Clinical Trials Sponsored by BioMarin+Pharmaceutical

Total 19816 results

BioMarin Pharmaceutical

Completed

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

MPS IV A

United Kingdom
BioMarin Pharmaceutical

Completed

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

MPS VI

United States, Australia, France, Brazil, Germany, Portugal, United Kingdom
BioMarin Pharmaceutical

Completed

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

Phenylketonurias

United States
BioMarin Pharmaceutical

Completed

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

Hypertension

United States
BioMarin Pharmaceutical

Approved for marketing

Sapropterin Expanded Access Program

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

Phenylketonurias

United States
BioMarin Pharmaceutical

Not yet recruiting

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Idiopathic Short Stature
BioMarin Pharmaceutical

Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Spain, Turkey, United Kingdom, Italy
BioMarin Pharmaceutical

Recruiting

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia

United States, Spain, Canada, France, Japan, Australia, Italy, Argentina, Brazil, Germany, United Kingdom
BioMarin Pharmaceutical

Recruiting

A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

Hemophilia A

Argentina
BioMarin Pharmaceutical

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (PEGASUS)

Phenylketonuria (PKU)

United States, Germany
BioMarin Pharmaceutical

Completed

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

Achondroplasia

United States, Australia, United Kingdom, Japan
BioMarin Pharmaceutical

Withdrawn

A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria

Nonalcoholic Fatty Liver Disease | Kidney Stone | Hyperoxaluria

United States
BioMarin Pharmaceutical

Active, not recruiting

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (HAErmony-1)

Hereditary Angioedema | HAE

Spain, United States, Australia
BioMarin Pharmaceutical

Active, not recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
BioMarin Pharmaceutical

Active, not recruiting

Cerliponase Alfa Observational Study in the US

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

United States
BioMarin Pharmaceutical

Active, not recruiting

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301)

Hemophilia A

United States, Taiwan, United Kingdom, Australia, Israel, Belgium, Brazil, France, Germany, Italy, Korea, Republic of, South Africa, Spain
BioMarin Pharmaceutical

Active, not recruiting

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

Severe Haemophilia A

United Kingdom
BioMarin Pharmaceutical

Active, not recruiting

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

Achondroplasia

United States, Australia, Germany, Spain, United Kingdom, Japan, Turkey
BioMarin Pharmaceutical

Active, not recruiting

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) (ACH)

Achondroplasia

United States, Australia, France, United Kingdom
BioMarin Pharmaceutical

Terminated

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

Achondroplasia

United States
BioMarin Pharmaceutical

Completed

A Prospective Clinical Study of Phenylketonuria (PKU)

Phenylketonurias

United States
BioMarin Pharmaceutical

Completed

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

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

United States, Germany, Italy, United Kingdom
BioMarin Pharmaceutical

Terminated

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

Late-onset Pompe Disease

United States, United Kingdom
BioMarin Pharmaceutical

Completed

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

Phenylketonuria | Hyperphenylalaninaemia

United States
BioMarin Pharmaceutical

Completed

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

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

United States, Germany, Italy, United Kingdom
BioMarin Pharmaceutical

Completed

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

Phenylketonuria (PKU)

United States
BioMarin Pharmaceutical

Completed

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

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU (Prism-2)

Phenylketonuria (PKU)

United States
BioMarin Pharmaceutical

Completed

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

Endothelial Dysfunction

United States
BioMarin Pharmaceutical

Completed

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

Achondroplasia

United States, Australia, France, United Kingdom
BioMarin Pharmaceutical

Completed

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU (PKU-015)

Phenylketonuria

United States, Canada
BioMarin Pharmaceutical

Completed

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 (PRISM303)

Phenylketonuria

United States
BioMarin Pharmaceutical

Completed

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

Phenylketonuria

United States
BioMarin Pharmaceutical

Terminated

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

Muscular Dystrophies

United States, Canada
BioMarin Pharmaceutical

Terminated

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Belgium, Netherlands, France, United Kingdom, Italy
BioMarin Pharmaceutical

Terminated

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

United States, Netherlands, Belgium, Germany, Argentina, Brazil, France, Italy, Sweden, Turkey
BioMarin Pharmaceutical

Completed

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old (SPARK)

Phenylketonuria

Italy, Turkey, United Kingdom, Germany, Netherlands, Slovakia, Austria, Belgium, Czechia
BioMarin Pharmaceutical

Completed

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

Taiwan, United States, Italy, United Kingdom
BioMarin Pharmaceutical

Terminated

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

Muscular Dystrophies
BioMarin Pharmaceutical

Terminated

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

Late-onset Pompe Patients Untreated or Treated With rhGAA

Spain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
BioMarin Pharmaceutical

Completed

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

Phenylketonurias

United States
BioMarin Pharmaceutical

Unknown

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

Rare Genetic Disorders

United States
BioMarin Pharmaceutical

Terminated

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

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

France, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy
BioMarin Pharmaceutical

Completed

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

Achondroplasia

United States
BioMarin Pharmaceutical

Completed

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Mucopolysaccharidosis VI | Maroteaux-Lamy Syndrome

United States, Portugal, France
BioMarin Pharmaceutical

Completed

Study of rhASB in Patients With Mucopolysaccharidosis VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Mucopolysaccharidosis VI

United States

Clinical Trials Sponsored by BioMarin+Pharmaceutical

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Sweden

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations