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20 Products |  |
157 Diseases | | 20 Products |
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112 Trials |  |
5109 News |  |
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- | Vimizim (elosulfase alfa) / BioMarin
Enrollment closed: Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (clinicaltrials.gov) - Aug 20, 2012
P2, N=25, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Recruiting --> Active, not recruiting
- | Vimizim (elosulfase alfa) / BioMarin
Enrollment closed: Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) (clinicaltrials.gov) - Aug 7, 2012
P2, N=15, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Recruiting --> Active, not recruiting Enrolling by invitation --> Active, not recruiting
- Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
New P3 trial: The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU (clinicaltrials.gov) - Jul 24, 2012
P3, N=0, Withdrawn, Sponsor: Stanford University
- |||| Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
Trial completion: ENDURE: Response to Kuvan (clinicaltrials.gov) - Jul 19, 2012
P4, N=59, Completed, Sponsor: Merck KGaA
Enrolling by invitation --> Active, not recruiting Recruiting --> Completed
- | Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
New P4 trial: Effects of Kuvan on Melatonin Secretion (clinicaltrials.gov) - Jun 10, 2012
P4, N=12, Enrolling by invitation, Sponsor: University of Southern California
- ||| Voxzogo (vosoritide) / BioMarin
Trial completion: A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers (clinicaltrials.gov) - Jun 6, 2012
P1, N=74, Completed, Sponsor: BioMarin Pharmaceutical
Recruiting --> Completed Recruiting --> Completed
- | Vimizim (elosulfase alfa) / BioMarin
New P2 trial: Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (clinicaltrials.gov) - May 29, 2012
P2, N=25, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
- Aldurazyme (laronidase) / BioMarin, Sanofi
Enrollment open: Administration of IV Laronidase Post Bone Marrow Transplant in Hurler (clinicaltrials.gov) - May 29, 2012
P1, N=10, Recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Recruiting --> Completed Not yet recruiting --> Recruiting
- | Palynziq (pegvaliase-pqpz) / BioMarin
Enrollment closed: Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) (clinicaltrials.gov) - May 29, 2012
P2, N=16, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Not yet recruiting --> Recruiting Completed --> Active, not recruiting
- Aldurazyme (laronidase) / BioMarin, Sanofi
Enrollment open: Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome (clinicaltrials.gov) - May 14, 2012
P=N/A, N=50, Recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Completed --> Active, not recruiting Not yet recruiting --> Recruiting
- ||||| Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
Enrollment closed: PKU-015: Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU (clinicaltrials.gov) - May 10, 2012
P3, N=230, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting
- ||| Voxzogo (vosoritide) / BioMarin
New P1 trial: A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers (clinicaltrials.gov) - May 1, 2012
P1, N=74, Completed, Sponsor: BioMarin Pharmaceutical
- Aldurazyme (laronidase) / BioMarin, Sanofi
Trial suspension: A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients (clinicaltrials.gov) - Apr 30, 2012
P4, N=25, Suspended, Sponsor: Genzyme, a Sanofi Company
Recruiting --> Active, not recruiting Recruiting --> Suspended
- | Palynziq (pegvaliase-pqpz) / BioMarin
Enrollment change: Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) (clinicaltrials.gov) - Apr 9, 2012
P2, N=16, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Recruiting --> Suspended N=36 --> 16
- | Palynziq (pegvaliase-pqpz) / BioMarin
Enrollment closed: Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU (clinicaltrials.gov) - Apr 9, 2012
P2, N=45, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
N=36 --> 16 Recruiting --> Active, not recruiting
- | Palynziq (pegvaliase-pqpz) / BioMarin
New P2 trial: A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks (clinicaltrials.gov) - Mar 20, 2012
P2, N=24, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
- | Palynziq (pegvaliase-pqpz) / BioMarin
Trial initiation date: Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) (clinicaltrials.gov) - Mar 4, 2012
P2, N=16, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Recruiting --> Active, not recruiting Initiation date: Nov 2010 --> Mar 2011
- Kyndrisa (drisapersen) / BioMarin
Enrollment change: DEMAND I: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects (clinicaltrials.gov) - Feb 1, 2012
P1, N=20, Completed, Sponsor: GlaxoSmithKline
Initiation date: Nov 2010 --> Mar 2011 N=32 --> 20
- | Kyndrisa (drisapersen) / BioMarin
Trial completion: DEMAND I: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects (clinicaltrials.gov) - Feb 1, 2012
P1, N=20, Completed, Sponsor: GlaxoSmithKline
N=32 --> 20 Active, not recruiting --> Completed
- | Vimizim (elosulfase alfa) / BioMarin
New P2 trial: Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) (clinicaltrials.gov) - Jan 22, 2012
P2, N=15, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
- Kuvan (sapropterin) / Merck (MSD), BioMarin, Daiichi Sankyo
Biomarker, Enrollment change: Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders (clinicaltrials.gov) - Dec 8, 2011
P=N/A, N=10, Completed, Sponsor: The University of Texas Health Science Center, Houston
Active, not recruiting --> Completed N=20 --> 10
- Kuvan (sapropterin) / Merck (MSD), BioMarin, Daiichi Sankyo
Biomarker, Trial completion: Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders (clinicaltrials.gov) - Dec 8, 2011
P=N/A, N=10, Completed, Sponsor: The University of Texas Health Science Center, Houston
N=20 --> 10 Recruiting --> Completed
- |||||||||| Kyndrisa (drisapersen) / BioMarin
New P3 trial: Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (clinicaltrials.gov) - Nov 24, 2011
P3, N=220, Active, not recruiting, Sponsor: GlaxoSmithKline
- Aldurazyme (laronidase) / BioMarin, Sanofi
Enrollment open: Intrathecal Enzyme Replacement for Hurler Syndrome (clinicaltrials.gov) - Nov 16, 2011
P1, N=25, Recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Recruiting --> Completed Suspended --> Recruiting
- Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
Trial completion, FDG PET: Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy (clinicaltrials.gov) - Nov 15, 2011
P=N/A, N=6, Completed, Sponsor: Children's Hospital of Philadelphia
Suspended --> Recruiting Recruiting --> Completed
- Kuvan (sapropterin) / Merck (MSD), BioMarin, Daiichi Sankyo
Enrollment open: Endothelial Facilitation in Alzheimer's Disease (clinicaltrials.gov) - Nov 1, 2011
P2, N=10, Recruiting, Sponsor: University of Massachusetts, Worcester
Recruiting --> Completed Not yet recruiting --> Recruiting
- ||||| Kyndrisa (drisapersen) / BioMarin
New P2 trial, IO biomarker: A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - Oct 27, 2011
P2, N=51, Completed, Sponsor: GlaxoSmithKline
- Kuvan (sapropterin) / Merck (MSD), BioMarin, Daiichi Sankyo
New P2/3 trial: Randomized Controlled Trial to Assess the Effects of Sapropterin on Hepatic and Systemic Hemodynamics in Patients With Liver Cirrhosis and Portal Hypertension (clinicaltrials.gov) - Oct 18, 2011
P2/3, N=42, Completed, Sponsor: Fundacion Clinic per a la Recerca Biomédica
- Aldurazyme (laronidase) / BioMarin, Sanofi
Enrollment closed: Stem Cell Transplant w/Laronidase for Hurler (clinicaltrials.gov) - Oct 17, 2011
P2, N=25, Active, not recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting
- Kuvan (sapropterin) / Merck (MSD), BioMarin, Daiichi Sankyo
New P2 trial: Endothelial Facilitation in Alzheimer's Disease (clinicaltrials.gov) - Sep 21, 2011
P2, N=10, Recruiting, Sponsor: University of Massachusetts, Worcester
- | reveglucosidase alfa (BMN 701) / BioMarin
New P2 trial: Extension Study for Patients Who Have Participated in a BMN 701 Study (clinicaltrials.gov) - Sep 15, 2011
P2, N=45, Recruiting, Sponsor: BioMarin Pharmaceutical
- Kyndrisa (drisapersen) / BioMarin
New P1 trial: An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy (EUDRACT) - Aug 30, 2011
P1, N=200, Ongoing, Sponsor: GlaxoSmithKline Research and Development LTD`GlaxoSmithKline, S.A.
- | Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
New P1/2 trial: Study of Kuvan Treatment in Adults With GTPCH Deficiency (clinicaltrials.gov) - Aug 28, 2011
P1/2, N=12, Completed, Sponsor: University of Utah
- |||||||||| Vimizim (elosulfase alfa) / BioMarin
New P3 trial: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (clinicaltrials.gov) - Aug 10, 2011
P3, N=173, Active, not recruiting, Sponsor: BioMarin Pharmaceutical