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 Safety of Vosoritide in At-risk Infants With Achondroplasia

Phase 2

Active, not recruiting

Conditions: Achondroplasia

Interventions: Biological: vosoritide

First Posted Date: 2020-09-18

Last Posted Date: 2024-10-09

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 20

Registration Number: NCT04554940

Locations: 🇦🇺
Murdoch Children's Research Institute, Parkville, Victoria, Australia
🇬🇧
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital, London, United Kingdom
🇬🇧
Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom

AAV Gene Therapy Study for Subjects with PKU

Phase 1

Active, not recruiting

Conditions: Phenylketonuria (PKU)

Interventions: Drug: BMN 307

First Posted Date: 2020-07-21

Last Posted Date: 2024-12-12

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 100

Registration Number: NCT04480567

Locations: 🇺🇸
University of South Florida, Tampa, Florida, United States
🇺🇸
Morristown Medical Center, Morristown, New Jersey, United States
🇬🇧
University Hospital Birmingham NHS Foundation Trust, Birmingham, United Kingdom

Cerliponase Alfa Observational Study in the US

Active, not recruiting

Conditions: Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Interventions: Drug: Cerliponase Alfa
Device: Administration Kit

First Posted Date: 2020-07-20

Last Posted Date: 2024-01-31

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 35

Registration Number: NCT04476862

Locations: 🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States
🇺🇸
Children's Hospital of Orange County, Orange, California, United States
🇺🇸
Children's Hospital of Colorado, Aurora, Colorado, United States

and more 13 locations

A Prospective Clinical Study of Phenylketonuria (PKU)

Completed

Conditions: Phenylketonurias

First Posted Date: 2020-06-30

Last Posted Date: 2023-03-14

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 32

Registration Number: NCT04452513

Locations: 🇺🇸
Emory University Hospital, Atlanta, Georgia, United States
🇺🇸
Ann & Robert H. Lurie Chilren's Hospital, Chicago, Illinois, United States
🇺🇸
University of Kentucky, Lexington, Kentucky, United States

and more 11 locations

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

Phase 3

Active, not recruiting

Conditions: Hemophilia A

Interventions: Biological: valoctocogene roxaparvovec

First Posted Date: 2020-03-26

Last Posted Date: 2024-04-04

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 22

Registration Number: NCT04323098

Locations: 🇧🇷
Campinas University Clinical Hospital, Campinas, Brazil
🇦🇺
Fiona Stanley Hospital, Perth, Australia
🇦🇺
The Royal Adelaide Hospital, Adelaide, Australia

and more 9 locations

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

Phase 2

Active, not recruiting

Conditions: Achondroplasia

First Posted Date: 2019-06-18

Last Posted Date: 2024-10-10

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 73

Registration Number: NCT03989947

Locations: 🇯🇵
Osaka University, Osaka, Japan
🇺🇸
Children's Hospital & Research Center Oakland, Oakland, California, United States
🇦🇺
Murdoch Children's Research Institute, Parkville, Victoria, Australia

and more 13 locations

Lifetime Impact Study for Achondroplasia

Completed

Conditions: Achondroplasia

First Posted Date: 2019-03-13

Last Posted Date: 2021-04-05

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 173

Registration Number: NCT03872531

Locations: 🇨🇴
Fundacion Cardioinfantil-instituto de cardiologia, Bogotá, Colombia
🇧🇷
Centro de Pesquisa Clínica do Instituto da Criança HC - FMUSP, São Paulo, Brazil
🇦🇷
Hospital de Pediatría "Prof. Dr. Juan P. Garrahan", Buenos Aires, Argentina

and more 1 locations

Retrospective Observational Safety Effectiveness With Kuvan in hpA

Completed

Conditions: Tetrahydrobiopterin Deficiency

First Posted Date: 2019-03-06

Last Posted Date: 2019-03-07

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 26

Registration Number: NCT03864029

Locations: 🇨🇳
West China Hospital of Sichuan University, Chengdu, Sichuan, China

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

Phase 3

Completed

Conditions: Phenylketonuria (PKU)

Interventions: Drug: Pegvaliase

First Posted Date: 2018-10-03

Last Posted Date: 2022-02-17

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 37

Registration Number: NCT03694353

Locations: 🇺🇸
University of Missouri Health Care, Columbia, Missouri, United States
🇺🇸
Washington University School of Medicine, Saint Louis, Missouri, United States
🇺🇸
University of Florida, Gainesville, Florida, United States

and more 14 locations

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: 🇺🇸
Emory University, Decatur, Georgia, United States
🇯🇵
Osaka University Hospital, Osaka, Japan
🇺🇸
Vanderbilt University Medical Center, Nashville, Tennessee, United States

and more 13 locations

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