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

Terminated

Conditions: Late-onset Pompe Disease

First Posted Date: 2014-07-16

Last Posted Date: 2023-01-09

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 8

Registration Number: NCT02191917

Locations: 🇺🇸
University of Florida, Gainesville, Florida, United States
🇬🇧
Royal Brompton Hospital, London, United Kingdom

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

Phase 2

Completed

Conditions: Achondroplasia

Interventions: Drug: BMN 111

First Posted Date: 2014-02-05

Last Posted Date: 2021-01-15

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 35

Registration Number: NCT02055157

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

and more 6 locations

Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Phase 2

Terminated

Conditions: Phenylketonuria

Interventions: Drug: Placebo
Drug: Sapropterin

First Posted Date: 2013-11-07

Last Posted Date: 2018-02-22

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 2

Registration Number: NCT01977820

Locations: 🇮🇹
Research Site, Bologna, Italy
🇨🇭
Research site, Lausanne, Switzerland

BMN 110 Phase 3B in Australian Patients

Phase 3

Completed

Conditions: Mucopolysaccharidosis IVA (Morquio A Syndrome)

Interventions: Drug: BMN 110

First Posted Date: 2013-10-21

Last Posted Date: 2019-09-26

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 13

Registration Number: NCT01966029

Locations: 🇦🇺
Children's Hospital at Westmead, Westmead, New South Wales, Australia
🇦🇺
Lady Cilento Children's Hospital (previous: Royal Children's Hospital), Brisbane, Queensland, Australia
🇦🇺
Murdoch Childrens Research Institute and Royal Children's Hospital, Melbourne, Victoria, Australia

and more 2 locations

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

Phase 4

Completed

Conditions: Phenylketonuria

Interventions: Drug: Kuvan®

First Posted Date: 2013-10-18

Last Posted Date: 2023-04-24

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 34

Registration Number: NCT01965912

Locations: 🇬🇧
Research site, Bristol, United Kingdom
🇮🇹
Research site - Bambino Gesu, Roma, Italy
🇮🇹
Research site - La Sapienza, Roma, Italy

and more 3 locations

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

Phase 1

Terminated

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Regimen Selection Phase Group 1 (COMPLETED)
Drug: Regimen Selection Phase Group 2
Drug: Treatment Phase Group 4
Drug: Dosing Extension
Drug: Regimen Selection Phase Group 3

First Posted Date: 2013-10-08

Last Posted Date: 2017-12-08

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 9

Registration Number: NCT01957059

Locations: 🇧🇪
UZ Leuven, Campus Gasthuisberg, Leuven, Belgium
🇫🇷
Institut de Myologie, Paris, France
🇮🇹
Policlinico Universitario Agostino Gemelli, Rome, Italy

and more 3 locations

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

Phase 3

Terminated

Conditions: Late-onset Pompe Disease

Interventions: Drug: BMN 701

First Posted Date: 2013-08-19

Last Posted Date: 2018-06-14

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 24

Registration Number: NCT01924845

Locations: 🇺🇸
Neuromuscular Research Centre, Phoenix, Arizona, United States
🇺🇸
University of California, Irvine, Orange, California, United States
🇺🇸
University of Florida, Gainesville, Florida, United States

and more 19 locations

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration

Phase 2

Terminated

Conditions: Muscular Dystrophies

Interventions: Drug: Drisapersen

First Posted Date: 2013-07-29

Last Posted Date: 2016-11-06

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 12

Registration Number: NCT01910649

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 1

Completed

Conditions: Jansky-Bielschowsky Disease
Batten Disease
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
CLN2 Disease

First Posted Date: 2013-07-24

Last Posted Date: 2019-03-08

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 24

Registration Number: NCT01907087

Locations: 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇩🇪
University Hamburg-Eppendorf, Hamburg, Germany
🇮🇹
Bambino Gesù Children's Hospital, Rome, Italy

and more 2 locations

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

Phase 3

Completed

Conditions: Phenylketonuria (PKU)

Interventions: Drug: BMN165 40mg/day
Drug: BMN165 20mg/day
Drug: Placebo

First Posted Date: 2013-07-01

Last Posted Date: 2021-05-21

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 215

Registration Number: NCT01889862

Locations: 🇺🇸
University of California, San Diego Clinical and Translational Research Institute, La Jolla, California, United States
🇺🇸
UCSF Benioff Children's Hospital Oakland, Oakland, California, United States
🇺🇸
Children's Hospital Colorado, Aurora, Colorado, United States

and more 26 locations

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