Biomarin Pharmaceutical

🇨🇦Canada

Country: 🇨🇦Canada

Ownership: -

Established: 1997-01-01

Employees: -

Market Cap: $17.2B

Website: http://www.biomarin.com

Clinical Trials

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

Completed

Conditions: Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

First Posted Date: 2009-11-19

Last Posted Date: 2022-04-15

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 627

Registration Number: NCT01016392

Locations: 🇫🇷
Research Site - Armand Trousseau, Paris, France
🇮🇹
Research Site - Ospedale Annunziata, Napoli, Italy
🇫🇷
Research Site - Necker, Paris, France

and more 2 locations

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Phase 2

Completed

Conditions: Phenylketonuria

Interventions: Drug: rAvPAL-PEG 0.001 mg/kg
Drug: rAvPAL-PEG 0.003 mg/kg
Drug: rAvPAL-PEG 0.01 mg/kg
Drug: rAvPAL-PEG 0.03 mg/kg
Drug: rAvPAL-PEG 0.1 mg/kg

First Posted Date: 2009-06-19

Last Posted Date: 2019-02-26

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 40

Registration Number: NCT00925054

Locations: 🇺🇸
Akron Children's Hospital, Akron, Ohio, United States
🇺🇸
Emory Universty, Decatur, Georgia, United States
🇺🇸
Washington University Center for Applied Research Sciences, Saint Louis, Missouri, United States

and more 8 locations

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Phase 2

Completed

Conditions: Phenylketonuria

Interventions: Drug: rAvPAL-PEG

First Posted Date: 2009-06-19

Last Posted Date: 2021-10-12

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 68

Registration Number: NCT00924703

Locations: 🇺🇸
University of Florida, Gainesville, Florida, United States
🇺🇸
Ann and Robert H Lurie Children's Hospital, Chicago, Illinois, United States
🇺🇸
The Children's Hospital, Aurora, Colorado, United States

and more 11 locations

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

Phase 1

Completed

Conditions: MPS IV A

Interventions: Drug: BMN 110

First Posted Date: 2009-04-21

Last Posted Date: 2014-06-30

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 20

Registration Number: NCT00884949

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

Phase 3

Completed

Conditions: Phenylketonuria

Interventions: Drug: sapropterin dihydrochloride

First Posted Date: 2009-02-06

Last Posted Date: 2020-12-22

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 95

Registration Number: NCT00838435

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

Phase 1

Completed

Conditions: Phenylketonuria

Interventions: Drug: sapropterin dihydrochloride
Drug: Moxifloxacin placebo
Drug: Moxifloxacin

First Posted Date: 2008-11-13

Last Posted Date: 2021-07-23

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 56

Registration Number: NCT00789568

Locations: 🇺🇸
PRACS Institute, Ltd., Fargo, North Dakota, United States

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Terminated

Conditions: MPS IV A
Mucopolysaccharidosis IVA
Morquio A Syndrome

First Posted Date: 2008-11-10

Last Posted Date: 2014-12-08

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 353

Registration Number: NCT00787995

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Completed

Conditions: Phenylketonuria
Hyperphenylalaninaemia

Interventions: Drug: Kuvan

First Posted Date: 2008-10-23

Last Posted Date: 2022-10-12

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 1887

Registration Number: NCT00778206

Locations: 🇺🇸
LAC and USC Medical Center, Los Angeles, California, United States
🇺🇸
UCLA, Los Angeles, California, United States
🇺🇸
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States

and more 42 locations

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

Phase 1

Completed

Conditions: Phenylketonuria

Interventions: Drug: rAvPAL-PEG

First Posted Date: 2008-03-13

Last Posted Date: 2017-02-01

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 25

Registration Number: NCT00634660

Locations: 🇺🇸
Mount Sinai Medical Center, New York, New York, United States
🇺🇸
University of Wisconsin, Madison, Wisconsin, United States
🇺🇸
Children's Memorial Hospital, Chicago, Illinois, United States

and more 5 locations

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Phase 2

Completed

Conditions: Endothelial Dysfunction

Interventions: Drug: Sapropterin Dihydrochloride
Drug: Sapropterin Dihydrochloride and Vitamin C

First Posted Date: 2007-09-20

Last Posted Date: 2021-05-17

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 52

Registration Number: NCT00532844

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