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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Cranium; Deformity Clear all
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCT05820152
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NFS-02 Injection
Drug
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Jul 21, 2026, 6:50 AM EDT
Completed No phase listed Observational

Monitoring System for Cranial Orthoses

NCT06425172
View directory record Official record
Conditions
Plagiocephaly, Brachycephaly, Deformational Plagiocephaly, Deformational Posterior Plagiocephaly
Interventions
Cranial Remolding Orthosis
Device
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
3 Months to 18 Months
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Terminated Phase 2 Interventional Results available

Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

NCT03095248
View directory record Official record
Conditions
Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma, Glioma
Interventions
Selumetinib
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years to 45 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 21, 2025 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 2 Interventional Results available

Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma

NCT00589784
View directory record Official record
Conditions
CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma, Neurofibromatosis
Interventions
Sunitinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
6
States / cities
Boston, Massachusetts • Basking Ridge, New Jersey • Commack, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2016 · Synced Jul 21, 2026, 6:50 AM EDT
No Longer Available No phase listed Expanded access

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

NCT02300753
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
EPI-743
Drug
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
8 Years to 65 Years
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2014 · Synced Jul 21, 2026, 6:50 AM EDT
Completed No phase listed Observational

Study on Moebius Syndrome and Congenital Facial Weakness Disorders

NCT02055248
View directory record Official record
Conditions
Brain Disorders, Birth Defects, Craniofacial Differences
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
207 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 2 Interventional

Eye Muscle Surgery to Treat Congenital Nystagmus

NCT00001866
View directory record Official record
Conditions
Nystagmus
Interventions
horizontal muscles of the eye will be cut and then reattached in the same position
Procedure
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 21, 2026, 6:50 AM EDT
Not listed No phase listed Observational

NF2 Natural History Consortium

NCT00004483
View directory record Official record
Conditions
Schwannoma, Vestibular, Neurofibromatosis 2, Meningioma
Interventions
Not listed
Lead sponsor
House Ear Institute
Other
Eligibility
5 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 28, 2005 · Synced Jul 21, 2026, 6:50 AM EDT
Active, not recruiting No phase listed Observational

MElanoma Research Lymph Node Prediction Implementation National_001

NCT04759781
View directory record Official record
Conditions
Melanoma
Interventions
Not listed
Lead sponsor
SkylineDx
Industry
Eligibility
18 Years and older
Enrollment
1,820 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
9
States / cities
Tampa, Florida • Atlanta, Georgia • Lexington, Kentucky + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies

NCT03059420
View directory record Official record
Conditions
Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome, Duane Radial Ray Syndrome, Mobius Syndrome, Brown Syndrome, Marcus Gunn Syndrome, Strabismus Congenital, Horizontal Gaze Palsy, Horizontal Gaze Palsy With Progressive Scoliosis, Facial Palsy, Facial Paresis, Hereditary, Congenital, Third Nerve Palsy, Fourth Nerve Palsy, Sixth Nerve Palsy, Synkinesis, Ocular Motility Disorders, Levator-Medial Rectus Synkinesis, Athabaskan Brainstem Dysgenesis, Tongue Paralysis, Ninth Nerve Disorder, Fifth Nerve Palsy, Seventh Nerve Palsy, Eleventh Nerve Disorder, Twelfth Nerve Disorder, Vagus Nerve Paralysis, Moebius Sequence
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Day and older
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Completed No phase listed Observational

AONDA Therapeutic Indication Study I

NCT05891106
View directory record Official record
Conditions
Bullous Keratopathy, Corneal Erosion, Entropion, Corneal Edema, Corneal Dystrophy, Corneal Ulcer, Foreign Body in Cornea, Bell Palsy, Keratoconjunctivitis, Filamentary Keratitis, Sicca Syndrome; Keratoconjunctivitis (Etiology)
Interventions
Lotrafilcon A contact lenses
Device
Lead sponsor
Alcon Research
Industry
Eligibility
18 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
3
States / cities
Orlando, Florida • Franklin Park, Illinois • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 19, 2023 · Synced Jul 21, 2026, 6:50 AM EDT
Terminated Phase 2 Interventional Results available

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced Jul 21, 2026, 6:50 AM EDT
Recruiting Phase 2 Interventional

Gadolinium and Ferumoxytol MRI in Diagnosing Patients With Abnormalities in the Central Nervous System

NCT03270059
View directory record Official record
Conditions
Central Nervous System Neoplasm, Cranial Nerve Disorder, Metastatic Malignant Neoplasm in the Brain
Interventions
Ferumoxytol, Gadolinium, Magnetic Resonance Imaging
Drug · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
10 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas

NCT04283669
View directory record Official record
Conditions
Neurofibromatosis 2, Progressive Vestibular Schwannoma (VS)
Interventions
Crizotinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
15
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 1Phase 2 Interventional

Temozolomide Plus Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Malignant Glioma or Recurrent CNS or Other Solid Tumors

NCT00005952
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Head and Neck Cancer, Kidney Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
Interventions
filgrastim, temozolomide, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Duke University
Other
Eligibility
Up to 18 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 19, 2013 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 2 Interventional Results available

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

NCT02693119
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 29, 2021 · Synced Jul 21, 2026, 6:50 AM EDT
Recruiting Not applicable Interventional

OPT-IN: Osteopathic Plagiocephaly Treatment for Infants and Neonates

NCT05848895
View directory record Official record
Conditions
Plagiocephaly, Nonsynostotic, Plagiocephaly, Positional, Plagiocephaly, Cranium; Deformity
Interventions
Osteopathic Manipulative Treatment, Standard of Care Repositioning
Procedure · Behavioral
Lead sponsor
Osteopathy's Promise to Children
Other
Eligibility
1 Day to 4 Months
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2025 · Synced Jul 21, 2026, 6:50 AM EDT
Not listed Not applicable Interventional

Stem Cell Ophthalmology Treatment Study

NCT01920867
View directory record Official record
Conditions
Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease, Glaucoma
Interventions
RB (Retrobulbar), ST (Subtenon), IV (Intravenous), IVIT (Intravitreal), IO (Intraocular)
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Westport, Connecticut
Source: ClinicalTrials.gov public record
Updated Oct 22, 2019 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

NCT02652767
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Jul 21, 2026, 6:50 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

New Non-invasive Modalities for Assessing Retinal Structure and Function

NCT03475173
View directory record Official record
Conditions
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
Interventions
LSFG-NAVI
Device
Lead sponsor
Randy Kardon
Other
Eligibility
18 Years to 99 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2028
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 1Phase 2 Interventional

Chemotherapy in Treating Patients With Solid Tumors

NCT00003103
View directory record Official record
Conditions
Bladder Cancer, Breast Cancer, Colorectal Cancer, Esophageal Cancer, Head and Neck Cancer, Kidney Cancer, Lung Cancer, Ovarian Cancer, Prostate Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
oblimersen sodium, docetaxel
Biological · Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2002
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2013 · Synced Jul 21, 2026, 6:50 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 16, 2022 · Synced Jul 21, 2026, 6:50 AM EDT
Completed Phase 1 Interventional

Liposomal Doxorubicin in Treating Children With Refractory Solid Tumors

NCT00019630
View directory record Official record
Conditions
Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer, Brain Tumor, Kidney Tumor
Interventions
doxorubicin HCl liposome
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Timeline
Started 1999
U.S. locations
2
States / cities
Bethesda, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 3, 2024 · Synced Jul 21, 2026, 6:50 AM EDT