DeepMed Research

Biomarin Pharmaceutical

🇨🇦Canada

Country: 🇨🇦Canada

Ownership: -

Established: 1997-01-01

Employees: -

Market Cap: $17.2B

Website: http://www.biomarin.com

Clinical Trials

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

Phase 4

Recruiting

Conditions: Phenylketonuria
PKU

Interventions: Drug: RDD to Palynziq

First Posted Date: 2025-01-17

Last Posted Date: 2025-01-23

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 10

Registration Number: NCT06780332

Locations: 🇺🇸
Uncommon Cures, Chevy Chase, Maryland, United States

Study of BMN 349 Single Dose in (PiZZ) and (PiMZ) Adult Participants

Phase 1

Recruiting

Conditions: Alpha 1-Antitrypsin Deficiency

Interventions: Drug: BMN 349
Drug: Placebo

First Posted Date: 2024-12-17

Last Posted Date: 2025-03-17

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 12

Registration Number: NCT06738017

Locations: 🇺🇸
University of California, San Diego, San Diego, California, United States
🇺🇸
Saint Louis University, Saint Louis, Missouri, United States
🇺🇸
Medpace Clinical Pharmacology Unit, Cincinnati, Ohio, United States

and more 1 locations

A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

Phase 2

Recruiting

Conditions: Short Stature Homeobox- Containing Gene SHOX Deficiency
Noonan Syndrome
Turner Syndrome

Interventions: Drug: Vosoritide Injection
Drug: Human Growth Hormone

First Posted Date: 2024-10-31

Last Posted Date: 2025-05-13

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 72

Registration Number: NCT06668805

Locations: 🇺🇸
Children's Hospital Colorado, Aurora, Colorado, United States
🇺🇸
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children), Wilmington, Delaware, United States
🇺🇸
Nicklaus Children's Hospital, Miami, Florida, United States

and more 10 locations

Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia

Phase 3

Conditions: Hypochondroplasia

Interventions: Drug: Placebo
Drug: Vosoritide

First Posted Date: 2024-06-12

Last Posted Date: 2025-03-19

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 80

Registration Number: NCT06455059

Locations: 🇺🇸
Nemours Alfred I. DuPont Hospital for Children, Wilmington, Delaware, United States
🇺🇸
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸
Medical College of Wisconsin, Milwaukee, Wisconsin, United States

and more 20 locations

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

Phase 2

Recruiting

Conditions: Idiopathic Short Stature

Interventions: Drug: Vosoritide Injection
Drug: Placebo
Drug: Human Growth Hormone

First Posted Date: 2024-04-24

Last Posted Date: 2025-01-20

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 100

Registration Number: NCT06382155

Locations: 🇺🇸
Centricity Research, Columbus, Georgia, United States
🇺🇸
Nemours Children's Health System - Corporate Headquarters, Pensacola, Florida, United States
🇺🇸
Rocky Mountain Clinical Research - Idaho Falls, Idaho Falls, Idaho, United States

and more 5 locations

A Study to Assess Growth in Children With Idiopathic Short Stature

Recruiting

Conditions: Idiopathic Short Stature

First Posted Date: 2024-03-13

Last Posted Date: 2025-04-17

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 300

Registration Number: NCT06309979

Locations: 🇺🇸
Center Of Excellence in Diabetes and Endocrinology, Sacramento, California, United States
🇺🇸
University of California San Francisco (UCSF), San Francisco, California, United States
🇺🇸
Nicklaus Children's Hospital, Miami, Florida, United States

and more 16 locations

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Recruiting

Conditions: Phenylketonuria (PKU)

First Posted Date: 2024-03-12

Last Posted Date: 2024-08-26

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 200

Registration Number: NCT06305234

Locations: 🇺🇸
UT Southwestern Medical Center, Dallas, Texas, United States
🇺🇸
Northwestern University, Chicago, Illinois, United States
🇺🇸
University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States

and more 3 locations

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

Phase 1

Recruiting

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: BMN 351

First Posted Date: 2024-02-28

Last Posted Date: 2024-12-05

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 18

Registration Number: NCT06280209

Locations: 🇳🇱
Leids Universitair Medisch Centrum, Leiden, Netherlands
🇹🇷
Yeditepe University Kosuyolu Hospital, Istanbul, Turkey
🇬🇧
Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom

and more 4 locations

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Phase 3

Active, not recruiting

Conditions: Hemophilia A

Interventions: Biological: Valoctocogene roxaparvovec

First Posted Date: 2024-01-25

Last Posted Date: 2024-05-07

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 6

Registration Number: NCT06224907

Locations: 🇯🇵
Asahikawa Medical University Hospital, Hokkaido, Asahikawa, Japan
🇯🇵
Nagoya University Hospital, Aichi, Nagoya, Japan
🇯🇵
Tokyo Medical University Hospital, Tokyo, Shinjuku-ku, Japan

and more 1 locations

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Recruiting

Conditions: Hypochondroplasia

First Posted Date: 2024-01-19

Last Posted Date: 2025-04-03

Lead Sponsor: BioMarin Pharmaceutical

Target Recruit Count: 400

Registration Number: NCT06212947

Locations: 🇯🇵
Institute of Science Tokyo Hospital, Tokyo, Japan
🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸
UCSF Benioff Children's Hospital, Oakland, California, United States

and more 40 locations

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