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Biomarin Pharmaceutical

🇨🇦Canada

Country: 🇨🇦Canada

Ownership: -

Established: 1997-01-01

Employees: -

Market Cap: $17.2B

Website: http://www.biomarin.com

Clinical Trials

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

Phase 2

Completed

Conditions: Achondroplasia

Interventions: Drug: Placebo
Drug: BMN 111

First Posted Date: 2018-07-11

Last Posted Date: 2024-06-13

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 75

Registration Number: NCT03583697

Locations: 🇺🇸
Children's Hospital & Research Center Oakland, Oakland, California, United States
🇺🇸
Harbor - UCLA Medical Center, Torrance, California, United States
🇺🇸
Alfred I. duPont Hospital for Children, Wilmington, Delaware, United States

and more 13 locations

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: 🇰🇷
Kyung Hee University Hospital at Gangdong, Seoul, Korea, Republic of
🇰🇷
Severance Hospital, Yonsei University Health System, Seoul, Korea, Republic of
🇿🇦
Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center, Johannesburg, South Africa

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: 🇺🇸
University of Florida, Gainesville, Florida, United States
🇺🇸
Emory University Department of Human Genetics, Decatur, Georgia, United States
🇺🇸
Ann and Robert H Lurie Children's Hospital, Chicago, Illinois, United States

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
🇩🇰
Aarhus Universitetshospital, Aarhus, Denmark
🇩🇪
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: 2025-01-09

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 119

Registration Number: NCT03424018

Locations: 🇺🇸
Children's Hospital & Research Center Oakland, Oakland, California, United States
🇺🇸
Harbor - UCLA Medical Center, Torrance, California, United States
🇺🇸
Alfred I. duPont Hospital for Children, Wilmington, Delaware, United States

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

Completed

Conditions: Hemophilia A

Interventions: Biological: valoctocogene roxaparvovec

First Posted Date: 2017-12-13

Last Posted Date: 2025-03-25

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 144

Registration Number: NCT03370913

Locations: 🇺🇸
Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, California, United States
🇺🇸
UC Davis Hemophilia Treatment Center, Sacramento, California, United States
🇺🇸
University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center, San Diego, California, United States

and more 46 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: 🇺🇸
Children's Hospital & Research Center Oakland, Oakland, California, United States
🇺🇸
Harbor - UCLA Medical Center, Torrance, California, United States
🇺🇸
Alfred I. duPont Hospital for Children, Wilmington, Delaware, 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: 🇧🇪
Universitair Ziekenhuis Leuven, Leuven, Belgium
🇮🇹
S.Anna Hospital, Ferrara, Italy
🇮🇹
Policlinico Univsersitario Agostino Gemelli, Rome, Italy

and more 2 locations

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