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Incompliant submissions 0
Total trials 0

My Organizations' Clinical Trials

Showing 50 of 107 entries
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Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

Phase: N/A Priority: Normal
NCT ID: NCT00272792 Org: User:
Start: 02/28/06 End: 11/30/06 Due: 11/30/07

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

Phase: N/A Priority: Normal
NCT ID: NCT00067470 Org: User:
Start: 09/30/03 End: 03/31/04 Due: 03/31/05

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

Phase: N/A Priority: Normal
NCT ID: NCT01114737 Org: User:
Start: 08/31/10 End: 03/31/13 Due: 03/31/14

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Phase: N/A Priority: Normal
NCT ID: NCT00532844 Org: User:
Start: 09/30/07 End: 03/31/09 Due: 03/31/10

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Phase: N/A Priority: Normal
NCT ID: NCT06280209 Org: User:
Start: 01/03/24 End: 09/30/26 Due: 09/30/27

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Phase: N/A Priority: Normal
NCT ID: NCT02485899 Org: User:
Start: 02/28/15 End: 12/10/20 Due: 12/10/21

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Phase: N/A Priority: Normal
NCT ID: NCT02329769 Org: User:
Start: 12/31/14 End: 08/31/16 Due: 08/31/17

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Phase: N/A Priority: Normal
NCT ID: NCT00924703 Org: User:
Start: 01/13/10 End: 01/31/19 Due: 01/31/20

BMN 110 US Expanded Access Program

Phase: N/A Priority: Normal
NCT ID: NCT01858103 Org: User:
Start: - End: - Due: -

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Phase: N/A Priority: Normal
NCT ID: NCT04323098 Org: User:
Start: 12/08/20 End: 01/31/27 Due: 01/31/28

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

Phase: N/A Priority: Normal
NCT ID: NCT00355264 Org: User:
Start: 08/31/06 End: 06/30/09 Due: 06/30/10

Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Phase: N/A Priority: Normal
NCT ID: NCT01037309 Org: User:
Start: 12/31/09 End: 10/31/13 Due: 10/31/14

Retrospective Observational Safety Effectiveness With Kuvan in hpA

Phase: N/A Priority: Normal
NCT ID: NCT03864029 Org: User:
Start: 10/10/17 End: 07/25/18 Due: 07/25/19

Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

Phase: N/A Priority: Normal
NCT ID: NCT02365376 Org: User:
Start: 06/30/13 End: - Due: -

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

Phase: N/A Priority: Normal
NCT ID: NCT01590446 Org: User:
Start: 02/29/12 End: 06/30/12 Due: 06/30/13

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration

Phase: N/A Priority: Normal
NCT ID: NCT01910649 Org: User:
Start: 03/31/08 End: 09/30/16 Due: 09/30/17

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

Phase: N/A Priority: Normal
NCT ID: NCT01376908 Org: User:
Start: 06/30/11 End: 02/17/17 Due: 02/17/18

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT02055157 Org: User:
Start: 01/13/14 End: 10/02/17 Due: 10/02/18

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Phase: N/A Priority: Normal
NCT ID: NCT01242111 Org: User:
Start: 11/30/10 End: 07/31/14 Due: 07/31/15

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Phase: N/A Priority: Normal
NCT ID: NCT00299000 Org: User:
Start: 05/31/06 End: 04/30/09 Due: 04/30/10

BMN 110 Phase 3B in Australian Patients

Phase: N/A Priority: Normal
NCT ID: NCT01966029 Org: User:
Start: 07/31/13 End: 12/31/16 Due: 12/31/17

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Phase: N/A Priority: Normal
NCT ID: NCT02221362 Org: User:
Start: 12/31/14 End: 09/30/16 Due: 09/30/17

Extension Study for Patients Who Have Participated in a BMN 701 Study

Phase: N/A Priority: Normal
NCT ID: NCT01435772 Org: User:
Start: 08/15/11 End: 09/09/16 Due: 09/09/17

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Phase: N/A Priority: Normal
NCT ID: NCT01415427 Org: User:
Start: 07/31/11 End: 06/16/16 Due: 06/16/17

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

Phase: N/A Priority: Normal
NCT ID: NCT03197766 Org: User:
Start: 12/12/16 End: 10/30/19 Due: 10/30/20