Biomarin Pharmaceutical
- Country
- 🇨🇦Canada
- Ownership
- -
- Established
- 1997-01-01
- Employees
- -
- Market Cap
- $17.2B
- Website
- http://www.biomarin.com
VIrtual STudy in Achondroplasia for the US (VISTA)
- Conditions
- Achondroplasia
- First Posted Date
- 2023-12-13
- Last Posted Date
- 2025-03-13
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 170
- Registration Number
- NCT06168201
- Locations
- 🇺🇸
UCSF, Oakland, California, United States
🇺🇸PicnicHealth, San Francisco, California, United States
A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
- Conditions
- Kidney StoneHyperoxaluriaNonalcoholic Fatty Liver Disease
- Interventions
- Drug: Placebo
- First Posted Date
- 2023-11-18
- Last Posted Date
- 2024-06-11
- Lead Sponsor
- BioMarin Pharmaceutical
- Registration Number
- NCT06138327
- Locations
- 🇺🇸
University of Alabama - Department of Urology, Birmingham, Alabama, United States
🇺🇸ProSciento, Inc., Chula Vista, Florida, United States
🇺🇸Medpace Clinical Pharmacology Unit, Cincinnati, Ohio, United States
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
- Conditions
- Phenylketonuria (PKU)
- First Posted Date
- 2023-04-14
- Last Posted Date
- 2024-05-13
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 450
- Registration Number
- NCT05813678
- Locations
- 🇺🇸
University of Texas Southwestern Medical Center, Dallas, Texas, United States
🇺🇸Medical College of Wisconsin, Milwaukee, Wisconsin, United States
🇮🇹Policlinico Sant'orsola Malpighi, Bologna, Italy
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
- Conditions
- Achondroplasia
- Interventions
- Drug: BMN 111 administration via vial and syringeCombination Product: BMN 111 administration via Injector Pen
- First Posted Date
- 2023-04-14
- Last Posted Date
- 2023-10-12
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 36
- Registration Number
- NCT05813314
- Locations
- 🇺🇸
Labcorp, Dallas, Texas, United States
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
- Conditions
- Hemophilia A
- First Posted Date
- 2023-03-14
- Last Posted Date
- 2024-04-24
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 172
- Registration Number
- NCT05768386
- Locations
- 🇺🇸
UCN Hemophilia and Thrombosis Center, Shandon, California, United States
🇬🇧Addenbrooke's Hospital, Hemophilia and Thrombophilia Center, Cambridge, United Kingdom
🇬🇧Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine, London, United Kingdom
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
- First Posted Date
- 2022-10-14
- Last Posted Date
- 2024-05-03
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 50
- Registration Number
- NCT05579548
- Locations
- 🇩🇪
Universitaetsklinikum Hamburg Eppendorf, Hamburg, Germany
🇮🇹Ospedale San Paolo, Milano, Italy
🇺🇸Syneos Health, Wilmington, North Carolina, United States
A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
- Conditions
- Hemophilia A
- Interventions
- Procedure: Biospecimen Collection
- First Posted Date
- 2022-10-14
- Last Posted Date
- 2024-08-19
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 321
- Registration Number
- NCT05580692
- Locations
- 🇸🇦
King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia
🇦🇷Instituto de Hematologia y Medicina Clinica Dr. RUben Davoli, Rosario, Argentina
🇦🇷Instituto Hematologia Arbesu, Godoy Cruz, Mendoza, Argentina
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Other: Diet Only
- First Posted Date
- 2022-03-08
- Last Posted Date
- 2024-06-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 55
- Registration Number
- NCT05270837
- Locations
- 🇺🇸
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States
🇺🇸Phoenix Children's Hospital, Phoenix, Arizona, United States
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
- Conditions
- Hereditary AngioedemaHAE
- Interventions
- Genetic: Dose 1 of BMN 331Genetic: Dose 2 of BMN 331Genetic: Dose 3 of BMN 331Genetic: Dose 4 of BMN 331Genetic: Dose 5 of BMN 331Genetic: Dose 6 of BMN 331Genetic: Dose 7 of BMN 331
- First Posted Date
- 2021-11-16
- Last Posted Date
- 2024-05-16
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 44
- Registration Number
- NCT05121376
- Locations
- 🇺🇸
Optimed Research, LTD, Columbus, Ohio, United States
🇺🇸University of California San Diego, San Diego, California, United States
🇺🇸Medical Research of Arizona, Scottsdale, Arizona, United States
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
- Conditions
- Hemophilia A With Anti Factor VIIIHemophilia A With Inhibitor
- Interventions
- Biological: Valoctocogene roxaparvovec
- First Posted Date
- 2020-12-28
- Last Posted Date
- 2024-07-05
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 10
- Registration Number
- NCT04684940
- Locations
- 🇨🇳
Kaohsiung Medical University - Chung-Ho Memorial Hospital, Kaohsiung, Taiwan
🇨🇳National Taiwan University Hospital, Taipei, Taiwan
🇮🇱Chaim Sheba Medical Center, Ramat Gan, Israel