All clinical trials on the European Union Clinical Trials Register (EUCTR) must report their results in the registry within a year of completion. This site tracks who's doing this and who isn't. Learn more »
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 |
|---|---|---|---|---|
| Completed, but no date, and reported results | 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 | |
| Reported results | 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 | 2005-11-12 | due-trials |
| Reported results | 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 | due-trials |
| Reported results | 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 | due-trials |
| Reported results | 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 | due-trials |
| Not reported | 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 | due-trials |
| Reported results | 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 Protocols 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 |
| Reported results | 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 | due-trials |
| Reported results | 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. | 2023-01-04 | due-trials |
| 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 |
| 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) Studio di estensione multicent... | 2016-06-16 | due-trials |
| Reported results | 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) | 2014-07-11 | due-trials |
| 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... | 2013-04-04 | not-yet-due |
| 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 | due-trials |
| Reported results | 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) ... | 2016-02-03 | due-trials |
| Exempt, with results | 2011-005040-10 | A phase I/II, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of PRO045 in subjects with Duchenne muscular dystrophy STUDIO IN APERTO DI FAS... | 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 |
| Reported results | 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 |
| Reported results | 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... | 2021-03-05 | due-trials |
| Reported results | 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 Estudio de fase 2, de 2 etapas y 2 cohortes, en e... | 2018-10-31 | due-trials |
| 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 |
| Reported results | 2013-004137-32 | A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia | 2017-10-02 | due-trials |
| Ongoing | 2014-002158-38 | A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease | not-yet-due | |
| Listed as ongoing, but also has a completion date and reported results | 2014-003480-37 | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | 2020-12-10 | bad-data |
| 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, and reported results | 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 Estudio de Fase 2, multicéntrico, abierto ... | 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, reported early | 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 | |
| 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 | due-trials |
| Exempt, with results | 2015-001985-25 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MP... | 2020-06-19 | not-yet-due |
| Listed as ongoing, but also has a completion date and 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 | bad-data |
| Reported results | 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. | 2019-10-30 | due-trials |
| Other | 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 | not-yet-due | |
| Other | 2016-003826-18 | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia, Age 0 to < 60 Months | not-yet-due | |
| Exempt | 2017-000662-29 | A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU... | not-yet-due | |
| Exempt, with results | 2017-000701-21 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia | 2021-02-11 | 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 | |
| Ongoing | 2017-003083-13 | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syn... | 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 | |
| Listed as ongoing, but also has a completion date | 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... | 2023-06-05 | bad-data |
| Ongoing, reported early | 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 | not-yet-due | |
| Other | 2018-004364-66 | A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | not-yet-due | |
| Exempt | 2019-001878-28 | A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects wi... | not-yet-due | |
| Ongoing | 2019-003213-34 | A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Active or Prior Inhibitors | not-yet-due | |
| Other | 2020-001055-40 | A randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia at risk of requiring cervicomedu... | not-yet-due | |
| Exempt | 2021-003603-18 | A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Subje... | not-yet-due | |
| Other | 2021-005058-27 | A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized Tw... | not-yet-due |