 Org: User:
Start: 06/30/13 End: 08/03/16 Due: 08/03/17

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

Phase: N/A Priority: Normal
NCT ID: NCT05579548 Org: User:
Start: 11/22/22 End: 10/04/32 Due: 10/04/33

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

Phase: N/A Priority: Normal
NCT ID: NCT00104260 Org: User:
Start: 12/31/04 End: 11/30/05 Due: 11/30/06

Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT06455059 Org: User:
Start: 06/06/24 End: 08/01/26 Due: 08/01/27

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Phase: N/A Priority: Normal
NCT ID: NCT00048711 Org: User:
Start: 03/31/02 End: 06/30/06 Due: 06/30/07

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

Phase: N/A Priority: Normal
NCT ID: NCT01212744 Org: User:
Start: 03/31/11 End: 04/30/15 Due: 04/30/16

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

Phase: N/A Priority: Normal
NCT ID: NCT05270837 Org: User:
Start: 06/17/22 End: 10/31/27 Due: 10/31/28

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

Phase: N/A Priority: Normal
NCT ID: NCT00634660 Org: User:
Start: 05/31/08 End: 10/31/09 Due: 10/31/10

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Phase: N/A Priority: Normal
NCT ID: NCT03370913 Org: User:
Start: 12/19/17 End: 11/20/24 Due: 11/20/25

A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature

Phase: N/A Priority: Normal
NCT ID: NCT06382155 Org: User:
Start: 10/21/24 End: 12/31/36 Due: 12/31/37

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

Phase: N/A Priority: Normal
NCT ID: NCT01819727 Org: User:
Start: 05/31/13 End: 11/25/15 Due: 11/25/16

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

Phase: N/A Priority: Normal
NCT ID: NCT05813314 Org: User:
Start: 03/15/23 End: 06/14/23 Due: 06/14/24

Cerliponase Alfa Observational Study in the US

Phase: N/A Priority: Normal
NCT ID: NCT04476862 Org: User:
Start: 08/19/20 End: 08/31/30 Due: 08/31/31

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

Phase: N/A Priority: Normal
NCT ID: NCT00789568 Org: User:
Start: 10/31/08 End: 10/31/09 Due: 10/31/10

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT03583697 Org: User:
Start: 06/13/18 End: 01/26/22 Due: 01/26/23

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Phase: N/A Priority: Normal
NCT ID: NCT02576795 Org: User:
Start: 09/28/15 End: 02/14/24 Due: 02/14/25

A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A

Phase: N/A Priority: Normal
NCT ID: NCT04560933 Org: User:
Start: 08/25/20 End: 10/20/23 Due: 10/20/24

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Phase: N/A Priority: Normal
NCT ID: NCT02468570 Org: User:
Start: 08/05/15 End: 02/01/17 Due: 02/01/18

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT03424018 Org: User:
Start: 12/12/17 End: 06/30/31 Due: 06/30/32

AAV Gene Therapy Study for Subjects with PKU

Phase: N/A Priority: Normal
NCT ID: NCT04480567 Org: User:
Start: 09/24/20 End: 12/31/27 Due: 12/31/28