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My Organizations' Clinical Trials

Showing 75 of 107 entries
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A Prospective Clinical Study of Phenylketonuria (PKU)

Phase: N/A Priority: Normal
NCT ID: NCT04452513 Org: User:
Start: 10/11/19 End: 06/02/22 Due: 06/02/23

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

Phase: N/A Priority: Normal
NCT ID: NCT05768386 Org: User:
Start: 01/01/23 End: 01/31/40 Due: 01/31/41

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

Phase: N/A Priority: Normal
NCT ID: NCT02724228 Org: User:
Start: 01/26/16 End: 02/29/28 Due: 02/28/29

Sapropterin Expanded Access Program

Phase: N/A Priority: Normal
NCT ID: NCT00484991 Org: User:
Start: - End: - Due: -

Gene Therapy Study in Severe Hemophilia a Patients with Antibodies Against AAV5

Phase: N/A Priority: Normal
NCT ID: NCT03520712 Org: User:
Start: 04/03/18 End: 08/07/24 Due: 08/07/25

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

Phase: N/A Priority: Normal
NCT ID: NCT00104247 Org: User:
Start: 03/31/05 End: 02/28/06 Due: 02/28/07

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

Phase: N/A Priority: Normal
NCT ID: NCT01803412 Org: User:
Start: 05/01/13 End: 10/01/16 Due: 10/01/17

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

Phase: N/A Priority: Normal
NCT ID: NCT00838435 Org: User:
Start: 02/28/09 End: 09/14/18 Due: 09/14/19

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Phase: N/A Priority: Normal
NCT ID: NCT00214773 Org: User:
Start: 07/31/05 End: 05/01/20 Due: 05/01/21

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Phase: N/A Priority: Normal
NCT ID: NCT03694353 Org: User:
Start: 09/13/18 End: 01/13/21 Due: 01/13/22

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

Phase: N/A Priority: Normal
NCT ID: NCT01016392 Org: User:
Start: 12/31/09 End: 05/31/21 Due: 05/31/22

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Phase: N/A Priority: Normal
NCT ID: NCT01515956 Org: User:
Start: 10/31/11 End: 02/29/16 Due: 02/28/17

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

Phase: N/A Priority: Normal
NCT ID: NCT01697319 Org: User:
Start: 08/31/12 End: 10/31/14 Due: 10/31/15

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Phase: N/A Priority: Normal
NCT ID: NCT00778206 Org: User:
Start: 09/30/08 End: 01/29/21 Due: 01/29/22

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

Phase: N/A Priority: Normal
NCT ID: NCT00332189 Org: User:
Start: 07/31/06 End: 08/31/09 Due: 08/31/10

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

Phase: N/A Priority: Normal
NCT ID: NCT06780332 Org: User:
Start: 01/31/25 End: 11/30/25 Due: 11/30/26

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Phase: N/A Priority: Normal
NCT ID: NCT00925054 Org: User:
Start: 09/30/09 End: 06/30/15 Due: 06/30/16

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

Phase: N/A Priority: Normal
NCT ID: NCT03989947 Org: User:
Start: 06/12/19 End: 05/31/38 Due: 05/31/39

VIrtual STudy in Achondroplasia for the US (VISTA)

Phase: N/A Priority: Normal
NCT ID: NCT06168201 Org: User:
Start: 02/21/23 End: 02/29/28 Due: 02/28/29

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Phase: N/A Priority: Normal
NCT ID: NCT05813678 Org: User:
Start: 06/06/22 End: 11/01/33 Due: 11/01/34

A Study to Assess Growth in Children With Idiopathic Short Stature

Phase: N/A Priority: Normal
NCT ID: NCT06309979 Org: User:
Start: 08/08/24 End: 12/31/40 Due: 12/31/41

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Phase: N/A Priority: Normal
NCT ID: NCT06224907 Org: User:
Start: 12/25/23 End: 03/31/29 Due: 03/31/30

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

Phase: N/A Priority: Normal
NCT ID: NCT04684940 Org: User:
Start: 12/10/20 End: 04/30/29 Due: 04/30/30

Study of rhASB in Patients With Mucopolysaccharidosis VI

Phase: N/A Priority: Normal
NCT ID: NCT00104234 Org: User:
Start: 02/29/04 End: 10/31/06 Due: 10/31/07

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

Phase: N/A Priority: Normal
NCT ID: NCT03505125 Org: User:
Start: 03/31/18 End: 07/31/19 Due: 07/31/20