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
Reported results 2004-000646-20 A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal He... 2005-12-27 due-trials
Listed as ongoing, but also has a completion date and reported results 2004-002795-42 Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study 2006-10-12 bad-data
Reported results 2005-000043-28 A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPH... 2008-09-12 due-trials
Not reported Terminated 2006-002696-40 A Randomized, Double-Blind, Placebo-Controlled, Dose-Escalation Study to Investigate the Safety, Tolerability, Pharmacokinetics and Antiviral Activity of Multiple Doses of ACH-0137171 in Subjects with... 2007-02-08 due-trials
Listed as ongoing, but also has a completion date and reported results 2006-005968-13 A 14-Day, Randomized, Double-Blind, Comparative Viral Kinetic Study of Elvucitabine Versus Lamivudine Administered Once Daily to HIV-1 Infected Subjects with a Documented M184V Variant 2007-11-20 bad-data
Listed as ongoing, but also has a completion date and reported results 2006-006194-25 An open-label, 48-week extension study of elvucitabine administered in combination with background antiretroviral agents in subjects who have completed 14 days of treatment in Protocol ACH443-014A 2008-10-23 bad-data
Listed as ongoing, but also has a completion date and reported results 2008-006952-23 AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADULT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS) 2013-03-05 bad-data
Reported results 2008-006953-41 AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS) 2011-10-17 due-trials
Listed as ongoing, but also has a completion date and reported results 2008-006954-17 AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADULT PATIENTS WITH PLASMA THERAPY-SENSITIVE ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS) 2013-10-09 bad-data
Reported results 2008-006955-28 AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-SENSITIVE ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS) 2013-02-13 due-trials
Reported results 2009-009369-32 Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children with Hypophosphatasia (HPP) 2017-02-07 due-trials
Trial is outside EEC, and reported results 2009-010402-11 AN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA bad-data
Reported results Terminated 2009-014669-13 A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Multi-Center Study of Eculizumab in Patients with Generalized Myasthenia Gravis (gMG) Who Have Moderate to Severe Muscle Weakness Despite Tr... 2011-03-16 due-trials
Reported results 2010-019630-28 A RANDOMIZED, OPEN-LABEL, MULTICENTER TRIAL TO DETERMINE SAFETY AND EFFICACY OF ECULIZUMAB IN THE PREVENTION OF ANTIBODY MEDIATED REJECTION (AMR) IN LIVING DONOR KIDNEY TRANSPLANT RECIPIENTS REQUIRING... 2015-11-13 due-trials
Listed as ongoing, but also has a completion date and reported results 2010-019631-35 AN OPEN-LABEL, SINGLE-ARM, MULTICENTER TRIAL TO DETERMINE SAFETY AND EFFICACY OF ECULIZUMAB IN THE PREVENTION OF ANTIBODY MEDIATED REJECTION (AMR) IN SENSITIZED RECIPIENTS OF A KIDNEY TRANSPLANT FROM ... 2017-05-22 bad-data
Listed as ongoing, but also has a completion date and reported results 2010-019850-42 An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and ... 2017-04-26 bad-data
Listed as ongoing, but also has a completion date and reported results 2010-020310-28 An open-label, multi-center clinical trial of eculizumab in pediatric patients with atypical hemolytic-uremic syndrome (aHUS) 2014-01-08 bad-data
Listed as ongoing, but also has a completion date and reported results 2010-020326-18 An open-label, multi-center clinical trial of eculizumab in adult patients with atypical hemolytic-uremic syndrome 2013-10-31 bad-data
Ongoing, reported early 2010-022092-65 A phase IIa, randomized, double-blind (subject and investigator blind, sponsor open) placebo-controlled trial to evaluate the safety, tolerability and antiviral activity of oral ACH-0141625 in combina... not-yet-due
Reported results 2011-000032-28 An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 in Children with Growth Failure Due to Lysosomal Aci... 2018-01-03 due-trials
Completed, but no date, and reported results 2011-002691-17 An open label, multi-center trial of eculizumab in patients with Shiga-toxin producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS) bad-data
Listed as ongoing, but also has a completion date and reported results 2011-002750-31 A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency 2018-12-11 bad-data
Reported results 2011-004287-30 A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency 2017-12-28 due-trials
Listed as ongoing, but also has a completion date and reported results 2013-001150-10 A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTI-CENTER TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN PATIENTS WITH RELAPSING NEUROMYELITIS OPTICA (NMO) 2018-05-17 bad-data
Ongoing 2013-001151-12 A PHASE III, OPEN-LABEL, EXTENSION TRIAL OF ECU-NMO-301 TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN PATIENTS WITH RELAPSING NEUROMYELITIS OPTICA (NMO) not-yet-due
Listed as ongoing, but also has a completion date and reported results 2013-002191-41 A PHASE III, OPEN-LABEL, EXTENSION TRIAL OF ECU-MG-301 TO EVALUATE THE SAFETY AND EFFICACY OF ECULIZUMAB IN SUBJECTS WITH REFRACTORY GENERALIZED MYASTHENIA GRAVIS (gMG) 2019-01-15 bad-data
Other 2013-002702-30 A PHASE 2/3, MULTICENTER, MULTICENTER, MULTINATIONAL, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALX1101 IN NEONATES WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A not-yet-due
Listed as ongoing, but also has a completion date and reported results 2013-003400-39 A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously 2017-10-16 bad-data
Listed as ongoing, but also has a completion date and reported results 2013-003588-73 AN OPEN-LABEL PROOF OF CONCEPT PHASE IIA TRIAL OF ALXN1007 FOR THE TREATMENT OF NON-CRITERIA MANIFESTATIONS OF ANTIPHOSPHOLIPID SYNDROME 2016-06-20 bad-data
Reported results 2013-003589-15 A RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, 2016-02-19 due-trials
Reported results 2013-004650-25 A Randomized, Parallel-Group, Double-Blind, Placebo-Controlled, Multi-Center Study of Eculizumab for the Prevention of Delayed Graft Function after Kidney Transplantation in Adult Subjects at Increase... 2016-11-22 due-trials
Listed as ongoing, but also has a completion date and reported results 2014-000533-22 A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency 2018-10-30 bad-data
Not reported 2014-001703-41 A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase ... 2018-11-07 due-trials
Completed, but no date 2015-000753-20 A PHASE I/II OPEN LABEL STUDY IN MPS IIIB SUBJECTS TO INVESTIGATE THE SAFETY, BIODISTRIBUTION, PHARMACOKINETICS, AND PHARMACODYNAMICS/EFFICACY OF SBC-103 ADMINISTERED INTRAVENOUSLY bad-data
Reported results 2015-000809-39 An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (human recombinant tissue-nonspecific alkaline phosphatase fusion protein) Treatment for Patients with Infantile- or Juvenile-onse... 2018-03-08 due-trials
Trial is outside EEC, and reported results 2015-001128-52 A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Historical Control Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of ENB-0040 (Human Recombinant Tissue-Nonsp... bad-data
Reported results Terminated 2015-001983-20 A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intrav... 2017-08-18 due-trials
Ongoing, reported early 2015-002674-20 A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Pa... not-yet-due
Reported results 2015-003131-35 A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia 2017-06-21 due-trials
Reported results 2015-003135-35 EVALUATION OF POTENTIAL PREDICTORS OF DISEASE PROGRESSION IN PATIENTS WITH aHUS, INCLUDING GENETICS, BIOMARKERS, AND TREATMENT 2017-09-21 due-trials
Ongoing 2016-001384-37 An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis (gMG) not-yet-due
Ongoing, reported early 2016-002025-11 A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not-yet-due
Ongoing, reported early 2016-002026-36 A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab not-yet-due
Ongoing 2016-002027-29 Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Patients with Atypical Hemolytic Uremic Syndrome (aHUS) not-yet-due
Ongoing 2016-002499-29 A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) not-yet-due
Not reported 2016-002652-25 A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) 2019-02-27 due-trials
Not reported 2016-003525-42 A Phase 2a Proof-of-Mechanism, Open-Label Study to Determine the Effect of ACH-0144471 on C3 Levels in Patients with Low C3 Levels Due to Either C3 Glomerulopathy (C3G) or Immune-Complex Membranoproli... 2019-01-09 due-trials
Ongoing 2016-003526-16 A Phase 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy not-yet-due
Ongoing 2017-000663-33 A Phase 2, Proof-of-Concept, Randomized, Double-Blinded, Placebo-Controlled Study of ACH-0144471 Treatment for 6 Months in Patients with C3 Glomerulopathy (C3G) not-yet-due
Ongoing 2017-000665-79 An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100 not-yet-due
Trial is outside EEC, and reported results 2017-001831-38 A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Concurrent Control Study of the Safety, Efficacy, and Pharmacokinetics of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Ph... bad-data
Ongoing 2017-002370-39 A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal ... not-yet-due
Ongoing 2017-002674-39 An Open-Label Phase 2 Proof-of-Concept Study in Patients with C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) Treated with ACH-0144471 not-yet-due
Other 2017-002820-26 A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) not-yet-due
Trial is outside EEC, and reported results 2017-003153-42 Extension Study of Protocol ENB-006-09 Evaluating the Long-Term Safety and Efficacy of bad-data
Ongoing 2017-004135-36 A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older wi... not-yet-due
Ongoing 2018-003243-39 A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia G... not-yet-due
Ongoing 2019-001829-26 A Phase 2/3 Open-Label, Single-Arm Trial to Evaluate the Safety and Activity of Eculizumab in Pediatric Patients with Relapsing Neuromyelitis Optica Spectrum Disorder not-yet-due
Ongoing 2019-003352-37 A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) not-yet-due
Ongoing 2019-003440-74 Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab not-yet-due
Ongoing 2019-003711-60 A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 f... not-yet-due
Ongoing 2019-003829-18 A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular H... not-yet-due
Ongoing 2019-003830-17 A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients a... not-yet-due
Completed, report not yet due Terminated 2019-004055-37 A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients with Warm Autoimmune Hemolytic Anemia 2020-05-28 not-yet-due
Ongoing 2019-004619-30 A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic ... not-yet-due
No trial status on register 2020-000144-61 A Phase 3, Open-label, Randomized, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT) bad-data
Ongoing 2020-001104-41 A Phase 2, open-label study to assess copper and molybdenum balance in participants with Wilson disease treated with ALXN1840 not-yet-due
Ongoing 2020-001497-30 A Phase 3 Open-label, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Intravenously Administered Ravulizumab Compared with Best Supportive Care in Patients with COVID-19 Severe Pne... not-yet-due