Clinical trials: Optic Neuropathies
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Retinal Diseases, Optic Nerve Diseases
Spectral Domain Optical Coherence Tomography Imaging of the Eyes of Neonates
Brief Summary The purpose of this study is to better characterize the retina and optic nerve in newborns using spectral domain optical coherence tomography (s-oct).
- Trial name
- OCT (NCT01404247)
- Testing
- Observational: to better characterize the retina and optic nerve in newborns using spectral domain optical coherence tomography (s-oct)., OCT imaging.
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Leber Hereditary Optic Neuropathy (LHON)
A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation
The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation.
- Trial name
- NCT07406854
- Testing
- rAAV2-ND4
- Where
- China
- Volunteers
- 95 people
- Sponsor
- Wuhan Neurophth Biotechnology Limited Company
Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form
A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
This is a sub-study of Study RM-493-040 (NCT05774756). The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with…
- Conditions
- Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form, Septo-Optic Dysplasia, Optic Nerve Hypoplasia
- Trial name
- NCT06760546
- Testing
- Setmelanotide
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 39 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Neurofibromatosis-1, Optic Glioma
PET/MRI in CNS and Extra-CNS Tumors of Patients With Neurofibromatosis-1 (NF1)
This prospective pilot study is designed to provide preliminary data on the use of Fluorodeoxyglucose Positron Emission Tomography-Magnetic Resonance Imaging (FDG-PET-MRI) in patients with neurofibromatosis-1 (NF1) associated…
- Conditions
- Neurofibromatosis-1, Optic Glioma, Plexiform Neurofibroma
- Trial name
- NCT01800032
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Multiple Sclerosis, NMO Spectrum Disorder
National, Multicentric Registry Study on Neuroimmunological Diseases in China
The aim of this study is to establish a real-world clinical neuroimmune disease research cohort, to follow up and observe the prognosis of patients with different subtypes and subgroups, and to provide support for the treatment…
- Conditions
- Multiple Sclerosis, NMO Spectrum Disorder, Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease, Myasthenia Gravis
- Trial name
- NIDBase (NCT06443333)
- Testing
- Data collection and follow-up observation, Data collection
- Where
- China
- Volunteers
- 7,000 people, healthy volunteers welcome
- Sponsor
- Xuanwu Hospital, Beijing
Antibody-mediated Rejection, NMO Spectrum Disorder
Biorepository and Registry for Plasma Exchange Patients
Patients who have immune mediated diseases commonly undergo plasma exchange (PLEX) procedures to remove pathological substances, typically believed to be antibodies.
- Conditions
- Antibody-mediated Rejection, NMO Spectrum Disorder, TTP, CIDP
- Trial name
- NCT05004493
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Charles M Knudson
Leber Hereditary Optic Neuropathy
RESCUE and REVERSE Long-term Follow-up
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in…
- Trial name
- RESTORE (NCT03406104)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 62 people
- Sponsor
- GenSight Biologics
Traumatic Optic Neuropathy
Traumatic Optic Neuropathy Treatment Trial 2
After introducing intravenous erythropoietin (EPO) as an option for treatment of patients with indirect traumatic optic neuropathy in 2011 and publishing non inferiority trial in Oct.2017), TONTT2 is aiming to find out the best…
- Trial name
- TONTT-2 (NCT03308448)
- Testing
- Recombinant human erythropoietin
- Where
- 3 sites in Iran
- Volunteers
- 93 people
- Sponsor
- Iran University of Medical Sciences
Healthy, Multiple Sclerosis
Normal Chinese Lifespan Brain Charts Initiative (NCLBCI)
Major brain diseases (including neuroimmune diseases, neurodegenerative diseases, and cerebrovascular diseases) are leading causes of death and disability in humans.
- Conditions
- Healthy, Multiple Sclerosis, Neuromyelitis Optica Spectrum Disorders, Neurodegenerative Diseases
- Trial name
- NCT06643858
- Testing
- No intervention
- Where
- China
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Beijing Tiantan Hospital
Brain and Central Nervous System Tumors
MK0752 in Treating Young Patients With Recurrent or Refractory CNS Cancer
RATIONALE: MK0752 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. PURPOSE: This phase I trial is studying the side effects and best dose of MK0752 in treating young patients with…
- Trial name
- NCT00572182
- Testing
- MK-0752
- Where
- 12 sites in United States
- Volunteers
- 33 people
- Sponsor
- Pediatric Brain Tumor Consortium
Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor
Irinotecan in Treating Children With Refractory Solid Tumors
This phase II trial is studying irinotecan to see how well it works in treating children with refractory solid tumors. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
- Conditions
- Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Craniopharyngioma, Childhood Grade I Meningioma
- Trial name
- NCT00004078
- Testing
- irinotecan hydrochloride
- Where
- United States
- Volunteers
- 181 people
- Sponsor
- Children's Oncology Group