Nervous System Malformations: Clinical Trials

389 completed, 166 recruiting, 117 unknown, 67 terminated. 248 Not applicable, 156 Phase 2, 93 Phase 1, 60 Phase 3, 18 Phase 4.

By phase and status

Phase	Trials	Status
Not applicable	248	Completed 116, Recruiting 45, Unknown 42, Active not recruiting 14, Not yet recruiting 10, Terminated 9, Withdrawn 7, Enrolling by invitation 5
Phase 2	156	Completed 69, Recruiting 25, Terminated 22, Unknown 12, Active not recruiting 11, Withdrawn 8, Not yet recruiting 7, Enrolling by invitation 1, Suspended 1
Phase 1	93	Completed 45, Recruiting 20, Terminated 13, Active not recruiting 7, Not yet recruiting 3, Unknown 2, Withdrawn 2, Suspended 1
Phase 3	60	Completed 25, Recruiting 10, Unknown 10, Terminated 9, Withdrawn 3, Not yet recruiting 2, Active not recruiting 1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 8, Unknown 6, Active not recruiting 2, 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
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of scAAV1.tMCK.NTF3 for Treatment of CMT1A scAAV1.tMCK.NTF3 - Charcot-Marie-Tooth Neuropathy Type 1A	Phase 1 / Phase 2	Withdrawn	Nationwide Children's Hospital		2027-04-01	2026-07-29
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
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	2026-10-01	2026-03-27
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
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
Clinical Investigation to Validate the Safety and Performance of the ABLE Kids Device in Pediatric Patients With Neurological Gait Disorders in a Clinical Setting ABLE Kids - Gait Impairment, Cerebral Palsy (CP)	Not applicable	Recruiting	ABLE Human Motion S.L.	15	2026-09-01	2026-09-08
Pilot Study of ZetaFuse™ Bone Graft for the Treatment of Cervical Degenerative Disc Disease Cervical Fusion (ZetaFuse™ Bone Graft) - Radiculopathy, Cervical, Myelopathy Cervical	Not applicable	Recruiting	Zetagen Therapeutics, Inc	10	2026-09-01	2025-04-24
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province Motor Neuron Diseases, Spinocerebellar Ataxias		Active not recruiting	First Affiliated Hospital of Wannan Medical College	180	2026-08-25	2026-09-24
Everolimus bAsed caLcineurin inhibiTor frEe immunosuppRession oNe Year AfTer lIver transplantatiON (ALTERNATION) Everolimus, Tacrolimus or Cyclosporine - Liver Allograft, Liver Diseases	Phase 3	Recruiting	Hannover Medical School	150	2026-08-19	2026-10-05
Postoperative Circulating Tumor DNA Monitoring in Stage I-III Driver-Mutant Colon Adenocarcinoma Longitudinal ctDNA-MRD NGS testing at scheduled postoperative time points - Colon Adenocarcinoma, Stage I Colon Cancer		Recruiting	Ruijin Hospital	392	2026-08-14	2026-09-02
Health-related Quality of Life in Chiari Malformation Type 1 - A National Cross-Sectional Patient Survey Chiari Malformation Type I		Enrolling by invitation	Karolinska Institutet	1004	2026-08-12	2026-09-14
Enema Device for Children With Spina Bifida Use of the foley catheter assistive device - Spina Bifida, Fecal Incontinence	Not applicable	Recruiting	University of Colorado, Denver	20	2026-08-11	2026-09-03
Coupled Gentamicin-Lactobacillus Rhamnosus in NLUTD Gentamicin, Lactobacillus Rhamnosus GG - Spinal Cord Injury, Neurogenic Bladder (NB)	Early Phase 1	Not yet recruiting	Medstar Health Research Institute	48	2026-08-01	2026-08-21

Showing 20 of 853. Sign in to see all of them.

Narrower conditions

Hereditary Sensory and Autonomic Neuropathies (174)  ·  Spinal Dysraphism (139)  ·  Tuberous Sclerosis (114)  ·  Charcot-Marie-Tooth Disease (111)  ·  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 (23)  ·  Microcephaly (22)  ·  Urinary Bladder, Neurogenic (22)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (852)  ·  Nervous System Diseases (844)  ·  Congenital Abnormalities (830)  ·  Genetic Diseases, Inborn (498)  ·  Neurodegenerative Diseases (467)  ·  Heredodegenerative Disorders, Nervous System (464)  ·  Neuromuscular Diseases (362)  ·  Peripheral Nervous System Diseases (349)  ·  Polyneuropathies (349)  ·  Central Nervous System Diseases (252)  ·  Pathological Conditions, Signs and Symptoms (234)  ·  Neoplasms (221)  ·  Brain Diseases (203)  ·  Neural Tube Defects (189)

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 (372)  ·  France (135)  ·  Italy (63)  ·  China (62)  ·  Canada (60)  ·  United Kingdom (58)  ·  Spain (56)  ·  Germany (50)  ·  Netherlands (40)  ·  Turkey (Türkiye) (39)  ·  Australia (37)  ·  Belgium (37)  ·  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)  ·  Adeline Vanderver, MD (2)  ·  Alejandra Gonzalez-Duarte, MD (2)  ·  Allison Ashley-Koch, PhD (2)  ·  Anna Jansen, MD, 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)