trial_id,trial_phase,orphan_designation_id,disease_orpha_code,disease_name,disease_icd10,disease_prevalence_per_100k,drug_candidate_name,mechanism_of_action,trial_design,trial_start_date,trial_status,sponsor_type,regulatory_jurisdiction,fast_track_designation,breakthrough_therapy,accelerated_approval_eligible,target_enrollment,actual_enrollment,num_sites,primary_endpoint,endpoint_type
XS-OD-0001,Phase_II,JP-2018-1000,ORPHA:77,Gaucher Disease Type 1,E75.22,1.0,XS-maba-100,Enzyme_Replacement,Platform,2022-06-27,Completed,Biotech,PMDA,True,True,False,112,82,12,Change from baseline in disease severity score,Clinical
XS-OD-0002,Phase_III,DES-2019-1001,ORPHA:324,Fabry Disease,E75.21,0.8,XS-nibb-101,Enzyme_Replacement,Single_Arm,2021-05-04,Completed,Pharma,FDA,True,True,False,83,68,10,Biomarker response rate at Week 24,Surrogate
XS-OD-0003,Phase_III,DES-2020-1002,ORPHA:365,Pompe Disease,E74.02,0.5,XS-tideumab-102,Enzyme_Replacement,Single_Arm,2023-02-17,Completed,Government,FDA,True,False,False,48,35,5,Overall survival at 12 months,Clinical
XS-OD-0004,Phase_II,EU/3-2021-1003,ORPHA:716,Phenylketonuria,E70.0,10.0,XS-genetinib-103,Small_Molecule,Adaptive,2018-10-24,Completed,Academic,EMA,True,True,False,48,46,6,Progression-free survival,Clinical
XS-OD-0005,Phase_II,DES-2022-1004,ORPHA:646,Niemann-Pick Type C,E75.242,0.3,XS-zymeersen-104,Substrate_Reduction,Adaptive,2025-05-22,Completed,Patient_Advocacy,FDA,True,False,False,43,35,3,Composite responder index,Composite
XS-OD-0006,Phase_I,JP-2023-1005,ORPHA:399,Huntington Disease,G10,5.0,XS-statrsen-105,Antisense_Oligonucleotide,Single_Arm,2022-04-23,Active,Pharma,PMDA,False,True,False,15,15,3,Patient-reported outcome improvement >=15%,PRO
XS-OD-0007,Phase_II,EU/3-2024-1006,ORPHA:803,Amyotrophic Lateral Sclerosis,G12.21,5.0,XS-parinvec-106,Gene_Therapy,RCT,2018-05-18,Completed,Patient_Advocacy,EMA,False,False,False,77,57,9,Enzyme activity level normalization,Surrogate
XS-OD-0008,Phase_II,EU/3-2018-1007,ORPHA:98896,Duchenne Muscular Dystrophy,G71.01,2.0,XS-lukastnel-107,Exon_Skipping,RCT,2022-02-20,Suspended,Pharma,EMA,False,True,False,35,33,7,Functional capacity assessment change,Clinical
XS-OD-0009,Phase_II,DES-2019-1008,ORPHA:70,Spinal Muscular Atrophy,G12.9,1.5,XS-cillinix-108,Gene_Therapy,RCT,2019-12-28,Terminated,Government,FDA,True,True,False,62,48,7,Change from baseline in disease severity score,Clinical
XS-OD-0010,Phase_III,DES-2020-1009,ORPHA:33069,Dravet Syndrome,G40.41,0.4,XS-virol-109,Small_Molecule,Adaptive,2018-08-18,Completed,Biotech,FDA,False,False,False,101,84,13,Biomarker response rate at Week 24,Surrogate
XS-OD-0011,Phase_IV,ML-2021-1010,ORPHA:232,Sickle Cell Disease,D57.1,30.0,XS-maba-110,Gene_Therapy,Crossover,2024-12-07,Recruiting,Patient_Advocacy,Multi-jurisdictional,False,False,False,239,204,42,Overall survival at 12 months,Clinical
XS-OD-0012,Phase_I,JP-2022-1011,ORPHA:447,Paroxysmal Nocturnal Hemoglobinuria,D59.5,1.3,XS-nibb-111,Monoclonal_Antibody,Single_Arm,2023-11-11,Completed,Pharma,PMDA,True,False,False,43,34,5,Progression-free survival,Clinical
XS-OD-0013,Phase_IV,DES-2023-1012,ORPHA:848,Beta-Thalassemia Major,D56.1,4.0,XS-tideumab-112,Gene_Therapy,Adaptive,2020-06-30,Completed,Academic,FDA,False,True,False,243,218,19,Composite responder index,Composite
XS-OD-0014,Phase_I,ML-2024-1013,ORPHA:169802,Hemophilia A,D66,12.0,XS-genetinib-113,Gene_Therapy,Single_Arm,2024-09-28,Completed,Biotech,Multi-jurisdictional,False,False,False,27,26,4,Patient-reported outcome improvement >=15%,PRO
XS-OD-0015,Phase_IV,DES-2018-1014,ORPHA:169806,Hemophilia B,D67,3.0,XS-zymeersen-114,Gene_Therapy,RCT,2025-07-31,Completed,Biotech,FDA,False,False,False,134,135,19,Enzyme activity level normalization,Surrogate
XS-OD-0016,Phase_I,JP-2019-1015,ORPHA:44890,GIST (Gastrointestinal Stromal Tumor),C49.A0,1.5,XS-statrsen-115,Kinase_Inhibitor,Adaptive,2020-09-21,Completed,Biotech,PMDA,False,False,False,24,22,5,Functional capacity assessment change,Clinical
XS-OD-0017,Phase_III,DES-2020-1016,ORPHA:70567,Cholangiocarcinoma,C22.1,2.0,XS-parinvec-116,Kinase_Inhibitor,RCT,2020-04-11,Completed,Government,FDA,True,False,True,104,95,13,Change from baseline in disease severity score,Clinical
XS-OD-0018,Phase_III,DES-2021-1017,ORPHA:50251,Mesothelioma,C45.0,1.0,XS-lukastnel-117,Immune_Checkpoint,RCT,2024-04-12,Active,Biotech,FDA,False,False,False,298,214,34,Biomarker response rate at Week 24,Surrogate
XS-OD-0019,Phase_II,JP-2022-1018,ORPHA:99868,Thymic Carcinoma,C37,0.2,XS-cillinix-118,Immune_Checkpoint,Single_Arm,2018-11-10,Completed,Biotech,PMDA,False,False,False,21,16,2,Overall survival at 12 months,Clinical
XS-OD-0020,Phase_I,ML-2023-1019,ORPHA:635,Neuroblastoma,C74.90,1.0,XS-virol-119,Monoclonal_Antibody,Adaptive,2023-12-25,Recruiting,Biotech,Multi-jurisdictional,False,False,False,23,23,3,Progression-free survival,Clinical
XS-OD-0021,Phase_IV,DES-2024-1020,ORPHA:156152,ANCA Vasculitis,M31.3,5.0,XS-maba-120,Monoclonal_Antibody,RCT,2021-12-14,Completed,Pharma,FDA,False,False,False,181,171,28,Composite responder index,Composite
XS-OD-0022,Phase_I,DES-2018-1021,ORPHA:91378,Hereditary Angioedema,D84.1,2.0,XS-nibb-121,Monoclonal_Antibody,Single_Arm,2025-12-10,Active,Pharma,FDA,False,False,False,20,14,3,Patient-reported outcome improvement >=15%,PRO
XS-OD-0023,Phase_I,EU/3-2019-1022,ORPHA:208650,CAPS (Cryopyrin-Associated Periodic Syndromes),E85.0,0.3,XS-tideumab-122,IL1_Inhibitor,Basket,2023-12-22,Completed,Government,EMA,False,False,False,22,15,2,Enzyme activity level normalization,Surrogate
XS-OD-0024,Phase_I,DES-2020-1023,ORPHA:2032,Idiopathic Pulmonary Fibrosis,J84.112,15.0,XS-genetinib-123,Small_Molecule,Single_Arm,2021-01-31,Terminated,Patient_Advocacy,FDA,False,False,False,26,27,5,Functional capacity assessment change,Clinical
XS-OD-0025,Phase_I,DES-2021-1024,ORPHA:182090,Pulmonary Arterial Hypertension,I27.0,5.0,XS-zymeersen-124,Endothelin_Receptor_Antagonist,Adaptive,2025-12-14,Active,Pharma,FDA,True,False,False,15,13,2,Change from baseline in disease severity score,Clinical
XS-OD-0026,Phase_I_II,JP-2022-1025,ORPHA:77,Gaucher Disease Type 1,E75.22,1.0,XS-statrsen-125,Enzyme_Replacement,Single_Arm,2019-09-20,Active,Academic,PMDA,False,False,False,52,46,6,Biomarker response rate at Week 24,Surrogate
XS-OD-0027,Phase_III,DES-2023-1026,ORPHA:324,Fabry Disease,E75.21,0.8,XS-parinvec-126,Enzyme_Replacement,Single_Arm,2019-11-05,Recruiting,Patient_Advocacy,FDA,False,False,True,146,108,19,Overall survival at 12 months,Clinical
XS-OD-0028,Phase_II,EU/3-2024-1027,ORPHA:365,Pompe Disease,E74.02,0.5,XS-lukastnel-127,Enzyme_Replacement,Single_Arm,2021-11-09,Active,Pharma,EMA,False,False,False,42,42,7,Progression-free survival,Clinical
XS-OD-0029,Phase_I,DES-2018-1028,ORPHA:716,Phenylketonuria,E70.0,10.0,XS-cillinix-128,Small_Molecule,Single_Arm,2024-02-20,Terminated,Biotech,FDA,False,False,False,22,21,2,Composite responder index,Composite
XS-OD-0030,Phase_I,JP-2019-1029,ORPHA:646,Niemann-Pick Type C,E75.242,0.3,XS-virol-129,Substrate_Reduction,Single_Arm,2019-11-18,Active,Pharma,PMDA,False,False,False,26,26,2,Patient-reported outcome improvement >=15%,PRO
XS-OD-0031,Phase_III,ML-2020-1030,ORPHA:399,Huntington Disease,G10,5.0,XS-maba-130,Antisense_Oligonucleotide,RCT,2019-08-27,Active,Biotech,Multi-jurisdictional,False,False,False,258,220,43,Enzyme activity level normalization,Surrogate
XS-OD-0032,Phase_II,DES-2021-1031,ORPHA:803,Amyotrophic Lateral Sclerosis,G12.21,5.0,XS-nibb-131,Gene_Therapy,RCT,2023-09-10,Completed,Biotech,FDA,True,False,False,104,92,8,Functional capacity assessment change,Clinical
XS-OD-0033,Phase_III,EU/3-2022-1032,ORPHA:98896,Duchenne Muscular Dystrophy,G71.01,2.0,XS-tideumab-132,Exon_Skipping,RCT,2025-05-03,Completed,Government,EMA,False,False,False,269,266,65,Change from baseline in disease severity score,Clinical
XS-OD-0034,Phase_III,EU/3-2023-1033,ORPHA:70,Spinal Muscular Atrophy,G12.9,1.5,XS-genetinib-133,Gene_Therapy,RCT,2023-07-30,Completed,Government,EMA,True,False,False,233,177,25,Biomarker response rate at Week 24,Surrogate
XS-OD-0035,Phase_III,EU/3-2024-1034,ORPHA:33069,Dravet Syndrome,G40.41,0.4,XS-zymeersen-134,Small_Molecule,Single_Arm,2023-11-04,Completed,Biotech,EMA,False,True,False,63,56,4,Overall survival at 12 months,Clinical
XS-OD-0036,Phase_I_II,DES-2018-1035,ORPHA:232,Sickle Cell Disease,D57.1,30.0,XS-statrsen-135,Gene_Therapy,Single_Arm,2022-07-04,Completed,Biotech,FDA,False,False,False,58,51,6,Progression-free survival,Clinical
XS-OD-0037,Phase_IV,ML-2019-1036,ORPHA:447,Paroxysmal Nocturnal Hemoglobinuria,D59.5,1.3,XS-parinvec-136,Monoclonal_Antibody,RCT,2021-09-18,Completed,Academic,Multi-jurisdictional,True,False,False,154,136,20,Composite responder index,Composite
XS-OD-0038,Phase_III,DES-2020-1037,ORPHA:848,Beta-Thalassemia Major,D56.1,4.0,XS-lukastnel-137,Gene_Therapy,Platform,2024-12-23,Active,Biotech,FDA,True,False,True,285,275,43,Patient-reported outcome improvement >=15%,PRO
XS-OD-0039,Phase_III,DES-2021-1038,ORPHA:169802,Hemophilia A,D66,12.0,XS-cillinix-138,Gene_Therapy,Adaptive,2023-05-27,Active,Biotech,FDA,False,False,False,251,200,25,Enzyme activity level normalization,Surrogate
XS-OD-0040,Phase_IV,JP-2022-1039,ORPHA:169806,Hemophilia B,D67,3.0,XS-virol-139,Gene_Therapy,RCT,2021-06-12,Active,Government,PMDA,False,True,False,233,206,44,Functional capacity assessment change,Clinical
XS-OD-0041,Phase_III,DES-2023-1040,ORPHA:44890,GIST (Gastrointestinal Stromal Tumor),C49.A0,1.5,XS-maba-140,Kinase_Inhibitor,Platform,2024-11-13,Active,Biotech,FDA,True,False,False,130,105,18,Change from baseline in disease severity score,Clinical
XS-OD-0042,Phase_III,ML-2024-1041,ORPHA:70567,Cholangiocarcinoma,C22.1,2.0,XS-nibb-141,Kinase_Inhibitor,RCT,2021-12-27,Completed,Biotech,Multi-jurisdictional,False,False,True,285,296,57,Biomarker response rate at Week 24,Surrogate
XS-OD-0043,Phase_II,ML-2018-1042,ORPHA:50251,Mesothelioma,C45.0,1.0,XS-tideumab-142,Immune_Checkpoint,RCT,2025-12-11,Terminated,Patient_Advocacy,Multi-jurisdictional,False,True,False,52,44,8,Overall survival at 12 months,Clinical
XS-OD-0044,Phase_I,DES-2019-1043,ORPHA:99868,Thymic Carcinoma,C37,0.2,XS-genetinib-143,Immune_Checkpoint,Single_Arm,2023-09-15,Terminated,Biotech,FDA,True,False,False,26,21,2,Progression-free survival,Clinical
XS-OD-0045,Phase_III,DES-2020-1044,ORPHA:635,Neuroblastoma,C74.90,1.0,XS-zymeersen-144,Monoclonal_Antibody,RCT,2021-04-11,Active,Government,FDA,True,True,True,139,124,30,Composite responder index,Composite
XS-OD-0046,Phase_II,DES-2021-1045,ORPHA:156152,ANCA Vasculitis,M31.3,5.0,XS-statrsen-145,Monoclonal_Antibody,Adaptive,2024-07-29,Completed,Biotech,FDA,True,False,False,105,83,12,Patient-reported outcome improvement >=15%,PRO
XS-OD-0047,Phase_III,DES-2022-1046,ORPHA:91378,Hereditary Angioedema,D84.1,2.0,XS-parinvec-146,Monoclonal_Antibody,RCT,2023-11-12,Completed,Pharma,FDA,False,False,False,225,231,51,Enzyme activity level normalization,Surrogate
XS-OD-0048,Phase_I,DES-2023-1047,ORPHA:208650,CAPS (Cryopyrin-Associated Periodic Syndromes),E85.0,0.3,XS-lukastnel-147,IL1_Inhibitor,Single_Arm,2020-02-03,Recruiting,Biotech,FDA,False,False,False,30,24,3,Functional capacity assessment change,Clinical
XS-OD-0049,Phase_III,ML-2024-1048,ORPHA:2032,Idiopathic Pulmonary Fibrosis,J84.112,15.0,XS-cillinix-148,Small_Molecule,Platform,2021-10-31,Active,Pharma,Multi-jurisdictional,False,False,False,253,184,27,Change from baseline in disease severity score,Clinical
XS-OD-0050,Phase_II_III,DES-2018-1049,ORPHA:182090,Pulmonary Arterial Hypertension,I27.0,5.0,XS-virol-149,Endothelin_Receptor_Antagonist,RCT,2023-08-28,Recruiting,Biotech,FDA,True,False,False,139,136,21,Biomarker response rate at Week 24,Surrogate