Biomarin Pharmaceutical

🇨🇦Canada

Country: 🇨🇦Canada

Ownership: -

Established: 1997-01-01

Employees: -

Market Cap: $17.2B

Website: http://www.biomarin.com

Clinical Trials

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

Phase 1

Active, not recruiting

Conditions: Hemophilia a
Gene Therapy
Clotting Disorders
Blood Disorder

Interventions: Biological: Valoctocogene Roxaparvovec

First Posted Date: 2018-05-11

Last Posted Date: 2024-04-24

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 3

Registration Number: NCT03520712

Locations: 🇨🇳
Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung, Taiwan
🇨🇳
National Taiwan University Hospital, Taipei, Taiwan
🇰🇷
Kyung Hee University Hospital at Gangdong, Seoul, Korea, Republic of

and more 6 locations

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

Completed

Conditions: Phenylketonuria (PKU)

First Posted Date: 2018-04-23

Last Posted Date: 2020-01-14

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 63

Registration Number: NCT03505125

Locations: 🇨🇦
Adult Metabolic Diseases Clinic, Vancouver General Hospital, Vancouver, British Columbia, Canada
🇫🇷
Necker Children's Hospital, Paris, Cedex 15, France
🇩🇪
Rheumatology Unit, Clinic for Inborn Errors of Metabolism, Leipzig, Germany

and more 11 locations

Lifetime Impact of Achondroplasia Study in Europe-LIAISE

Completed

Conditions: Achondroplasia

First Posted Date: 2018-02-28

Last Posted Date: 2021-04-05

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 196

Registration Number: NCT03449368

Locations: 🇦🇹
Medizinische Universitat Wien, Vienna, Austria
🇮🇹
Maternal and Child Health - University of Genova, Genova, Italy
🇩🇪
Klinik für Kinder- und Jugendmedizin, Cologne, Germany

and more 10 locations

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

Phase 3

Active, not recruiting

Conditions: Achondroplasia

Interventions: Drug: BMN 111

First Posted Date: 2018-02-06

Last Posted Date: 2024-10-09

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 119

Registration Number: NCT03424018

Locations: 🇯🇵
Saitama Children's Medical Center, Saitama, Japan
🇩🇪
Universitätsklinikum Münster, Münster, Germany
🇪🇸
Institut Catala de Traumatologica I Medicina de l'Esport, Barcelona, Spain

and more 21 locations

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

Phase 3

Completed

Conditions: Hemophilia A

Interventions: Biological: Valoctocogene Roxaparvovec

First Posted Date: 2018-01-08

Last Posted Date: 2023-10-03

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 1

Registration Number: NCT03392974

Locations: 🇺🇸
Hemophilia Center of Western Pennsylvania, Pittsburgh, Pennsylvania, United States

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

Phase 3

Active, not recruiting

Conditions: Hemophilia A

Interventions: Biological: valoctocogene roxaparvovec

First Posted Date: 2017-12-13

Last Posted Date: 2023-12-20

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 144

Registration Number: NCT03370913

Locations: 🇨🇳
Tri-Service General Hospital, Taipei, Taiwan
🇮🇱
Chaim Sheba Medical Center, Ramat Gan, Israel
🇩🇪
Vivantes Clinic im Friedrichshain- Landsberger Allee, Berlin, Germany

and more 45 locations

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

Phase 3

Completed

Conditions: Achondroplasia

Interventions: Drug: BMN 111
Drug: Placebo

First Posted Date: 2017-06-23

Last Posted Date: 2022-03-02

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 121

Registration Number: NCT03197766

Locations: 🇺🇸
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸
Emory University, Decatur, Georgia, United States
🇺🇸
Johns Hopkins University, Baltimore, Maryland, United States

and more 21 locations

A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

Conditions: CLN2 Disease

First Posted Date: 2016-11-15

Last Posted Date: 2017-07-24

Lead Sponsor: BioMarin Pharmaceutical

Registration Number: NCT02963350

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

Phase 2

Terminated

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: BMN 044 IV 6 mg/kg
Drug: BMN 044 IV 9 mg/kg
Drug: BMN 044 SC 6 mg/kg

First Posted Date: 2016-11-08

Last Posted Date: 2018-01-26

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 7

Registration Number: NCT02958202

Locations: 🇳🇱
Leiden University Medical Center, Leiden, Netherlands
🇮🇹
S.Anna Hospital, Ferrara, Italy
🇮🇹
Policlinico Univsersitario Agostino Gemelli, Rome, Italy

and more 2 locations

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

Phase 2

Active, not recruiting

Conditions: Achondroplasia

Interventions: Drug: BMN 111

First Posted Date: 2016-03-31

Last Posted Date: 2024-10-09

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 30

Registration Number: NCT02724228

Locations: 🇺🇸
Ann and Robert H. Lurie Childrens Hospital of Chicago, Chicago, Illinois, United States
🇺🇸
Children's Hospital & Research Center Oakland, Oakland, California, United States
🇺🇸
Harbor - UCLA Medical Center, Torrance, California, United States

and more 6 locations

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