BioMarin Pharmaceutical Clinical Trials
Sponsors / BioMarin Pharmaceutical
155 ClinicalTrials.gov entries name BioMarin Pharmaceutical as the lead sponsor. The portfolio currently includes 14 recruiting studies, 12 active non-recruiting studies, and 92 completed studies. 25 trials in this portfolio were terminated or withdrawn before completion. The most common reasons cited for termination include slow or insufficient recruitment, business or strategic decisions. Cumulative planned enrollment across all listed trials is 13,287 participants. These counts reflect the current state of the public ClinicalTrials.gov registry.
155
Total Trials
14
Recruiting
12
Other Active
92
Completed
25
Terminated / Withdrawn
13,287
Total Enrollment
Termination reasons: Other60% · Recruitment16% · Business8% · Regulatory8% · Safety8%
Recent Terminations
- Blood Disorder"Early rollover into long-term extension study"
- Hemophilia A"Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved."
- Achondroplasia"The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns."
- Maroteaux-Lamy Syndrome"Slow accrual"
- Duchenne Muscular Dystrophy"Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD."
- NCT02221362 Stopped Sep 2016Late-onset Pompe Patients Untreated or Treated With rhGAA
- Duchenne Muscular Dystrophy
- Duchenne Muscular Dystrophy
- NCT02191917 Stopped Jun 2016Late-onset Pompe Disease"Study was terminated early due to a company decision not based on safety concerns"
- Phenylketonuria"Recruitment challenges"
Active Trials (12 total)
- NCT07441876
- NCT04800692
- Achondroplasia PHASE2NCT02724228
- Achondroplasia PHASE3NCT03424018
- Achondroplasia PHASE2NCT03989947
- Achondroplasia PHASE2NCT04554940
- Idiopathic Short Stature PHASE2NCT06382155
- Hypochondroplasia PHASE2NCT07126262
- Noonan Syndrome PHASE2NCT06668805
- Duchenne Muscular Dystrophy PHASE1/PHASE2NCT06280209
- Hypochondroplasia PHASE3NCT06455059
- NCT04404530
- NCT04476862
- NCT06168201
- NCT05579548
- NCT06309979
- Phenylketonuria (PKU) PHASE3NCT05270837
- NCT06738017
- Hemophilia A With Anti Factor VIII PHASE1/PHASE2NCT04684940
- NCT06212947