These trials completed more than 12 months ago and should have reported results. Some have, some have not.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
No trial status on register 2004-000642-21 A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI bad-data
Ongoing 2004-002071-16 A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels not-yet-due
Listed as ongoing, but also has a completion date 2004-004512-23 A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels 2006-02-15 bad-data
Listed as ongoing, but also has a completion date 2004-004513-41 A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels 2006-10-10 bad-data
Listed as ongoing, but also has a completion date 2005-003512-30 A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) 2009-05-27 bad-data
Listed as ongoing, but also has a completion date 2005-003777-24 A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children... 2006-11-06 bad-data
Not reported 2005-003778-13 A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency 2009-06-10 due-trials
Listed as ongoing, but also has a completion date 2006-000839-10 A Phase 3b, Multicenter, Open–Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Studies PKU–004 or PKU–006 2009-08-24 bad-data
Exempt, with results 2008-007365-23 A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome) 2011-02-09 not-yet-due
Listed as ongoing, but also has a completion date 2009-015768-33 A Phase IIIb, Multicenter, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan) in Phenylketonuria (PKU) Patients <4... 2017-02-17 bad-data
Ongoing 2009-015844-41 A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. not-yet-due
Listed as ongoing, but also has a completion date and reported results 2010-020198-18 A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Muco... 2012-08-23 bad-data
Listed as ongoing, but also has a completion date and reported results 2010-020199-45 A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) 2016-06-16 bad-data
Ongoing, reported early 2010-021048-16 A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) not-yet-due
Reported results 2010-021850-20 A Phase 3, Multicenter, Double-blind, Placebo-controlled Randomized Discontinuation Study Followed by an Open-label Extension Period to Evaluate the Efficacy and Safety of Amifampridine Phosphate (3,4... 2016-07-06 due-trials
Exempt, with results 2010-023561-22 A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Di... 2013-03-05 not-yet-due
Listed as ongoing, but also has a completion date and reported results 2011-001805-28 A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study 2016-09-12 bad-data
Ongoing, reported early 2011-003197-84 A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome) not-yet-due
Exempt, with results 2011-005040-10 A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrop... 2016-09-09 not-yet-due
Listed as ongoing, but also has a completion date and reported results 2011-005042-35 A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in... 2016-09-09 bad-data
Reported results 2011-005682-20 A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) 2014-11-20 due-trials
Reported results 2011-005703-33 A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation 2014-10-22 due-trials
Not reported 2012-005430-11 A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipofus... 2015-11-30 due-trials
Reported results 2013-001768-48 A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease 2016-09-12 due-trials
Ongoing, reported early 2013-002716-28 A Phase 3, Open-Label, Randomized, Parallel, 2-Arm, Multi-Center Study of BMN 673 versus Physician’s Choice in Germline BRCA Mutation Subjects with Locally Advanced and/or Metastatic Breast Cancer, Wh... not-yet-due
Completed, reported early 2013-003076-12 A Phase 2, 2-Stage. 2-Cohort Study of BMN 673 Administered to Germline BRCA Mutation Subjects with Locally Advanced and/or Metastatic Breast Cancer 2018-10-31 not-yet-due
Reported results 2013-003605-26 A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy 2016-08-05 due-trials
Listed as ongoing, but also has a completion date 2013-004137-32 A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia 2017-10-02 bad-data
Ongoing 2014-002158-38 A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease not-yet-due
Ongoing 2014-003480-37 A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease not-yet-due
Exempt 2014-003880-38 A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A not-yet-due
Completed, but no date 2014-004011-37 A Phase 2, Multicenter, Open-Label, Exploratory Biomarker Study of Talazoparib (BMN 673) Monotherapy in Patients with Recurrent or Metastatic Solid Tumors bad-data
Reported results 2014-005296-81 A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Mus... 2016-09-09 due-trials
Ongoing 2015-000891-85 A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease not-yet-due
Listed as ongoing, but also has a completion date and reported results 2015-001955-54 An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. 2016-09-09 bad-data
Exempt 2015-001985-25 A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (M... not-yet-due
Reported results 2015-003681-87 A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy 2016-09-09 due-trials
Ongoing 2015-003836-11 A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. not-yet-due
No trial status on register 2015-004004-30 A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia bad-data
Exempt 2017-000701-21 A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia not-yet-due
Other 2017-002404-28 A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia not-yet-due
Other 2017-003083-13 A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Sy... not-yet-due
Ongoing 2017-003215-19 A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residu... not-yet-due
Ongoing 2017-003573-34 A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemophili... not-yet-due
No trial status on register 2018-000648-25 A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management bad-data