TheraRadar

Pharma Intelligence, Simplified

Landscape Rare Disease

Spinal Muscular Atrophy

334 clinical trials

151 active
/
334 total (since 2015)
44
Phase 1 Active
98 total
89
Phase 2 Active
172 total
37
Phase 3 Active
84 total
11
Phase 4 Active
22 total

Top Sponsors (Industry)

Sorted by active Active Done Failed
Roche 6 3 0
Wuhan Createrna Science and Technology Co., Ltd 5 4 0
Novartis 4 10 0
Regeneron 4 3 2
GeneCradle Inc 4 0 0
Chongqing Precision Biotech Co., Ltd 4 0 0
Nanjing Chia-tai Tianqing Pharmaceutical 3 0 0
NovelMed Therapeutics 3 0 0
AstraZeneca 2 12 1
Biogen 2 2 1
Haisco Pharmaceutical Group Co., Ltd. 2 3 0
Scholar Rock, Inc. 2 2 0
Longbio Pharma 2 1 0
Lantu Biopharma 2 0 0
Novartis Gene Therapies 1 4 1
NCT05331937 RECRUITING
TMS for Exposure Therapy Resistant OCD
Amsterdam UMC, location VUmc n=250
NCT07448415 NOT YET RECRUITING
Evaluation of the Efficacy of Oral Tranexamic Acid and Glutathione-assisted Microneedling in Treatment of Melasma
Assiut University n=42
NCT07444476 NOT YET RECRUITING
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
Biogen n=90
NCT05389449 ACTIVE NOT RECRUITING
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
Alexion Pharmaceuticals, Inc. n=80
NCT07154745 RECRUITING
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
Regeneron Pharmaceuticals n=35
NCT05133531 RECRUITING
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
Regeneron Pharmaceuticals n=190
NCT05744921 RECRUITING
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Regeneron Pharmaceuticals n=202
NCT03480360 ACTIVE NOT RECRUITING
Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators' Expression
Dartmouth-Hitchcock Medical Center n=21
NCT04434092 ACTIVE NOT RECRUITING
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Hoffmann-La Roche n=210
NCT04654468 ACTIVE NOT RECRUITING
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
Hoffmann-La Roche n=51
NCT05337553 ACTIVE NOT RECRUITING
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Biohaven Pharmaceuticals, Inc. n=269
NCT04432584 ACTIVE NOT RECRUITING
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Hoffmann-La Roche n=190
NCT05335876 RECRUITING
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Novartis Pharmaceuticals n=175
NCT06934967 RECRUITING
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Novartis Pharmaceuticals n=12
NCT06449001 RECRUITING
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
Alexion Pharmaceuticals, Inc. n=6
NCT05115110 ACTIVE NOT RECRUITING
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Hoffmann-La Roche n=259
NCT07265232 RECRUITING
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Lantu Biopharma n=15
NCT07249853 NOT YET RECRUITING
Cilostazol for Preventing Delayed Cerebral Ischemia in Aneurysmal Subarachnoid Hemorrhage
Beijing Tiantan Hospital n=316
NCT07177859 NOT YET RECRUITING
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
Nanjing Chia-tai Tianqing Pharmaceutical n=78
NCT07177872 NOT YET RECRUITING
A Long-term Efficacy and Safety of NTQ5082 Capsules
Nanjing Chia-tai Tianqing Pharmaceutical n=78
NCT07052214 RECRUITING
PSMA PET Combined With MRI for the Detection of PCa
Telix Pharmaceuticals (Innovations) Pty Limited n=204
NCT07144956 NOT YET RECRUITING
Cilostazol With Nimodipine to Improve Outcome After Aneurysmal Subarachnoid Hemorrhage
Centre Hospitalier St Anne n=630
NCT07005661 NOT YET RECRUITING
Use of PRP in Open Surgery for Type A Aortic Dissection
Beijing Anzhen Hospital n=250
NCT06971094 RECRUITING
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
GeneCradle Inc n=50
NCT05626855 ACTIVE NOT RECRUITING
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
Scholar Rock, Inc. n=238
NCT06932744 RECRUITING
Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
Wuhan Createrna Science and Technology Co., Ltd n=66
NCT06932471 RECRUITING
Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
Wuhan Createrna Science and Technology Co., Ltd n=20
NCT05067790 ACTIVE NOT RECRUITING
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
Biogen n=45
NCT04042025 ACTIVE NOT RECRUITING
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Novartis Gene Therapies n=85
NCT06933914 RECRUITING
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
Wuhan Createrna Science and Technology Co., Ltd n=120
NCT04747613 ACTIVE NOT RECRUITING
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
Novartis Pharmaceuticals n=208
NCT06762405 RECRUITING
PRODIGE 90 - (FFCD 2204) Neoadjuvant Dostarlimab with Short Course Radiotherapy in a Watch-and-wait Strategy for Microsatellite Unstable or Mismatch Repair-deficient Locally Advanced Rectal Cancer Patients
Centre Hospitalier Universitaire Dijon n=68
NCT05995405 ACTIVE NOT RECRUITING
Safety and Tolerability of GTX-104 Compared with Oral Nimodipine in Patients with ASAH
Grace Therapeutics Inc. n=100
NCT06559072 NOT YET RECRUITING
Ultra-early STatin in Patients With Aneurysmal subaRachnoid Hemorrhage (Ue-STAR)
The George Institute n=522
NCT05622162 ACTIVE NOT RECRUITING
Prospective Comparative Study for Patients With Biochemical Recurrence Prostate Cancer Detecting by 18F-JK-PSMA-7
ITEL Telecomunicazioni Srl n=106
NCT06138353 RECRUITING
Shuxuening Injection for the Prevention of CVS in Patients With aSAH(SXN-CVS)
Xiaolin Chen, MD n=50
NCT02544308 ACTIVE NOT RECRUITING
Trial of Immunomodulatory Therapy in High Risk Solitary Bone Plasmacytoma
University College, London n=36
NCT07052838 COMPLETED
Efficacy and Safety of HSK39297 in Anti-C5 Treated PNH Patients With Anemia
Haisco Pharmaceutical Group Co., Ltd. n=36
NCT03306277 COMPLETED
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Novartis Gene Therapies n=22
NCT03837184 COMPLETED
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Novartis Gene Therapies n=2
NCT03505099 COMPLETED
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
Novartis Gene Therapies n=30
NCT03461289 COMPLETED
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Novartis Gene Therapies n=33
NCT05156320 COMPLETED
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
Scholar Rock, Inc. n=188
NCT06578949 COMPLETED
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Alexion Pharmaceuticals, Inc. n=18
NCT05630001 COMPLETED
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
Novartis Pharmaceuticals n=52
NCT05386680 COMPLETED
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
Novartis Pharmaceuticals n=27
NCT05089656 COMPLETED
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
Novartis Pharmaceuticals n=126
NCT06715943 COMPLETED
Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment
Chengdu Suncadia Medicine Co., Ltd. n=39
NCT06593938 COMPLETED
A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
Chengdu Suncadia Medicine Co., Ltd. n=76
NCT06987864 COMPLETED
An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
Biocad n=24
NCT04469465 COMPLETED
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
Alexion Pharmaceuticals, Inc. n=86
NCT05886244 COMPLETED
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Alexion Pharmaceuticals, Inc. n=25
NCT03829449 TERMINATED
rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study
AKARI Therapeutics n=15
NCT03588026 COMPLETED
Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576
AKARI Therapeutics n=9
NCT05131204 TERMINATED
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
Regeneron Pharmaceuticals n=3
NCT04141371 COMPLETED
Molar Sodium Lactate Filling in Severe Meningeal Hemorrhage
Centre Hospitalier Universitaire de Nice n=3
NCT03056040 COMPLETED
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Alexion Pharmaceuticals, Inc. n=202
NCT05134649 COMPLETED
A Study To Assess the Safety of OnabotulinumtoxinA (BOTOX) Intramuscular Injection in Adult Participants With Platysma Prominence
AbbVie n=292
NCT04949399 COMPLETED
A Study To Assess the Safety and Effects of OnabotulinumtoxinA (BOTOX) Intramuscular Injection in Adult Participants With Moderate to Severe Platysma Prominence
AbbVie n=408
NCT02594124 COMPLETED
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
Biogen n=292
NCT04851873 COMPLETED
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
Novartis Pharmaceuticals n=24
NCT04820530 COMPLETED
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
Novartis Pharmaceuticals n=40
NCT04558918 COMPLETED
Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
Novartis Pharmaceuticals n=97
NCT03748823 COMPLETED
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Alexion Pharmaceuticals, Inc. n=139
NCT04994535 COMPLETED
A Study To Assess the Safety and Effects of OnabotulinumtoxinA (BOTOX) Intramuscular Injection in Adult Participants With Platysma Prominence
AbbVie n=426
NCT02946463 COMPLETED
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Alexion Pharmaceuticals, Inc. n=272
NCT04058158 COMPLETED
A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria
Samsung Bioepis Co., Ltd. n=50
NCT03585270 COMPLETED
Clinical Research Study With Clazosentan to Evaluate Its Effects on Preventing Complications Due to the Narrowing of the Blood Vessels (Vasospasm) in the Brain, Caused by Bleeding Onto the Surface of the Brain
Idorsia Pharmaceuticals Ltd. n=409
NCT05969587 COMPLETED
Cysteamine Compared to Hydroquinone in Melasma
China Medical University Hospital n=28
NCT04162470 TERMINATED
REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.
Regeneron Pharmaceuticals n=24
NCT03818607 COMPLETED
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
Amgen n=42
NCT05032118 WITHDRAWN
Pilot Study of Ketamine Sedation for Aneurysmal Subarachnoid Hemorrhage
Jenna L Leclerc MD, PhD
NCT03406507 COMPLETED
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
Alexion Pharmaceuticals, Inc. n=13
NCT04085601 COMPLETED
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Apellis Pharmaceuticals, Inc. n=53
NCT03500549 COMPLETED
Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Apellis Pharmaceuticals, Inc. n=80
NCT04463056 COMPLETED
Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH
AO GENERIUM n=32
NCT04816942 COMPLETED
Clinical Efficacy of Early Administration of Convalescent Plasma Among COVID-19 Cases in Egypt
Ministry of Health and Population, Egypt n=102
NCT04060264 COMPLETED
Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria
Biocad n=28
NCT04679103 COMPLETED
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
AO GENERIUM n=50
NCT03751163 COMPLETED
Oral Tranexamic Acid and Topical Hydroquinone in the Treatment of Melasma
Fakultas Kedokteran Universitas Indonesia n=50
NCT02790632 TERMINATED
Study of EG-1962 Compared to Standard of Care Oral Nimodipine in Adults With Aneurysmal Subarachnoid Hemorrhage
Edge Therapeutics Inc n=374
NCT03594240 COMPLETED
Vitamin B Complex and Diabetic Nephropathy in Type 1 Diabetes
Ain Shams University n=80
NCT02931149 COMPLETED
Evaluation of the Efficacy of Platelet-rich Plasma (PRP) on Advanced Endoscopic Resection Techniques
Germans Trias i Pujol Hospital n=11
NCT03220334 COMPLETED
Healing Effects of Autologous Platelet Rich Plasma on Gastric Ulcer Caused by Endoscopic Submucosal Dissection
Keimyung University Dongsan Medical Center n=20