Nervous System Malformations: Clinical Trials

393 completed, 164 recruiting, 117 unknown, 67 terminated. 250 Not applicable, 158 Phase 2, 94 Phase 1, 60 Phase 3, 18 Phase 4.

By phase and status

Phase	Trials	Status
Not applicable	250	Completed 117, Recruiting 43, Unknown 42, Active not recruiting 16, Not yet recruiting 11, Terminated 9, Withdrawn 7, Enrolling by invitation 5
Phase 2	158	Completed 70, Recruiting 24, Terminated 22, Unknown 12, Active not recruiting 11, Not yet recruiting 9, Withdrawn 8, Enrolling by invitation 1, Suspended 1
Phase 1	94	Completed 45, Recruiting 20, Terminated 13, Active not recruiting 7, Not yet recruiting 4, Unknown 2, Withdrawn 2, Suspended 1
Phase 3	60	Completed 25, Recruiting 10, Unknown 10, Terminated 9, Withdrawn 3, Not yet recruiting 2, Active not recruiting 1
Phase 4	18	Completed 9, Unknown 6, Active not recruiting 1, Recruiting 1, Terminated 1
Early Phase 1	6	Completed 2, Not yet recruiting 2, Recruiting 1, Terminated 1

Follow Nervous System Malformations: new trials by email Literature Review of Nervous System Malformations trials Research Report on Nervous System Malformations

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Parenting Program for Young Children With Spina Bifida Positive Parenting Program - Spina Bifida	Not applicable	Not yet recruiting	Phoenix Children's Hospital	30	2028-01-01	2026-11-04
Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A scAAV1.tMCK.NTF3 - Charcot-Marie-Tooth Neuropathy Type 1A	Phase 1 / Phase 2	Withdrawn	Nationwide Children's Hospital		2027-04-01	2026-07-29
Fetal Embolization for Severe Vein of Galen Malformation - A Feasibility and Safety Trial Fetal embolization of severe Vein of Galen Malformation using a liquid embolic system - Vein of Galen Malformation	Not applicable	Not yet recruiting	Michael A Belfort	10	2027-03-01	2026-11-10
Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults Mirdametinib - LGCMN, Large and Giant Congenital Melanocytic Nevi	Phase 2	Not yet recruiting	National Cancer Institute (NCI)	15	2027-01-04	2026-10-29
Bioengineered Penile Tissue Constructs for Irreversibly Damaged Penile Corpora Autologous tissue engineered corpora - Urologic Diseases, Male Urogenital Diseases	Phase 1	Not yet recruiting	Wake Forest University Health Sciences	10	2027-01-01	2026-10-23
Aspirin and PAClitaxel in Head & Neck Squamous Cell Carcinoma Aspirin, Paclitaxel - Head and Neck (HNSCC), Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M)	Phase 2	Not yet recruiting	Washington University School of Medicine	40	2026-12-31	2026-10-28
Muscle MRI Fat Fraction Before Surgery to Predict Strength Recovery After Foot-Drop Surgery in Charcot-Marie-Tooth Disease Tibialis posterior tendon transfer, Individualized adjunct procedures for cavovarus deformity correction, Ankle-foot orthosis (AFO) - Foot Drop, Charcot-Marie-Tooth Disease Type 1A		Not yet recruiting	Peking University Third Hospital	100	2026-12-01	2026-10-26
Study to Establish Safety, Tolerability and Pharmacokinetics of NRL-1049 in Healthy Adult Participants Following Multiple-Dose Administration NRL-1049, Placebo - Cerebral Cavernous Malformation	Phase 1	Not yet recruiting	Neurelis, Inc.	40	2026-11-11	2026-10-30
Clinical, Electrophysiological and Genetic Characteristics of Hereditary and Inflammatory Polyneuropathies in France Genetic and Inflammatory Polyneuropathies		Not yet recruiting	Assistance Publique - Hôpitaux de Paris	1000	2026-11-01	2026-10-01
China Treatment Trial for Tumor in Sellar Region With Unruptured Intracranial Aneurysm Tumor resection followed by aneurysm treatment, Aneurysm treatment followed by tumor resection, Observation - Unruptured Intracranial Aneurysms, Pituitary Neuroendocrine Tumor		Active not recruiting	Cheng sen	608	2026-10-20	2026-10-21
Motor Imagery-Supported Thoracic Expansion in Children With Spina Bifida Thoracic Expansion Exercises, Motor Imagery-Supported Thoracic Expansion Exercises - Spina Bifida	Not applicable	Not yet recruiting	Karabuk University	30	2026-10-12	2026-09-29
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province Motor Neuron Diseases, Spinocerebellar Ataxias		Recruiting	First Affiliated Hospital of Wannan Medical College	180	2026-09-30	2026-10-23
Abnormality of the Central Canal of the Spinal Cord in Syringomyelia in a Patient With a Basal Skull Malformation MRI - Chiari Malformation Type I, Syringomyelia	Not applicable	Not yet recruiting	University Hospital, Bordeaux	50	2026-09-30	2026-07-22
tSCS in Children With Spina Bifida Transcutaneous stimulator - Spina Bifida, Myelomeningocele	Not applicable	Recruiting	Bailey Petersen	20	2026-09-30	2026-09-15
Cryotherapy, PGE2, NGF Levels and Pain After Endodontic Retreatment Intracanal cryotherapy, Room-temperature saline irrigation - Asymptomatic Apical Periodontitis, Endodontic Retreatment	Not applicable	Recruiting	Alanya Alaaddin Keykubat University	100	2026-09-22	2026-10-27
Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics Sturge-Weber Syndrome, Neurocutaneous Syndromes		Not yet recruiting	Assiut University	100	2026-09-20	2026-09-28
First-Trimester Screening of Open Neural Tube Defects Using Biometrics and Maternal Serum Markers Neural Tube Defects (NTDs)		Recruiting	University Hospital, Strasbourg, France	100	2026-09-14	2026-10-07
Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1) AV078, Placebo - Tuberous Sclerosis Complex, Epilepsy	Phase 2	Not yet recruiting	Aeovian Pharmaceuticals, Inc.	42	2026-09-01	2026-09-29
Concurrent Training vs Soleus Push-Ups on Neurogenesis-Related Biomarkers in Diabetic Neuropathy Patients Concurrent Training, Soleus Push-ups - Diabetic Peripheral Neuropathy Type 2	Not applicable	Not yet recruiting	Riphah International University	99	2026-09-01	2026-06-30
Fluorescence Guided Focal Cortical Dysplasia Surgery 5-ALA (Gliolan) - Drug Resistant Epilepsy	Phase 2	Not yet recruiting	Hospices Civils de Lyon	5	2026-09-01	2026-06-18

Showing 20 of 859. Sign in to see all of them.

Narrower conditions

Hereditary Sensory and Autonomic Neuropathies (175)  ·  Spinal Dysraphism (140)  ·  Tuberous Sclerosis (114)  ·  Charcot-Marie-Tooth Disease (112)  ·  Meningomyelocele (58)  ·  Spastic Paraplegia, Hereditary (49)  ·  Neural Tube Defects (47)  ·  Cerebral Palsy (45)  ·  Intracranial Arteriovenous Malformations (43)  ·  Epilepsy (42)  ·  Spinal Cord Injuries (41)  ·  Arnold-Chiari Malformation (34)  ·  Congenital Abnormalities (24)  ·  Microcephaly (22)  ·  Urinary Bladder, Neurogenic (22)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (857)  ·  Nervous System Diseases (850)  ·  Congenital Abnormalities (835)  ·  Genetic Diseases, Inborn (500)  ·  Neurodegenerative Diseases (469)  ·  Heredodegenerative Disorders, Nervous System (466)  ·  Neuromuscular Diseases (364)  ·  Peripheral Nervous System Diseases (351)  ·  Polyneuropathies (351)  ·  Central Nervous System Diseases (254)  ·  Pathological Conditions, Signs and Symptoms (235)  ·  Neoplasms (224)  ·  Brain Diseases (205)  ·  Neural Tube Defects (190)

Interventions

Everolimus (22)  ·  Sirolimus (20)  ·  Fulvestrant (10)  ·  Alpelisib (9)  ·  Blood Specimen Collection (9)  ·  Surveys and Questionnaires (8)  ·  Cannabidiol (7)  ·  tanezumab (7)  ·  Cetuximab (6)  ·  Fetoscopy (6)  ·  Nerve Growth Factor (6)  ·  Saline Solution (6)  ·  Exercise (5)  ·  Folic Acid (5)  ·  Letrozole (4)

Sponsors

Assistance Publique - Hôpitaux de Paris (23)  ·  Novartis Pharmaceuticals (18)  ·  NYU Langone Health (16)  ·  Beijing Tiantan Hospital (9)  ·  National Institute of Neurological Disorders and Stroke (NINDS) (9)  ·  Pfizer (9)  ·  Boston Children's Hospital (8)  ·  Rennes University Hospital (8)  ·  University Hospital, Clermont-Ferrand (8)  ·  AstraZeneca (7)  ·  Children's Hospital Medical Center, Cincinnati (7)  ·  National Heart, Lung, and Blood Institute (NHLBI) (7)  ·  National Human Genome Research Institute (NHGRI) (7)  ·  Assiut University (6)  ·  Baylor College of Medicine (6)

Countries

United States (376)  ·  France (135)  ·  Italy (63)  ·  China (62)  ·  Canada (60)  ·  United Kingdom (58)  ·  Spain (56)  ·  Germany (50)  ·  Netherlands (40)  ·  Turkey (Türkiye) (39)  ·  Belgium (37)  ·  Australia (36)  ·  Poland (22)  ·  Denmark (21)  ·  South Korea (20)

Top investigators

Horacio Kaufmann, MD (5)  ·  Dana Anaby, PhD (4)  ·  Michael E Shy, MD (4)  ·  Darcy Krueger, MD, PhD (3)  ·  Joel Moss, M.D. (3)  ·  Shuo Wang, MD (3)  ·  Thomas C Bulea, Ph.D. (3)  ·  A. Reghan Foley, M.D. (2)  ·  Adeline Vanderver, MD (2)  ·  Alejandra Gonzalez-Duarte, MD (2)  ·  Allison Ashley-Koch, PhD (2)

Results publications

PMID 21306277, PMID 30936738, PMID 26554876, PMID 29023494, PMID 37460782, PMID 41491634, PMID 24327518, PMID 25519680, PMID 27424289, PMID 28672195, PMID 29383130, PMID 29846250, PMID 30053159, PMID 30291925, PMID 31101621, PMID 31379715, PMID 31533931, PMID 31831767, PMID 31882719, PMID 31894292, PMID 31912454, PMID 34162982, PMID 34957550, PMID 37395948, PMID 38507752, PMID 38996393, PMID 39486058, PMID 10389801, PMID 10411196, PMID 10540002, PMID 10739772, PMID 11232233, PMID 11344264, PMID 1135616, PMID 11382253, PMID 11383973, PMID 11605920, PMID 11892506, PMID 12235608, PMID 12473512, PMID 12671856, PMID 12851579, PMID 13211501, PMID 14501247, PMID 14564276, PMID 14694254, PMID 14738943, PMID 15098220, PMID 15610704, PMID 15680560

NIH funding

2017 (6), 2023 (6), 2025 (6), 2015 (5), 2018 (5), U54NS065712 (4), U54NS065705 (3), 2008 (2), 2016 (2), 2019 (2), U01TR001263 (2), U54NS092090 (2), U54NS092091 (2), 2009 (1), 5RC1NS068179-02 (1)