Biomarin Pharmaceutical

🇨🇦Canada

Country: 🇨🇦Canada

Ownership: -

Established: 1997-01-01

Employees: -

Market Cap: $17.2B

Website: http://www.biomarin.com

Clinical Trials

A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Phase 2

Completed

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

Interventions: Biological: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
Device: Intraventricular access device

First Posted Date: 2016-02-10

Last Posted Date: 2023-03-13

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 14

Registration Number: NCT02678689

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

and more 1 locations

Extension Study of Drisapersen in DMD Subjects

Conditions: Duchenne Muscular Dystrophy

First Posted Date: 2015-12-22

Last Posted Date: 2018-01-24

Lead Sponsor: BioMarin Pharmaceutical

Registration Number: NCT02636686

Locations: 🇧🇪
Universitair Ziekenhuis Gent, Afdeling Neurologie, Gent, Belgium
🇧🇪
Queen Fabiola Children's University Hospital, Brussels, Belgium
🇯🇵
National Hospital Organization, Saitama, Japan

and more 36 locations

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Phase 1

Completed

Conditions: Severe Haemophilia A

Interventions: Biological: valoctocogene roxaparvovec

First Posted Date: 2015-10-15

Last Posted Date: 2024-08-06

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 15

Registration Number: NCT02576795

Locations: 🇬🇧
Queen Elizabeth Hospital Birmingham, Birmingham, United Kingdom
🇬🇧
Addenbrooke's Hospital, Cambridge, United Kingdom
🇬🇧
St. Thomas' Hospital, London, United Kingdom

and more 2 locations

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Phase 1

Completed

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

Interventions: Biological: BMN 190
Device: Intracerebroventricular (ICV) access device

First Posted Date: 2015-06-30

Last Posted Date: 2022-08-24

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 23

Registration Number: NCT02485899

Locations: 🇩🇪
Universitaetsklinikum Hamburg-Eppendorf, Hamburg, Germany
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇬🇧
Great Ormond Street Childrens Hospital, London, United Kingdom

and more 1 locations

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Completed

Conditions: Phenylketonuria

First Posted Date: 2015-06-11

Last Posted Date: 2020-06-04

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 9

Registration Number: NCT02468570

Locations: 🇺🇸
University of Nebraska Medical Center, Omaha, Nebraska, United States
🇺🇸
University of Kentucky Medical Center, Lexington, Kentucky, United States
🇺🇸
Oregon Health and Science University, Portland, Oregon, United States

and more 1 locations

Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

Conditions: Rare Genetic Disorders

First Posted Date: 2015-02-18

Last Posted Date: 2015-02-18

Lead Sponsor: BioMarin Pharmaceutical

Registration Number: NCT02365376

Locations: 🇺🇸
Lurie Children's Hospital of Chicago, Chicago, Illinois, United States

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Phase 2

Terminated

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: PRO044 SC 6 mg/kg
Drug: PRO044 IV 6 mg/kg
Drug: PRO044 IV 9 mg/kg

First Posted Date: 2015-01-01

Last Posted Date: 2017-12-08

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 15

Registration Number: NCT02329769

Locations: 🇧🇪
UZ Leuven, Leuven, Belgium
🇳🇱
Leids Universitair Medisch Centrum, Leiden, Netherlands
🇮🇹
S.Anna Hospital, Ferrara, Italy

and more 2 locations

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Completed

Conditions: Mucopolysaccharidosis IV Type A
MPS IVA
Morquio A Syndrome

First Posted Date: 2014-11-19

Last Posted Date: 2024-03-05

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 418

Registration Number: NCT02294877

Locations: 🇺🇸
University of Southern California, Los Angeles, California, United States
🇺🇸
Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, United States
🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States

and more 76 locations

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Terminated

Conditions: Late-onset Pompe Patients Untreated or Treated With rhGAA

First Posted Date: 2014-08-20

Last Posted Date: 2016-10-31

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 30

Registration Number: NCT02221362

Locations: 🇸🇬
National University Hospital, Singapore, Singapore
🇦🇺
Westmead Hospital, Dept. of Genetic Medicine, Westmead, New South Wales, Australia
🇪🇸
Hospital de la Santa Creu I Sant Pau, Barcelona, Spain

and more 12 locations

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

Terminated

Conditions: Late-onset Pompe Disease

Interventions: Procedure: Respiratory muscle strength measurements by different techniques.

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

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