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Incompliant submissions 0
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My Organizations' Clinical Trials

Showing 25 of 107 entries
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Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Phase: N/A Priority: Normal
NCT ID: NCT01230801 Org: User:
Start: 01/17/11 End: 03/06/13 Due: 03/06/14

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Phase: N/A Priority: Normal
NCT ID: NCT02678689 Org: User:
Start: 01/22/16 End: 04/20/22 Due: 04/20/23

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT04554940 Org: User:
Start: 10/10/20 End: 12/31/27 Due: 12/31/28

Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Phase: N/A Priority: Normal
NCT ID: NCT01977820 Org: User:
Start: 02/28/14 End: 11/30/14 Due: 11/30/15

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Phase: N/A Priority: Normal
NCT ID: NCT01609062 Org: User:
Start: 04/30/12 End: 11/30/14 Due: 11/30/15

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

Phase: N/A Priority: Normal
NCT ID: NCT01387854 Org: User:
Start: 06/30/11 End: 02/28/13 Due: 02/28/14

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Phase: N/A Priority: Normal
NCT ID: NCT03392974 Org: User:
Start: 03/14/18 End: 06/05/23 Due: 06/05/24

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Phase: N/A Priority: Normal
NCT ID: NCT02191917 Org: User:
Start: 10/21/14 End: 06/22/16 Due: 06/22/17

Extension Study of Drisapersen in DMD Subjects

Phase: N/A Priority: Normal
NCT ID: NCT02636686 Org: User:
Start: - End: - Due: -

Lifetime Impact of Achondroplasia Study in Europe-LIAISE

Phase: N/A Priority: Normal
NCT ID: NCT03449368 Org: User:
Start: 12/17/17 End: 04/29/20 Due: 04/29/21

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Phase: N/A Priority: Normal
NCT ID: NCT01753804 Org: User:
Start: 09/01/12 End: 10/01/16 Due: 10/01/17

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Phase: N/A Priority: Normal
NCT ID: NCT01907087 Org: User:
Start: 09/30/13 End: 11/30/15 Due: 11/30/16

A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

Phase: N/A Priority: Normal
NCT ID: NCT00445978 Org: User:
Start: 05/31/07 End: 06/30/09 Due: 06/30/10

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Phase: N/A Priority: Normal
NCT ID: NCT00787995 Org: User:
Start: 10/31/08 End: 07/31/14 Due: 07/31/15

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT06212947 Org: User:
Start: 11/27/23 End: 12/31/43 Due: 12/31/44

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

Phase: N/A Priority: Normal
NCT ID: NCT01275066 Org: User:
Start: 02/28/11 End: 08/31/12 Due: 08/31/13

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Phase: N/A Priority: Normal
NCT ID: NCT06738017 Org: User:
Start: 02/21/25 End: 08/31/25 Due: 08/31/26

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

Phase: N/A Priority: Normal
NCT ID: NCT01889862 Org: User:
Start: 07/29/13 End: 02/05/19 Due: 02/05/20

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Phase: N/A Priority: Normal
NCT ID: NCT01603095 Org: User:
Start: 04/30/12 End: 02/28/21 Due: 02/28/22

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

Phase: N/A Priority: Normal
NCT ID: NCT05121376 Org: User:
Start: 02/15/22 End: 11/30/28 Due: 11/30/29

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Phase: N/A Priority: Normal
NCT ID: NCT01924845 Org: User:
Start: 04/30/14 End: 09/12/16 Due: 09/12/17

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

Phase: N/A Priority: Normal
NCT ID: NCT01826474 Org: User:
Start: 01/31/13 End: 08/31/16 Due: 08/31/17

A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria

Phase: N/A Priority: Normal
NCT ID: NCT06138327 Org: User:
Start: 09/26/23 End: 03/25/24 Due: 03/25/25

Kuvan®'s Effect on the Cognition of Children With Phenylketonuria

Phase: N/A Priority: Normal
NCT ID: NCT01965912 Org: User:
Start: 10/31/13 End: 01/04/23 Due: 01/04/24

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Phase: N/A Priority: Normal
NCT ID: NCT02294877 Org: User:
Start: 09/30/14 End: 02/21/24 Due: 02/21/25